Q4 2020 Amicus Therapeutics Inc Earnings Call

Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics fourth quarter 2020 fiscal year results conference call and webcast.

Operator: Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics Full Quarter 2020 Fiscal Year Results Conference Call and Webcast. At this time, all participants are in listen-only mode. Later, we will conduct a question and answer session, and instructions will follow at that time. If anyone should require assistance during the conference, please press star and then zero on your touchtone phone.

At this time, all participants are in listen only mode.

Later, we will conduct a question and answer session and instructions will follow at that time, if any once you require assistance during the conference. Please press Star and then zero on your Touchtone phone.

As a reminder, this conference is being recorded I would now like to turn the conference over to your host Mr. Andrew <unk> head of Investor Relations you may begin.

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

Good morning.

Andrew Faughnan: Thank you for joining our conference call to discuss Amicus Therapeutics' School Year 2020 Financial Results and Corporate Highlights. Speaking on today's call, we have John Crowley, Chairman and Chief Executive Officer, Bradley Campbell, President and Chief Operating Officer, Daphne Quimi, Chief Financial Officer, and Dr. Jeff Castelli, Chief Development Officer. Joining for Q&A are Dr. Hung Do, Chief Science Officer, and Dr. Mitchell Goldman, Senior Vice President of Clinical Research.

Thank you for joining our conference call to discuss amicus therapeutics full year 2020 financial results and corporate highlights speaking on today's call. We have John Crowley, Chairman and Chief Executive Officer, probably Campbell, President and Chief Operating Officer, Scott Mcqueen, Chief Financial Officer, and Dr. Jeff Castelli, Chief Development Officer, joining for Q&A, we'll have.

Dr Hung do Chief Science Officer, and Dr. Mitchell Goldman Senior Vice President President of clinical research.

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 the forward-looking statements made in 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.

Referenced on slide two 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 are.

Our forward looking statements should not be regarded as a representation by us at any of our plans will be achieved any or all of the forward looking statements made on this call may turn out to be wrong and can be affected by inaccurate assumptions, we might make or by known or unknown risks and uncertainties. You are cautioned not to place undue reliance on any forward looking statements, which speak on each of the date hereof.

Andrew Faughnan: You are cautioned not to place reliance on any forward-looking statements which speak only to the date 0 of. All forward-looking statements are qualified in their entirety by this cautionary statement. And we undertake no obligation to revise or update this presentation and conference call to reflect events or circumstances after the date hereof. For a full discussion of such forward-looking statements and risks and uncertainties that may impact them, we refer you to the forward-looking statements and risk factors section of our annual report on Form 10-K for the year ended December 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.

All forward looking statements are qualified in their entirety by this cautionary statement on we are.

Undertakes no obligation to revise or update this presentation and conference call true black to.

To reflect events or circumstances. After the date hereof for a full discussion of such forward looking statements and risks and uncertainties that may impact them. We refer you to the forward looking statements and risk factors section of our on our annual report on form 10-K for the year ended December 31, 2020 to be filed later today with the Securities and Exchange Commission.

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

John Crowley: John.

Great. Thank you Andrew and welcome everyone to our full year 2020 results Conference call Yesterday February 28th marked the 14th annual World rare disease day the.

John Crowley: Great, thank you, Andrew, and welcome everyone to our full year 2020 results conference call. Yesterday, February 28th, marked the 14th annual World Rare Disease Day. The goal of this day each year is to raise awareness with policymakers and the public of rare diseases and their impact on the lives of the millions of people and their families living with any one of the more than 7,000 rare diseases. If you're on this call, you care about amicus intermission, and you have an interest in rare diseases.

The goal of this day to years to raise awareness with policymakers and the public of rare diseases and their impact on the lives of the millions of people and their families living with any one of the more than 7000 rare diseases. If you were on this call you care about amicus on our mission and you have an interest in rare diseases.

John Crowley: I'd encourage you to go to the website rarediseaseday.org and learn more, including a moving and inspiring video of six heroes from six continents all living with a rare disease. The impact of these diseases is always so personal. The need for new or newer and better medicines is so great. Yesterday morning, I sat down at my computer to edit these remarks for today. I opened first an email from our team in Europe that let me know of the death earlier yesterday of a brave young woman living with Pompe disease. Her name was Rosella Pacero. She was 21 years old and living in Naples, Italy.

Encourage you to go to the website rare disease day, Dot org and learn more including a moving and creating video of six heroes from six continents, all living with a rare disease.

The impact of these diseases, there's always so personal.

The need for new or newer and better medicines is so great.

Yesterday morning, I sat down on my computer to edit these remarks today.

I opened first an email from our team in Europe that let me know if the deaths earlier yesterday of a brave young woman living with Pompe disease.

Her name was rosella per Sara.

She was 21 years old and living in Naples, Italy.

I first came to nowhere for us rosella when she was two years old in the fall of 2000 and to her.

John Crowley: I first met Rosella when she was two years old in the fall of 2002. Her parents had chained themselves to the front gate of a hospital in Naples, Italy, begging for compassionate use of the then-experimental enzyme replacement therapy that we were developing at Genzyme. I was running the Pompei program at the time, and Henry Tremere, with his compassion and wisdom, allowed us to send the experimental enzyme to Rosella.

Her parents had chained themselves to the front gate or a hospital in Naples, Italy.

Begging for compassionate use of the den experimental enzyme replacement therapy that we were developing at genzyme.

I was running the Pompe <unk> program at the time, and Henry true mirror with us compassion and wisdom.

Allowed us to send the experimental enzyme two rosella.

She was the first Pompeii patient in the world to receive this E. R. T on compassionate use.

John Crowley: She was the first Pompe patient in the world to receive this ERT on compassionate use. And then, in December 2018, while live on Italian television, in front of an audience of millions, a few days before Christmas.

And then in December 2018, while live on Italian television in front of an audience of millions a few days before Christmas.

I had the honor and a privilege to meet rosella in person per the first time.

John Crowley: I had the honor and the privilege of meeting Rosella in person for the first time. It was a moving experience, as you might imagine. We offer our prayers and our thoughts for Rizella and her family. This is the very human side of this business that we have chosen. Rosella's life, her spirit, and, sadly, her past, remind us of the great urgency with which we need new treatment options for everyone living with Pompe disease and all rare diseases, and we need them as soon as possible.

It was a moving experience as you might imagine.

We offer our prayers and our thoughts for resilience and her family.

This is the very human side of this business that we have chosen.

Rosella life for spirit and sadly her passing.

A mind us at the greater urgency with which we need new treatment.

Options for everyone.

The living with Pompe disease, and all rare diseases, and we need them as soon as possible.

So with that very important reminder of our mission, we will turn now to amicus business.

John Crowley: So with that very important reminder of our mission, we'll turn now to Amicus Business. For Amicus, 2020 was a year of excellent growth and execution across all aspects of our business. Science, Clinical, Regulatory, and, as you see, our commercial effort as we continue to build one of the next great global biotechnology companies poised to impact people around the world living with rare diseases. As we did in this morning's press release, I'd like to highlight several key accomplishments.

Parameter as 2020 was a year of excellent growth and execution across all aspects of our business, including science clinical regulatory and as you see our commercial effort as we continue to build one of the next great global biotechnology companies poised to impact people around the world living with rare.

Other diseases.

As we did in this morning's press release I'd like to highlight several key accomplishments.

First yellow fold continues its strong launch performance and remains the cornerstone of our success with.

John Crowley: First, Galliford continues its strong launch performance and remains the cornerstone of our success. With $261 million in 2020 revenue, Gallup Old Launch continues to exceed expectations and comes in above our guidance range that was set out last year.

With $261 million in 2020 revenue the Gallup polled launch continues to exceed expectations and coming in above our guidance range that we set out last year.

The full year revenue represents performance across the global business.

John Crowley: The full-year revenue represents performance across the global business, including new patient starts from both switch and treatment naive patients throughout the year. Second, our key regulatory and R&D timelines remain on track. Following results from the Phase III Propel Study of ATGAA in late-onset Pompe disease, we plan to complete the rolling BLA submission in the second quarter of this year and anticipate additional regulatory submissions in the European Union and in other geographies throughout 2021.

<unk>, new patient starts from both switch and treatment naive patients throughout the year.

Second our key regulatory and R&D timelines remain on track following results from their phase III propel study of a T T. A E in late onset pompe disease.

We plan to complete the rolling BLA submission in the second quarter of this year and anticipate additional regulatory submissions in the European Union and in other geographies throughout 2021.

We continue to have great confidence in a T. G. A a day benefit people living with Pompe disease.

John Crowley: We continue to have great confidence in ATGAA to benefit people living with Pompe disease, and we intend to continue to move it rapidly toward its regulatory submissions for approval. In our gene therapy pipeline, we also continue to move forward our lead batten disease programs for CLN6 and CLN3, as well as our most advanced preclinical gene therapy programs. Announced for the first time this morning, our CLN6 program has been granted fast-track designation by the USFDA.

And we intend to continue to move it rapidly toward its regulatory submissions for approval.

Within our gene therapy pipeline. We also continue to move forward, our lead batten disease programs for <unk>, six and <unk> three as.

As well as our most advanced preclinical gene therapy programs.

<unk> for the first time this morning, our C. L. N. Six program has been granted fast track designation by the U S. F D. A.

John Crowley: This follows the EMA granting prime designation to this CLN program in late 2020. Through our broad and important research collaboration with Dr. Jim Wilson and the University of Pennsylvania, we are highly encouraged by the preclinical data and progress from our Fabry disease to the therapy clinical candidates. Given the data we have seen to date, this is the second program to highlight the capabilities and potential this collaboration can bring to people living with rare diseases. And third, the Amicus cash position is sufficient to achieve self-sustainability without the need for any future dilutive financing, which will continue our continued revenue growth.

This follows the EMA granting the prime designation to this program in late 2020.

Through our broad and important research collaboration with Dr. Jim Wilson and the University of Pennsylvania, We are highly encouraged by the preclinical data and progress from our Fabry disease gene therapy clinical candidate.

Given the data we have seen to date. This is the second program to highlight the capabilities and potential this collaboration can bring to people living with rare diseases.

And third the amicus cash position is sufficient to achieve self sustainability without the need for any future financings.

Our continued revenue growth.

Prudent expense management and growth potential has allowed us to reach this important milestone.

John Crowley: Prudent expense management and growth potential have allowed us to reach this important milestone as we continue to realize our vision of delivering groundbreaking and potentially curative new medicines for people living with rare diseases around the world. Next, on slide five, we'll set our key strategic priorities for this year. We are well on track to achieve our five key strategic priorities for 2021, including Galliford, our precision medicine for fever. We will continue to drive Gallifold to more people living with febrile disease with amenable variants in existing and new markets; we look to achieve global product revenue of 300 million to $315 million this year, which reflects the strong momentum and demand behind this precision medicine globally, despite some COVID-related disruptions that we saw in the latter part of 2020 and which continue into early this year.

Because we continue.

To realize our vision of delivering groundbreaking and potentially curative new medicines for people living with rare diseases around the world.

Next on slide five we will set our again, our key strategic priorities for this year.

We are well on track to achieve our five key strategic priorities for 2021, including.

Yellow fold, our precision medicine for fabry.

We will continue to drive <unk> to more people living with fabry disease with amenable variance in existing and new markets. We look to achieve global product revenue of 300 million to $315 million. This year, which reflects the strong momentum and demand behind this precision medicine globally.

Despite some COVID-19 related disruptions that we saw in the latter part of 2020 and which continue into early this year.

Second we remained steadfast and passionate in our commitment to advancing a T. G. A a global regulatory submissions as quickly as possible for the benefit of all people living with Pompe disease.

John Crowley: Second, we remain steadfast and passionate in our commitment to advancing ATGAA to global regulatory submissions as quickly as possible for the benefit of all people living with Pompe disease. Third, we are advancing our industry-leading rare disease gene therapy portfolio. Stemming from our Global Research and Gene Therapy Center of Excellence in Philadelphia, we will be advancing the clinical development, manufacturing, and regulatory discussions across multiple programs in our gene therapy pipeline. Fourth, in addition, we are progressing our manufacturing capabilities and capacity to build world-class technical operations to support all gene therapy programs.

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

Stemming from our global research and gene therapy Center of excellence in Philadelphia.

We will be advancing the clinical development manufacturing and regulatory discussions across multiple programs and our gene therapy pipeline.

Fourth in addition, we are progressing our manufacturing capabilities and capacity to build world class technical operations to support all gene therapy programs.

And five 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. So with that introduction, let me now here on the call over to Bradley Campbell, our President and Chief operating officer to further highlight the Gallophone performance spread.

John Crowley: And five, 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. So with that introduction, let me now hand the call over to Bradley Campbell, our President and Chief Operating Officer, to further highlight Galliford's performance. Brad.

Great. Thanks, John Good morning, everyone.

Bradley L. Campbell: Great, thanks, John. Good morning, everyone.

As John mentioned I will now walk us through in more detail our Gulf old performance for the year.

Bradley L. Campbell: As John mentioned, I'll now walk us through in more detail our Gallifold performance for the year. On slide 7, we give our global snapshot of Gallifold commercial progress. And for the full year again, total product revenue was $260.9 million, driven by strong patient demand, favorable reimbursement dynamics, and business continuity. The geographic breakdown of revenue during the full year was $180.8 million, or 69% of revenue generated outside of the United States, and the remaining $80.1 million, or 31%, coming from within the United States.

On slide seven we give our global snapshot of the Gulf old commercial progress and for the full year again total product revenue was $269 million driven by strong patient demand favorable reimbursement dynamics and business continuity.

The geographic breakdown of revenue during the full year was $180 8 million or 69% of revenue generated outside of the United States and the remaining $80 1 million or 31% coming from within the United States and we would expect that split to continue to be roughly 70 30, as we continue to grow both parts of the business.

Bradley L. Campbell: And we would expect that split to continue to be roughly 70-30 as we continue to grow both parts of the business. Turning now to slide eight, Q4 was another strong quarter. The business continues to be incredibly resilient with patience added in all of our major. As we previously highlighted, with the resurgence of COVID in the fourth quarter, we did see some slowdown in new patient starts, which led to lag time and gallop treatment initiation, and some of that continues into the new year.

Turning now to slide eight Q4 was another strong quarter. The business continues to be incredibly resilient with patients added in all of our major markets. As we previously highlighted with the resurgence of Covid in the fourth quarter. We did see some slowdown in new patient starts which led to a lag time in <unk> treatment initiation and some of that continues into the new year.

Importantly, though our supply chain remains fully intact, our customers have confidence they can access gala hold and our field team has been able to achieve a substantial majority of their pre COVID-19 touch points through a combination of in person digital telephonic and other means of interacting with their physicians and most importantly, we continue to add patients in our key geographies coming into the new year.

Bradley L. Campbell: Importantly, though, our supply chain remains fully intact, our customers have confidence they can access Gallifold, and our field team has been able to achieve a substantial majority of their pre-COVID touchpoints through a combination of in-person, digital, telephonic, and other means of interacting with their physicians. And, most importantly, we continue to add patients in our key geographies coming into the new year. Of course, we'll continue to monitor the pandemic's impact and duration, but the good news is today we are confident we will deliver on our guidance and see continued growth this year and going forward, and we expect the COVID impact to recede throughout the year. From a numbers perspective, you can see that 2020 sales increased 43% from 2019, which includes a 1% benefit from foreign exchange. From a true operational performance perspective, sales increased by 42% compared to last year.

Of course, we'll continue to monitor the pandemic impact on duration, but the good news is today. We are confident we will deliver on our guidance and see continued growth this year and going forward and we expect the COVID-19 impact to recede throughout the year.

From a numbers perspective, you can see the 2020 sales increased 43% from 2019.

Which includes a 1% benefit from foreign exchange from a true operational performance perspective sales increased by 42% compared to last year on.

On the left hand side, we show our quarterly performance over the past several quarters and as we've mentioned in past calls while we continue to expect strong growth versus same quarter last year due to a variety of factors the rate of growth from quarter to quarter as typically non linear.

Bradley L. Campbell: On the left-hand side, we show our quarterly performance over the past several quarters. And as we've mentioned in past calls, while we continue to expect strong growth for the same quarters last year, due to a variety of factors, the rate of growth from quarter to quarter is typically nonlinear. On slide nine, we've called out several of the drivers and metrics which will lay the foundation for continued growth in 2021. It starts with the momentum from 2020, where we finished with over 1,400 patients on Galliford and opened up several new launches.

On slide nine we've called out several of the drivers and metrics, which will lay the foundation for continued growth in 2021.

It starts with the momentum from 2020, where we finished with over 1400 patients on <unk> and opened up several new launch countries.

These over 1400 patients on <unk> now represent about a 49% global market share of treated amenable patients.

Bradley L. Campbell: These over 1,400 patients on Galifold now represent about a 49% global market share of treated, amenable patients. So while we're achieving higher market shares in countries where we've been approved the longest, there's still plenty of opportunity to continue to switch patients over to Gallup. We also know that there are significant numbers of diagnosed untreated patients who have amenable mutations.

So while we are achieving higher market shares in countries, where we have been approved the longest theres still plenty of opportunity to continue to switch patients over to Gulf World.

We also know that there are significant numbers of diagnosed untreated patients who have amenable mutations and you see now that we have a global mix of about 60% switch and 40% of patients who are naive to treatment.

Bradley L. Campbell: And you see now that we have a global mix of about 60% switch and 40% of patients who are naive to treatment. As we've said previously, over the next few years, we expect to see that rate of switch in naive patients to be about 50-50, and in the long term, we expect to see that percentage reverse in favor of naive patient growth. All of that is underpinned by compliance and adherence rates that continue to exceed 90%, reiterating our belief that those patients who go on Gallifold stay on Galliford.

As we've said previously over the next few years, we'd expect to see that rate of switch and naive patients to be about 50 50 and in the long term, we expect to see that percentage reverse in favor of naive patient growth.

All of that is underpinned by compliance and adherence rates that continue to exceed 90% reiterating our belief that those patients who go on <unk> stay on California.

The value of <unk> has also been well recognized by payers with neither 100 percentage of insurance reauthorization granted in 2021 with the U S payers and a perfect track record of successful negotiation and renegotiation of reimbursement outside the United States. So our relentless focus on ensuring access to <unk> continues.

Bradley L. Campbell: The value of Galliford has also been well recognized by payers, with nearly 100% of insurance reauthorizations granted in 2021 with U.S. payers and a perfect track record of successful negotiation and renegotiation of reimbursement outside the United States. Thus, our relentless focus on ensuring access to Galliford continues. Turning now to slide 10, another important driver of growth for GALIFOLD is the continued geographic expansion. As a reminder, GALIFOLD has received regulatory approval in over 40 countries around the world and has commercial sales in more than 30 of those today. In 2020, we added multiple new markets, including Poland, Iceland, Luxembourg, Argentina, and Greece, to the list of countries with reimbursement for Gallup Hold.

Turning now to slide 10, another important driver of growth from Gallup holders. The continued geographic expansion as a reminder, galliford has received regulatory approval in over 40 countries around the globe and commercial sales and more than 30 of those today.

In 2020, we added multiple new markets, including Poland, Iceland, Luxemburg, Argentina in Greece to the list of countries with reimbursement from <unk>.

In 2021, we expect to add at least five additional countries to that list as we look to continue to expand access to those fabry patients with amenable variance around the globe.

Bradley L. Campbell: In 2021, we expect to add at least five additional countries to that list as we look to continue to expand access to those Fabry patients with minimal variance around the world. Moving ahead to slide 11, I'll highlight our guidance for the... Despite the recent COVID-related headwinds in certain geographies that we've already mentioned, demand for Gallifold worldwide has never been stronger, now with queues of potential new Gallifold patients building in multiple geographies.

Okay.

Moving ahead to slide 11, I'll highlight our guidance for the year despite the.

A recent COVID-19 related headwinds in certain geographies that we've already mentioned demand for Gallup hold worldwide has never been stronger now with Qs of potential new Gallup old patients building in multiple geographies. We're confident in our guidance of 300 to 350 $15 million in full year global sales, which continues our strong track record of.

Bradley L. Campbell: We're confident in our guidance of $300 to $315 million in full-year global sales, which continues our strong track record of double-digit growth year on year. As part of that guidance, we project net new patient starts this year will be even greater than in 2020. And we expect this growth in patients and corresponding revenue to be weighted to the second half of the year as the COVID impact continues to ease. Given this dynamic, as well as the nonlinear quarter-to-quarter growth and ordering patterns that we've seen in previous years, we expect sequential growth from Q4 to Q1 to be relatively fast.

Double digit growth year on year as.

As part of that guidance, we project net new patient starts this year will be even greater than in 2020.

And we expect this growth in patients and corresponding revenue to be weighted to the second half of the year as the Covid impact continues to EPS.

Given this dynamic as well as the non linear quarter to quarter growth and ordering patterns that we've seen in previous years, we expect sequential growth from Q4 to Q1 to be relatively flat.

Now on slide 12, with several years of performance behind US. We can confidently say, we are on that path to $500 million sales opportunity in 2023, we expect to.

Bradley L. Campbell: Now on slide 12, with several years of performance behind us, we can confidently say we are on that path to $500 million in sales opportunities in 2023. We expect to generate $1 billion in cumulative revenue over the next three years, which goes a long way towards funding our R&D and OpEx over that period. We also have even further conference, in the $1 billion revenue opportunity at peak as we continue to see significant growth in the Fabrae market globally, driven by continued diagnosis from high-risk screening, newborn screening, and other diagnostic initiatives, which we're investing in as well. In fact, if you didn't get a chance, I'd recommend that you do.

To generate $1 billion in cumulative revenue over the next three years, which goes a long way towards funding, our R&D and opex over that period.

We also have even further confidence.

In the 1 billion dollar revenue opportunity at peak as we continue to see significant growth from the fabry market globally.

Driven by continued diagnosis from high risk screening newborn screening and other diagnostic initiatives, which we're investing in as well.

In fact that you didn't get a chance I'd recommend that.

See the posters and presentations at the World Lvn meeting in February that highlight two great New initiative that we sponsored.

Bradley L. Campbell: See the posters and presentations at the World LVN meeting in February that highlight two great new initiatives that we've sponsored: screening for Fabry and idiopathic pain populations in Germany, as well as some exciting new work using AI, artificial intelligence, to improve diagnosis of Fabry here in the United States. Finally, we have orphan exclusivity in the U.S. and in Europe, in addition to our 24 Orange Book listed patents that give us IP coverage into the late 2030s, 11 of which provide protection through 2038.

Screening for Fabry in idiopathic pain populations in Germany, as well as some exciting new work using AI artificial intelligence to improve diagnosis of fabry here in the United States.

Finally, we have orphan exclusivity in the U S and in Europe. In addition to our 24 Orange book listed patents that give us IP coverage into the late 2011 of which provide protection through 2038.

Bradley L. Campbell: So continued opportunity to provide access to Galliford globally for a long period. And with that, let me turn the call now over to Dr. Jeff Castelli, our Chief Development Officer, who will highlight our APGAA program and our gene therapy pipeline.

Continued opportunity to provide access to <unk> globally for a long period to come.

And with that let me turn the call now over to Dr. Jeff Castelli, Our Chief Development Officer, who will highlight our atg AA program and our gene therapy pipeline.

Jeff.

Thank you Brad and good morning, everyone.

Jeffrey P. Castelli: Thank you, Brad, and good morning, everyone. Moving on to our R&D updates on slide 14, we'll start with ATJA, our novel next-generation therapy for Ponte disease. Before we provide a recap of the recent Phase 3 Propel data, and building on John's earlier comments, it's important to recognize the significant unmet need that remains today in Pompeii. In addition to the individual human tragedies, we've seen multiple publications in natural history studies of Pompe patients that all highlight the initial benefit of treatment that is followed by continued long-term decline on key measures of disease.

Moving on to our R&D updates on slide 14, we will start with a T. J a R novel next generation therapy for Pompe disease.

Before I provide a recap of the recent phase III propel data and building on John's earlier comments.

It's important to recognize the significant unmet need that remains today on pump line.

In addition to the individual human tragedies, we've seen multiple publications and natural history studies of Pompe patients that all highlight the initial benefit of treatment that is followed by continued long term decline on key measures of disease.

Jeffrey P. Castelli: Highlighted here on slide 14 is a very recent study of 68 adult patients from Spain, Taiwan, Italy, and Germany with Pompe disease who were on the approved enzyme replacement therapy for at least three years. This real-world study shows an initial benefit from the ERT in the first few years with a secondary sustained decline in multiple outcome measures, specifically highlighted here, our six minute walk in FEC. And you can see that the weakest efficacy is actually for lung capacity, consecutively driving the need for additional ventilatory support over time. The authors go on to note that respiratory insufficiency is the most frequent cause of death in Pompeii, as well as recognize the need for new treatment options for these patients.

<unk> here on Slide 14 is a very recent study.

68, adult patients from Spain, Taiwan, Italy, and Germany, with Pompe disease, who were on the approved enzyme replacement therapy for at least three years.

This real World study shows an initial benefit from <unk> in the first few years with a secondary sustained decline in multiple outcome measures specifically hydraulic here are six minute walk on at D. C.

And you can see that the weakest efficacy is actually for the lung capacity.

And consecutively driving the need for additional ventilatory support over time.

<unk> go on to note that respiratory insufficiency is the most frequent cause on death in pompe as well as recognizing the need for new treatment options for these patients.

Before we go into the data a key point to remember is that the Iraqi experienced population described here on slide 14 has only ever been studied in a controlled setting in our phase III propel us clinical trial all other controlled late onset Pompe disease studies had been on participants that are naive to <unk> treatment.

Jeffrey P. Castelli: Before we go into the data, a key point to remember is that the ERT-experienced population described here on slide 14 has only ever been studied in a controlled setting in our Phase 3 PROPEL clinical trial. All other controlled late-onset Pompe disease studies have been in participants that are naive to ERT treatment. But that is just background.

Yes.

With that as background.

We will move to slide 15 true.

Jeffrey P. Castelli: We will move to slide 15, to review some of the key data from the COOPEL study. As a reminder, this was a double-blind, randomized study assessing the efficacy and safety of ATJA in adult treatment naive and ERT switch participants against the currently approved therapy. We enrolled 123 patients at 62 sites in 24 countries. 117 patients completed the study, and all 117 have voluntarily enrolled in the extension study and continue to receive ATGA as their only disease-modifying treatment.

To review some of the key data from the propel study.

As a reminder, this was a double blind randomized study assessing the efficacy and safety of a T. J, a an adult treatment nave and <unk> switch participants against the currently approved therapy.

We enrolled 123 patients at 62 sites in 24 countries.

117 patients completed the study and all 117 have voluntary enrolled in the extension study and continue to receive AT&T, a us are only disease modifying treatment.

Jeffrey P. Castelli: On the primary endpoint of six-minute walk distance, patients on ATGA outperformed those on aglucosidase alpha in the overall population of ERT-experienced and ERT-naive patients, with a difference between groups of 14 meters. However, this difference did not reach statistical significance or superiority.

On the primary endpoint of six minute walk us things patients on a T. J outperformed those on <unk> Alfa and the overall population of <unk> experienced an ear T naive patients with a difference between groups of 14 meters.

This difference did not reach statistical significance for superiority.

However on a first key secondary end point on <unk>.

Jeffrey P. Castelli: However, on the first key secondary endpoint, a percent predicted for hospital capacity, or FBC, in the overall population, ATGA showed a nominally statistically significant and clinically meaningful difference for superiority versus the approved standard of care of glucosidase alpha, with a difference of 3% and a P value of 0.023. This is a really impactful finding, as this was the main endpoint upon which aglucosidase alpha was approved, and as mentioned earlier, progressive loss of pulmonary function is the leading cause of mortality in Pompeii. Moving ahead on slide six.

<unk> predicted forced vital capacity or F. D C and the overall population atg a showed a nominally statistically significant and clinically meaningful difference for superiority versus approved standard of care <unk> Alfa for the difference of 3% and a P value of 0.0 to three.

This is a really impactful finding us. This was the main end point upon which <unk> also was approved and as mentioned earlier progressive loss of pulmonary function is the leading cause of mortality in pompe.

Moving ahead on slide 16, the day to hear captures the 95 <unk> experienced patients those have been on therapy for an average of seven years and most similar to the patient as described in the publication I mentioned earlier.

Jeffrey P. Castelli: The data here captures 95 ERT-experienced patients, those who have been on therapy for an average of seven years and are most similar to the patients described in the publication I mentioned earlier. We saw that ERT-experienced patients switching from aglokosidase alpha to ATGA walked approximately 17 meters farther than those remaining on aglokosidase alpha, with a P equal to.046. In these patients switching from aglokosidase alpha to ATGA, we also saw an improvement in percent predicted FDC compared to a decline in patients remaining on aglokosidase alpha, with a difference of 4.1% and a P value of 0.001.

We saw that EBIT experienced patients switching from other <unk> alpha to a T. J walked approximately 17 meters farther than those remaining on Agua <unk> Alpha with a P equal to 0.046 in these patients switching from other properties offer to a T. J. We also saw an improvement in percent predicted SBC compared to.

The decline in patients remaining on Agua <unk> Alpha with a difference of four 1% and a P value of 0.006.

Slide 17 presents a summary of the top line efficacy and biomarker data grouping.

Jeffrey P. Castelli: Slide 17 presents a summary of the top-line efficacy and biomarker data, grouping the data into domains of motor function, muscle strength, pulmonary function, patient-reported outcomes, and biomarkers. And here you can see that the totality of data across the primary and key secondary ends favor ATJA over Agricosidase Alpha in both

Grouping the data into domains of motor function muscle strength pulmonary function patient reported outcomes and Biomarkers and here you can see that they'd show quality of data across the primary and key secondary endpoints favor a T J, a <unk> alpha and both the overall and ear team experienced populations.

Jeffrey P. Castelli: overall.

Jeffrey P. Castelli: Law, and ERT experience population. We'll be on the Friday.

Moving on to slide 18.

Jeffrey P. Castelli: We first want to remind everyone that the PROPEL study tested for superiority versus an approved therapy, not placebo, and that the bar for approval of the second product generally requires demonstration only of non-inferiority to approved therapy. As shown earlier, ATGA demonstrated nominal superiority on the first key secondary endpoint of FEC over approved therapy. On slide 18, we show here post hoc non-inferiority analyses for the primary endpoints of the six-minute walk and the first key secondary endpoint of FDC just as a reference.

First one to remind everyone that the propel study tested for superiority versus an approved therapy not placebo net the bar for approval on the second product generally requires demonstration only of non inferiority to approved therapy.

As shown earlier atg, a demonstrated nominal superiority on the first key secondary endpoint that B C over approved therapy.

On slide 18, we show here post hoc non inferiority analysis for the primary endpoint six minute walk and the first key secondary end point on that these see just us reference.

As you can see these non inferiority analyses are highly significant.

Jeffrey P. Castelli: As you can see, these non-inferior analyses are highly significant. The non-inferiority test for FEC was pre-specified in the FAP, but it was only to be conducted if superiority was missed on both 6-minute walk and FEC. Thus, this analysis did not need to be performed as we hit on nominal superiority, which by default also indicates non-inferiority.

The non <unk> tests per SEC was prespecified in the us, but with only to be conducted if superiority was missed on both six minute walk and at D. C. Thus this analysis did not need to be performed as we hit on nominal superiority.

Which by default also indicates non inferiority.

And non inferiority analysis for six minute walk was not pre specified in the S. E T. Because <unk> alpha historically did not demonstrate a significant improvement on the endpoint of six minute walk.

Jeffrey P. Castelli: A non-inferiority analysis for the six minute walk.

Jeffrey P. Castelli: and S.A.P. because aglucosidase-alpha historically did not demonstrate a significant improvement on the endpoint of the 6-minute walk, which impacted the interpretability of a non-inferiority analysis. However, as shown here, post-hoc demonstration of non-embryonic six-minute walk is clearly apparent across a range of clinically reasonable margins. Based on the totality of the data, moving to slide 19, we see ATJ having a leading position in the treatment landscape for Pompe disease. Here we have our next step surrounding the development, regulatory,

Impacted interpret ability of a non inferiority analysis, however, as shown here.

Hock demonstration of non inferiority in six minute walk us clearly apparent across a range of clinically reasonable margins.

Based on the totality of the data moving to slide 19, we see a T J, having a leading position in the treatment landscape for Pompe disease.

Here, we have our next step surrounding the development regulatory and manufacturing strategy for the <unk> program first a rolling BLA submission, which was initiated with the FDA in the fourth quarter of 2020 remains on track with the submission of the final module to be completed in the second quarter of 2021.

Jeffrey P. Castelli: First, our rolling DLA submission, which was initiated with the FDA in the fourth quarter of 2020, remains on track with the submission of the final module to be completed in the second quarter of 2021. Additionally, our MAA submission with the EU is expected to be completed in the second half of this year, and we expect additional global filings to follow.

Additionally, our MAA submission with the EU is expected to be completed in the second half of this year and we expect additional global filings to follow.

We will look to expand our ongoing open label adolescent study in children, aged 12 to 17 and Additionally, our clinical study for Pompe patients with infantile onset disease is expected to begin this year.

Jeffrey P. Castelli: We will look to expand our ongoing open-label adolescent study in children age 12 to 17. And additionally, our clinical study for Pompe patients with infantile-onset disease is expected to begin this year. And finally, in response to the many requests for compassionate use, otherwise known as Expanded Access, that we receive for children with Infantile Onset Pompe Disease. Our Expanded Access Programs for Pompe Infantile Patients and Adult Onset Patients are open, and have enrolled multiple patients. Further expanded access for all Pompe patients is being considered.

Finally in response to the many requests for compassionate use.

While none of US expanded access that we've received for children with infantile onset pompe disease or expanded access programs from Pompeii infantile patients and adult onset patients are open and have enrolled multiple patients.

Further expanded access for all Pompeii patients is being considered.

Yes.

Moving on now to slide 21, I will briefly highlight our industry, leading portfolio of gene therapies for rare diseases.

Jeffrey P. Castelli: Moving on now to slide 21, I will briefly highlight our industry-leading portfolio of gene therapies for rare diseases. Several Amicus presentations were given at the World Symposium Conference in February covering our CLN6, CLN3, and Fabry gene therapy programs. Our FabRAID program continues to advance ahead of schedule with an Amicus transgene engineered for stability combined with the University of Penn gene transfer technology, and the data presented at World further validates this innovative and synergistic approach.

Several amicus presentations were given at the World Symposium conference in February covering our ceiling six on three in Fabry gene therapy programs.

Our Fabry program continues to advance ahead of schedule with an amicus transgene engineered for stability combined with our University of Penn Gene transfer technology and the data presented at World further validates the innovative and synergistic approach, we continue to make progress across our preclinical gene therapy programs with Penn and expect.

Jeffrey P. Castelli: We continue to make progress across our preclinical gene therapy programs at Penn and expect further updates and data this year. As a reminder, as part of this collaboration, Amicus has rights to 50 plus diseases, including seven currently in active preclinical programs. On slide 22, and previously presented at World, just in February, we captured the initial results from the first in-human study of our CLN3 bat disease gene therapy.

Updates and data this year.

As a reminder, as part of this collaboration and it gets has rights to 50 plus diseases, including seven currently in active preclinical programs.

On slide 22, and previously presented at World just in February.

Capture the initial results from the first in human study of our C. O N three batten disease gene therapy.

Jeffrey P. Castelli: TLN3 Batten disease is one of the most common neurodegenerative disorders affecting children, which leads to blindness, motor impairment, learning difficulties, epilepsy, and ultimately premature death. The primary outcome measures are determined using the Physical Impairment Subscale of the Unified Batten Disease Rating Scale, also called UBDRS, which is a clinical rating instrument developed specifically to assess disease progression in children with CLN3. And it includes evaluations of growth and fine motor skills, vision, and speech.

Still on <unk> Batten disease is one of the most common neurodegenerative disorders effective Inc.

In children, which leads to blindness motor impairment learning difficulties epilepsy, and ultimately premature death.

Primary outcome measures are determined using the physical impairment subscale of the unified Batten disease rating scale also called <unk>, which is a clinical rating instrument developed specifically to assess disease progression in children with sale on three.

And it includes the valuations of gross and fine motor skills vision and speech.

Higher scores indicate greater physical impairment.

Jeffrey P. Castelli: Higher scores indicate greater physical impairment. As seen on the right hand of the slide, for the three subjects treated in the low dose cohort, the mean yearly rate of change in UBDRS physical impairment score was stable, with only an increase of 0.07 over those 12 months. This is a favorable measure versus the almost three-point increase expected in untreated patients based on published natural history of over 82 children that were followed. On safety, the treatment was generally well tolerated, with the majority of adverse events being mild or moderate and unrelated to treatment. There were no patterns of AEs related to AAD or CLN3 immunogenicity that were observed.

As seen on the right hand of the slide for the three subjects treated in the low dose cohort. The mean yearly rate of change and you'd be duress physical impairment score was stable with only an increase of 0.07 over those 12 months a favorable measure vs. He almost three point increase expected in untreated patients based on.

On published natural history of over 82 children that were followed.

On safety the treatment was generally well tolerated with the majority of adverse events being mild or moderate and unrelated to treatment. There were no patterns of aes related to AAD or C. O on three immunogenicity that were observed.

For this program, we look to advanced manufacturing and regulatory discussions and plan to submit the protocol for the next clinical study to the IND by the end of the year.

Daphne E. Quimi: For this program, we look to advance the manufacturing and regulatory discussions and plan to submit the protocol for the next clinical study to the IND by the end of the year. With that, I would like to now turn the call over to Daphne Quimi to review our financial results, guidance, and outlook.

With that I would like to now turn the call over to Daphne <unk> Clini to review, our financial results guidance and outlook Daphne.

Thank you Jack and good morning, everyone. Our financial overview begins on slide 23, with our income statement for the full year ending December 31 2020.

Daphne E. Quimi: Thank you, Jeff, and good morning, everyone. Our financial overview begins on slide 23, with our income statement for the full year ending December 31, 2020. For the full year, we achieved gallifold revenue of $260.9 million, which is a 43% increase over 2019. This includes a year-over-year operational revenue growth measured at constant currency exchange rates of 42%, further benefited by a positive currency impact of 1%. Cost of goods sold as a percentage of net sales was 11.9% in the year as compared to 12.1% for the prior year period. The decrease in cost of goods sold as a percent of revenue was due to the proportion of sales in countries subject to a higher royalty burden.

For the full year, we achieved counseled revenue $260 9 million, which is a 43% increase over 2019.

This includes a year over year operational revenue growth measured at constant currency exchange rates of 42% further benefited by a positive currency impact of 1%.

Cost of goods sold as a percentage of net sales was 11, 9% in here as compared to $12 one per cent for the prior year period.

Cost of goods sold as a percentage of revenue decrease was due to the proportion of sales in countries subject to higher royalty burden.

Daphne E. Quimi: Total operating expenses were $476.8 million in 2020, an increase as compared to $464.3 million in 2019. On a non-GAAP basis, total operating expenses were $415.7 million in 2020 as compared to $411.8 million in 2019. The increase in research and development costs reflected our continued investment to support the gene therapy program pipeline and the PROPEL study, as well as realignment with our strategic priorities. Our investment in research and development includes the impact of the implementation of cost-reduction measures, as did the decrease in selling general and administrative expenses. We define non-GAAP operating expense as research and development and SG&A expenses excluding share-based compensation expense, changes in the fair value of contingent consideration, and depreciation.

Total operating expenses were $476 8 million in 2020 increased as compared to $464 3 million in 2019.

On a non-GAAP basis total operating expenses were $415 7 million in 2020 as compared to $411 8 million in 2019.

The increase in research and development costs reflected our continued investment to support the gene therapy program pipeline and the propel study as well as re alignment with our strategic priorities.

Our investment in research and development includes the impact of implementation of cost reduction measures as does the decrease in selling and selling general and administrative expenses.

We define non-GAAP operating expense as research and development and SG&A expenses, excluding share based compensation expense changes in fair value of contingent consideration and depreciation.

Net loss for the full year, 2020, with $276 8 million or $8.07 per share as compared to the net loss of $356 4 million or $1.48 per share from the prior year period.

Daphne E. Quimi: The net loss for the full year 2020 was $276.8 million, or $1.07 per share, as compared to the net loss of $356.4 million, or $1.48 per share, for the prior year period. As of December 31, 2020, we had approximately 262 million shares outstanding. Turning now to slide 24, following our $400 million Senior Secure Term Loan facility in the third quarter, we are on a path to self-sustainability without the need for any future diluted financing.

As of December 31, 2020, we had approximately 262 million shares outstanding.

Turning now to slide 24, following our $400 million senior secured term loan facility in the third quarter we on.

We're on a path to self sustainability without the need for any future can win financing. We have achieved this milestone by our continued revenue growth with council as well, we're driving efficiencies cost savings and careful expense management.

Daphne E. Quimi: We have achieved this milestone through our continued revenue growth with Gallup Hold, as well as driving efficiencies, cost savings, and careful expense management. Securing this financing with market-setting terms gives us a strong financial platform to advance both patient and amicus shareholder interest. For the third straight year, we expect total non-GAAP operating expenses in 2021 to remain relatively flat from 2020 as we leverage the global commercial infrastructure that is already in place for the ATGAA launch and other products in our pipeline, and as we transition the costs associated with the development of ATGAA to multiple gene therapy programs in our pipeline, and we maintain financial discipline while meeting our objectives.

Securing this financing with markets net income gives us a strong financial platform to advance both patient and amicus shareholder interests.

But the third straight year, we expect total non-GAAP operating expenses from 2021, which remained relatively flat from 2020 as we leverage the global commercial infrastructure that is already in place for the M. T. G I ate lunch and other products from our pipeline.

And as we transition costs associated with the development of a T. J a true multiple gene therapy programs on our pipeline and we maintain financial discipline and while meeting our objectives.

Daphne E. Quimi: To reiterate, all high-priority research programs in the gene therapy portfolio are moving ahead on schedule, and we continue to fully support the work with the Wilson Lab at Penn. A few comments about our cash position and 2021 financial guidance. Cash equivalents and marketable securities were $483.3 million at December 31, 2020, compared to $452.7 million at December 31, 2019. We are issuing our full-year Gallup Revenue Guidance of $300 million to $315 million in addition to 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.

To reiterate all high priority research program in the gene therapy from it.

Portfolio moving ahead on schedule and we continue to fully support the work with the Wilson lab at Penn.

A few comments about our cash position in 'twenty and 'twenty, one financial guidance cash.

Cash cash equivalents in marketable securities for $483 3 million at December 31, 2020, compared to $452 7 million at December 31 2019.

We are issuing a full year galiform revenue guidance of 300 million to 300 million. In addition to our non-GAAP operating expense guidance of 410 million per $420 million and with that let me turn the call back to John for closing remarks.

Great. Thank you, Daphne and Jeff and Bradley as well so as you can see we have been relentlessly focused on performance across the business. Despite the unprecedented change and challenges brought about by the global pandemic.

John Crowley: Great, thank you Daphne and Jeff and Bradley as well. So, as you can see, we've been relentlessly focused on performance across the business, despite the unprecedented change and challenges brought about by the global pandemic. We have a great global team of passionate entrepreneurs at Amicus who have led and will continue to lead us through this. And I am confident that as the world emerges from the COVID crisis, Amicus will emerge even stronger. Operator, with that, we're happy to take questions.

We have a great global team of passionate entrepreneur us at amicus, who have led and will continue to lead us through this and I am confident that as the world emerges from the Covid crisis amicus will emerge even stronger on prior to with that we're happy to take questions.

Ladies and gentlemen, if you have a question please press star.

Operator: Ladies and gentlemen, if you have a question, please press star and then the number one on your touch-tone phone. At this time, we ask that you only ask one question. If you have additional questions, please enter back into the queue. If your question has been answered, you may remove yourself from the queue by pressing the pound key. Thank you. Your first question comes from the line of Tazeen Ahmad, with Bank of America. Hi guys, good morning. Thanks for taking the time. Hi Tazeen, morning.

And then the number one on your Touchtone phone at this time, we ask that you only ask one question.

If you have additional questions. Please enter back into the queue.

If your question has been answered you may remove yourself from the queue by pressing the pound T. Thank you yes.

First question comes from the line of <unk> Ahmad.

With bank of America.

Hi, guys. Good morning, Thanks for taking in light of the morning.

John.

Just.

John Crowley: Hi John. Hi.

Maybe can you frame for us from us.

John Crowley: Just, maybe can you frame for us some expectations in terms of upcoming catalysts as it relates to Pompeii and the competitive landscape? So the Santa Fe has a May 18th PDUFA coming up for NEO.

In terms of upcoming catalysts on the relate to.

Tom pay on that and the competitive landscape.

Tennessee has a may 18 coming up from Meow.

And.

Can you just walk us through how we should think about the competitive landscape.

John Crowley: Can you just walk us through how we should think about the competitive landscape, depending on the type of label that they get? Is it your expectation that they will get a full label, meaning inclusive of switch patients? And what would that mean if they're first to market? And then the second scenario is, if they just end up with a label for treatment-naive patients, how do you think that portends for your application that's gonna be completed around the same time? Thank you.

Depending on the type of Labor I think GAAP is it is it your expectation that they will get US a label, meaning inclusive of switch patients.

And what would that mean, a fair price to market and then the second scenario and if they just end up with a label for treatment naive patients.

Do you think that portends for your application.

Gonna be completed around the same time. Thank you.

Sure I really can't comment much on on the neo product or its regulatory pathways I will say of course. It was studied in a controlled fashion only in treatment naive patients and I think that's a very significant distinction between what we studied in a majority ERP switch population so for us we.

John Crowley: Sure, I really can't comment much on the NEO product or its regulatory pathways. I will say, of course, it was studied in a controlled fashion only in treatment-naive patients, and I think that's a very significant distinction between what we studied in a majority ERT switch population.

John Crowley: So, for us, you know, we remain focused on completing our U.S. regulatory submission, the VLA submission, by the end of the second quarter. And then, again, as we've said to Tazeen, we'll continue regulatory filings throughout 2021 in Europe and beyond. In addition to that, we continue all of our preparations for additional clinical studies in infants and children that are ongoing. We continue our Compassionate Use Program. So, there are a lot of activities, a lot of milestones ahead in the next 6 to 12 months for Amicus, and that's where we remain focused.

<unk> focused on completing our U S regulatory submission of the BLA submission by the end of the second quarter and then again as we said to the and we will continue the filings throughout 2021 in Europe and beyond.

In addition to that we continue all of our preparations for additional clinical studies in infants and children that are ongoing we continue our compassionate use program. So there's a line a lot of activities a lot of milestones ahead on the next six to 12 months for amicus and that's where we remain focused I think our data speaks for itself in terms of.

John Crowley: I think our data speaks for itself in terms of the impact on patients who are ERT-experienced, particularly those who have been on for many, many years. The vast majority of the Pompe patients treated in this current billion-dollar-plus market have been on the approved medicine for a number of years. And some of the most powerful data that we've shown is in that ERT switch population, particularly those patients who have been on it now for more than 2 years, again, which is the vast majority of In the community, the Pompeii community, these are incredibly talented and experienced physician scientists who will be driven by the data, and the data will carry the day.

The impact on patients who are <unk> key experienced particularly those who have been on for many many years. The vast majority of the pompe patients treated in this current $1 billion plus market had been on the approved medicine for a number of years and some of the most powerful data that we've shown is in that <unk>.

Which population, particularly those patients who have been on now more than two years again, which is the vast majority of the population. So I've got great confidence in the community. The Pompe <unk> community. These are incredibly talented and experienced physician scientists who will be driven by the data and the data will carry the day.

Your next question comes from the line of Ritu <unk> with Cowen.

Operator: Your next question comes from the line of Ritu Baral with Calin. Hi guys, this is Anvita on for RITU this morning.

Hi, guys just us on the Doe on Friday until this morning.

From a P T a.

Jeffrey P. Castelli: For ATP-A, what additional secondary analyses might be forthcoming at medical meetings this year that could probably help further eliminate the drug's profile and differences from Neo-GA? And then, if I can just slip in one more question, what gives you more confidence, like what you're going to negotiate for the CLN6 pathway with the FDA would also translate to CLN3 or vice versa? Thanks for taking the questions.

What additional secondary analyses might be forthcoming at medical meetings. This year that could probably help but isn't it the drug's profile on defense as from the NGA and then if I can just slip in one more question.

What gives you more confidence like why can't go on to negotiate for C. N N six pathway would that make on nature of it with the MTA.

Also translate to see it and three our likelihood Isa.

For taking our questions.

Sure. So two different questions. There will take the first one on Pompe pay what additional secondary data do we expect we continue to run multiple secondary analysis looking at a range of the secondary endpoints that were pre specified in this study and us as we do that we have continued and increased confidence in a T. G. A a and its impact on these patients.

Jeffrey P. Castelli: Sure, so there are two different questions there. Let's take the first one on Pompeii.

Jeffrey P. Castelli: What additional secondary data do we expect? You know, we continue to run multiple secondary analyses looking at a range of the secondary endpoints that were pre-specified in the study, and as we do that, we have continued and increased competence in ATGAA and its impact on these patients. Jeff, do you want to comment on what people can expect in the future at medical conferences? Jeff, I think your phone, I think if you talk through the phone. I'm sorry, thank you, John. I did the wrong thing by you. Thank you. That's all right.

Jeff do you want to comment on on what people could expect debt ahead at medical conferences.

Jeff I think your phone.

I think if you talk to the phone I've got from sorry, Thank you John.

Thank you that's all right.

Jeffrey P. Castelli: As John mentioned, it really will be focused on looking at the additional secondary endpoints. So, the top-line data is really focused on the primary endpoint plus the predefined key secondary endpoints. So, we'll have additional measures of motor strength, motor function, muscle strength, pulmonary function, PROs, and really look to see that those additional measures continue to show the same pattern that we've seen in the primary and key secondaries, which is the vast majority of all these endpoints favoring ATGA, showing general improvements from baseline. And we think that as we see more and more of those endpoints all showing that same directionality supporting the main end We'll continue to also dig into subpopulations.

As John mentioned is really will be focused on looking at the additional secondary endpoint. So the top line data really focused on the primary endpoint but to.

Predefined key secondary endpoints so.

So we will have additional measures of motor strength.

On motor function and muscle strength pulmonary function PR.

And really looking to see that those additional measures continue to show the same pattern that we've seen in the primary and key secondary on switches.

The vast majority of all of these endpoints favoring a T J, a showing general improvements from baseline and we think that the as we see more and more of those endpoints all showing that same directionality supporting the main endpoints that that will strengthen what we've seen.

We will continue to also dig into subpopulations of patients that have been on air team longer or shorter periods of time, but.

Jeffrey P. Castelli: We'll continue to...

Jeffrey P. Castelli: the time. But really, the key data we believe we did disclose, you know, in the top line, so we don't see anything that's kind of...

But really the key data we believe we did disclose it on the top line. So we don't see anything that's going to dramatically change that one way or the other will just be supportive in nature.

Jeffrey P. Castelli: And then Jeff, maybe if you just want to tackle, I'm sorry, Operator, if you just want to tackle the CLN6 part of the question, kind of where we are there and some key activities ahead.

Yes.

And then John that would be if you just wanted to tackle I'm sorry, operator debt. If you just wanted to tackle this seeland fixed part of the question kind of where we are there and some key activities ahead.

Yeah. So C. L. On six we continue to follow the <unk> kids that have been treated now over two years post gene transfer I would continue to be really excited by the stabilization we've seen versus the expected decline in natural history.

Jeffrey P. Castelli: Yeah, so CLN6, you know, we continue to follow the 13 kids that have been treated now for over two years post-gene transfer. I would continue to be really excited by the stabilization we've seen versus the expected decline in natural history. We've been really working.

We've been really working hard on all the GMP manufacturing engaging with regulators to define the next clinical trial.

Jeffrey P. Castelli: We've been really working hard on all the GMP manufacturing.

And look to here in the second part of the year to start that next study up dosing.

Jeffrey P. Castelli: [inaudible]

Bradley L. Campbell: Your next question comes from the line of Anup Ram. Hi guys, good morning. This is Tess on the call for Anupam today. Just one from us on the commercial side, what are the core countries of geographical focus for Gallup Polls in 2021, and what are the considerations for Deeper Penetration versus Geographic Expansion.

Dosing kids by the end of the year with GMP material made us Thermo Fisher.

So really a lot of manufacturing and regulatory interactions this year, leading up to that clinical protocol is starting to dose additional children.

Your next.

Next question comes from the line of.

Wow.

Okay.

Yeah.

Hi, guys. Good morning, just as cash on the call from <unk>.

And just one from us on the commercial side.

What are the core countries of geographic expansion for gallon sold in 2021, and what are the considerations for deeper penetration versus geographic expansion. Thanks, so much.

Bradley L. Campbell: Thanks so much.

Bradley L. Campbell: Great, thank you. Bradley, I'll let you go ahead and seal that, please.

Bradley L. Campbell: Sure, yeah, we're not giving specific countries just from a competitive perspective, but I can tell you the areas of most geographic growth continue to be Latin America, Asia Pacific, and then Middle East and North Africa countries. And as I said, we're expecting over five to come online from a reimbursement perspective this year, so lots of growth there. Remember, too, from a geographic expansion perspective, there are a number of countries that we've only launched in in the last year or two.

Great. Thank you Bradley I'll, let you go ahead and field that please.

Sure Yes.

We're not giving specific countries just from a competitive perspective, but I can tell you the areas of most geographic growth continued to be Latin America Asia Pacific and then.

Middle East North Africa countries, and as I said, we're expecting over five to come online from a reimbursement perspective. This year, so lots of growth there.

Remember too from a geographic expansion perspective, there are a number of countries that we've only launched in the last year or to Japan in the US frankly to continue to be high growth countries, but also those those newer launch countries, which individually are relatively small but collectively become <unk>.

Bradley L. Campbell: Japan and the U.S., frankly, continue to be high-growth countries, but also those newer launch countries, which individually are relatively small but collectively have become, you know, substantial growth drivers. And I would say it's kind of a third, a third, a third.

Substantial growth drivers and I would say, it's kind of a third a third a third part of us continuing to switch patients in.

Bradley L. Campbell: Part of it is continuing to switch patients in markets where we've been launched. Part of it, as I mentioned, is continuing to find new patients and supporting a growing number of initiatives to do that. And then part of it is, just as we talked about, the geographic expansion. The last point I'll add is that, as the numbers are just being reported, the Fabre market today passed $1.6 billion in global sales, including our product, which is, I think, well on the way to a $2 billion-plus market. And so not only are we confident in our path to that $500 million milestone, but we're also looking forward to continuing to grow past that toward that billion-dollar opportunity.

Markets, where we have been launched part of it as I mentioned is continuing.

To find new patients and supporting a growing number of initiatives to do that and then part of it is us.

Just as we talked about the geographic expansion. The last point I'll add is that as the numbers I think were just being reported on the fabry market today passed $1 6 billion in global sales, including our product, which is I think well on our way to two 2 billion plus market.

So not only are we confident on our path to that 500 dollar milestone, but we're also looking forward to continue to grow past that towards that billion dollar opportunity.

Thank you your next.

Operator: Your next question comes from the line of Salveen Richter with Goldman Sachs. Thank you for taking our question, and good morning. This is Elizabeth on behalf of Salveen.

Your next question comes from the line of solving breakthroughs with Goldman Sachs.

Thank you for taking my question and good morning. This is a little bit back on <unk>. Just wondering when we may expect Pompeii and Fabry gene therapy to enter the clinic on we're looking for some updates here and then we're seeing the competitive landscape grow in and the dynamics emerge specifically for fabry.

Jeffrey P. Castelli: Just wondering when we may expect, you know, Pompei and Fabre gene therapies to enter the clinic. We're looking for, you know, some updates here, and then we're seeing the competitive landscape grow and the dynamics emerge specifically for Fabre gene therapy. Maybe could you remind us of some areas of differentiation from competitors here? Thank you.

Hi, gene therapy, maybe could you remind us on some areas of differentiation from competitors here. Thank you.

Yeah, that's actually may be a great place to start with our Fabry gene therapy. Again. This was jointly developed by the amicus science teams and Jim Wilson and his team again, combining the protein engineering experience at amicus with the vector technology and experience at the Wilson lab at U Penn and.

Jeffrey P. Castelli: Yeah, that's actually a great place to start with our Fabry gene therapy. Again, this was jointly developed by the Amicus science teams and Jim Wilson and his team. Again, combining the protein engineering experience at Amicus with the vector technology and experience at the Wilson Lab at UPenn. And maybe, Hung, if I can ask you just to describe, remind everybody again, what we released back in November, and again, what was highlighted at the world meeting just a few weeks ago.

And maybe hung if I can ask you just to describe remind everybody again, what we released back in November again, what was highlighted at the World meeting just a few weeks ago because it is a very unique approach and we think a very differentiated dataset in fabry disease. So.

Jeffrey P. Castelli: Because it is a very unique approach and we think a very differentiated data set in Fabry disease. So, Hung, if you'd comment on the Fabry gene therapy that we've developed, and then Jeff, I'll turn it to you for development activities and timelines. Yeah, sure, John. Hi, Foggy.

So hung if you'd comment on the February gene therapy.

And then Jeff I'll turn it to you for development activities and timelines.

Yeah, sure John Hi, Celgene.

So I think the main distinguishing feature for our Fabry gene therapy approaches that the protein that's actually produced from the gene therapy has made us a dimer us to say it's comprised of two identical subunits and they need to be balance together, Florida, Florida to be functional the reality is that protein Duffy.

Jeff Hung: I think that the main distinguishing feature of our Fabry-King therapy approach is that

Jeff Hung: The protein that's actually produced from the gene therapy is made as a dimer. That is to say, it's comprised of two identical subunits, and they need to be bound together for...

Operator: https://www.youtube.com

Not very stable and therefore, it actually starts to fall apart from becomes inactive what we have done us to engineer the transgene. So what actually gets produce maintains debt dimer structure.

Jeff Hung: And therefore, it actually starts to fall apart and become inactive. What we have done...

Structure. So it maintains its activity and so the.

Jeff Hung: [inaudible] As a result, the protein that's made is much more stable and much more potent. And so, therefore, we're able to achieve significantly greater in vivo efficacy while producing the same amount of protein relative to the wild-type construct. So Jeff, let me turn it over to you in terms of talking about the development of timelines.

The end result is actually on the protein that's made us much more stable and much more potent and so therefore, we're able to achieve significantly greater in people efficacy, while producing the same amount of protein relative to the wild type constructs.

So Jeff let me turn it over to you in terms of talking about the development timelines.

Yes.

Sure. Thanks, Tom.

Jeffrey P. Castelli: Thanks, everyone.

Yes.

Based on that Great science of hunger subscribed in Fabry and then also remember for Pompe <unk>, our transgene there was engineered for.

Jeffrey P. Castelli: So, based on that great science that Hung just described in Fabry and then also, remember, for Pompeii, our transgene there was engineered for optimized uptake into cells. Those IND candidates with Amicus-engineered transgenes using Penn's proprietary capsid technologies are declared as IND candidates in ongoing two paths of development. One is focused on the IND-enabling toxicology work, and the other is on the CMC process development work to get GMP material for that first clinical trial. Those parallel paths are ongoing.

Optimize uptake into cells.

Those candidates for amicus engineered trans genes using patented proprietary constant technologies.

Our our declared a <unk> 90 candidates and an ongoing two paths of development. One is focused on the IND, enabling toxicology work and the other is on the CMC.

Process development work to get GMP material for that first clinical trial.

Those parallel paths are ongoing.

Jeffrey P. Castelli: What is likely to be rate-limiting is the manufacturing there. We intend to go into those next clinical trials with material from something that is as close to possible as our commercial process. And as we have more clarity on the timeline for manufacturing GMP material, we'll provide updates on the specifics of the IND, but we're making really good progress on both paths across both programs.

It is likely to be right when they didn't give us the manufacturing there we intend to go into those next clinical trials with material from something that is as close to possible as our commercial process and as we have more clarity on the time line to the manufacturing GMP material will provide updates on the specifics on the IMD, but.

We're making really good progress on both paths across both programs.

Great. Thank you, Jeff Elizabeth I'll, just add too for the Fabry gene therapy market I think it's very exciting for fabry patients that were finally, starting to see after so many years. Some of these fabry gene therapies come into the clinic, obviously, they're very different range of technologies with a.

John Crowley: I think it's very exciting for febrile patients that we're finally starting to see, after so many years, some of these febrile gene therapies come into the clinic. Obviously, a very different range of technologies with different burdens, frankly, on patients.

Different burdens frankly on patients we do see in the decade or more ahead for gene therapy in Fabry disease, there will be a role we do think it would be most applicable us certainly in the early years post approval to non amenable patients those patients for whom only enzyme replacement therapy is there.

John Crowley: We do see that in the decade or more ahead for gene therapy in febrile disease, there will be a role. We do think it would be most applicable, certainly in the early years post-approval, to non-amenable patients, those patients for whom only enzyme replacement therapy is their choice. We do think, for patients with amenable variants, those patients on Galifold, that they would be the last to adopt a febrile gene therapy, given the nature of Galifold as an oral precision medicine, its convenience, and its profile.

Joyce we do some for patients with amenable variance those patients on Gallup polled that they would be the last to adopt a fabry gene therapy, given the nature of <unk> and world precision medicine the convenience.

Profile. So we're committed to fabry gene therapy, we think we have a very differentiated approach and we look forward to taking it into.

John Crowley: We're committed to febrile gene therapy. We think we have a very differentiated approach, and we look forward to taking it into the clinic. We do know, though, that in febrile diseases, especially given the approved therapies, these will certainly be robust development programs for anybody in this field.

Into the clinic ahead, and we do know, though in February, especially given the approved therapies.

These will will certainly be robust development programs for anybody in this field.

Great. Thank you your net.

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

Your next question comes from the line of small heat sales with Citigroup.

Great. Thanks for taking my question.

John Crowley: Thanks for taking my question. Just one question on the guidance. So if I look at the year-over-year growth in 2021, and even look at the upper end of $315 million, you're guiding for a 21% annual increase. However, from this point to the 2023 guidance of $500 million plus, it calculates to about 26% CAGR. So just wondering if you could help us understand what goes behind this acceleration of growth assumption there.

Just one question on on the guidance.

So if I look at the year over year growth in 2021, and even at looking at the upper range of $10 million to $15 million you are guiding for 21% annual increase.

From this point through 2022 guidance of $500 million flow.

It calculates to about 26% CAGR.

So just wondering if you could help us understand what goes behind us exploration of growth assumption there. Thank you.

John Crowley: Yeah, Moet, thank you. It's good to talk with you.

Oh. Thank you good to talk with you I'll ask Brad to field that I'll, just say upfront again to remind everybody continued growth great demand continues we do see the building queue of patients and we see much of that growth then being alleviated post COVID-19, we expect beginning in the mid year.

John Crowley: I'll ask Brad to field that. I'll just say up front again to remind everybody, continued growth, and great demand continues. We do see the building queue of patients, and we see much of that growth then being alleviated post-COVID. We expect, you know, beginning in the mid-year, with particularly strong growth in the second half of this year and into 2022. So, Bradley, I'll ask you to provide more context and color

With particularly strong growth in the second half of this year into 2022, So Bradley I'll ask you to provide more context from color there. Please.

Bradley L. Campbell: Yeah, John, I think that's right. I think, you know, we are assuming a second half recovery this year from some of the challenges of COVID that we saw at the end of last year. And we do see some acceleration coming out of that and expect that to continue as we get towards that $500 million milestone. You know, we continue to see diagnosis rates increase, the overall market is growing, and we're continuing to launch in new countries.

Yes, John I think that's right I think we are ex.

Assuming a second half recovery this year from from some of the challenges of Covid that we saw at the end of last year.

And we do see some acceleration coming out of that and.

And expect that to continue as we get towards that $500 million milestone.

We continue to see diagnosis rates increase the overall market is growing we're continuing to launch a new countries. So lots of opportunity to sort of take advantage of that acceleration going into the next few years and really continuing to grow beyond that.

Bradley L. Campbell: So lots of opportunity to sort of take advantage of that acceleration going into the next few years and really continuing to grow beyond that. Again, as I mentioned earlier, we see this as potential to grow upwards of a billion dollars based on all the dynamics we're seeing. So what we know is that demand continues to be strong, that patients continue to stay on GALFOLD at that over 90% compliance and adherence rates, and we continue to focus on those three growth drivers I mentioned earlier.

As I mentioned earlier, we see this as a potential to grow upwards of $1 billion based on all the dynamics, we're seeing so what.

What we know is that demand continues to be strong debt.

Patients continue to stay on Gulf holder that over 90% compliance and adherence rates and we continue to focus on those three growth drivers I mentioned earlier.

I hope that's helpful. Mike Thank you.

Bradley L. Campbell: I hope that's helpful, Moet. Thank you.

Operator: Your next question comes from the line of Joseph Schwartz with SBB Learing. Hi, I'm Juri Dailing here in for Joe. Thanks for taking our question. I just wanted to ask you a question about your gene therapy program for Pompeii. I was just wondering if your strategies for your Pompeii gene therapy program have changed based on the heterogeneity of the patient population and your ProPAL results. You know, could you talk about how you plan to achieve what you have for this program?

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

Sure.

Hi, I'm Jerry dialing in for Joe Thanks for taking our question.

Wanted to ask you a question on your gene therapy.

Program from Pompe, Paul I was just wondering if your strategy or your Pompe gene therapy program has changed based on that.

Many of the patient population in your per powers. All you know could you talk about how you plan to achieve what you envision force program.

Sure. Thanks, Mary I'll, just say that it hasnt changed in view of propel obviously propel and all of the clinical work and published studies continue to inform us.

John Crowley: Sure. Thanks, Marie.

John Crowley: I'll just say that it hasn't changed in view of Propel. Obviously, Propel and all the clinical work and published studies continue to inform the development program for Pompei gene therapy. Again, to remind everybody, Pompei gene therapy has been in development in the field broadly for well more than 20 years. When we came to this in Pompei, we wanted to bring a different approach.

Development program for Pompe gene therapy, again to remind everybody Pompe gene therapy has been in development in the field broadly for well more than 20 years. When we came to this income pay we wanted to bring a different approach again reminding everybody that our technology is to engineer the transgene to allow for Maxim.

John Crowley: Again, reminding everybody that our technology is to engineer the transgene to allow for maximal targeting to muscle of the protein expressed by the gene therapy, which will lead not only to better efficacy but potentially improve safety and, obviously, much more manufacturable of a product. So, in that regard, the core technology and the core focus remains the same. I'm sure there'll be continued learnings as we move ahead. We're considering patient populations all the way from infants through adolescents to adults as well. That development program is something we're working on in earnest now.

Targeting to muscle of the protein expressed by the gene therapy, which will lead not only we believe to better efficacy, but potentially improved safety and obviously much more manufacturer book of a product. So in that regard that core technology and our core focus remains the same I'm sure there'll be continued earnings.

As we move ahead, we're considering the patient populations all the way from infants through adolescence, two adults as well and that development program is something we're working on an earned us now.

Operator: Your next question comes from the line of Kristen Kluska with Canterbury Jail. Hi, good morning. Thanks for taking my question. Hi, Kristen. Good morning.

Your next question comes from the line of Kristen cluster with Cantor Fitzgerald.

Hi, Good morning, Thanks for taking my question on Chris Good morning, and John Thanks for sharing your experience with Rosebel on for all the work. Your team is conducting for these patients with rare diseases.

Jeffrey P. Castelli: And, John, thanks for sharing your experience with Rosella and for all the work your team is conducting for these patients with rare diseases. So, the question I have here is, as you continue to advance your gene therapy pipeline forward, could you remind us which indications you think have already established natural history data in place? So, for example, you were able to demonstrate the improvements for CLN6 and CLN3 Batten disease in light of your understanding of the natural course of these diseases. So, to that end, are you also thinking of conducting any other natural history studies for some of the earlier programs?

So the question I have here is.

As you continue to advance our gene therapy pipeline forward could you remind us which indications you think there are already established natural history data in place. So for example, you were able to demonstrate the improvements for <unk> six and ceiling three batten disease in light of your understanding of the natural course of disease diseases. So.

To that end are you also thinking of conducting any other natural history studies for some of the earlier programs.

Yeah, I'll turn it to Jeff. Thank you, Chris that's a great question and it's going to be very important in all of US neuro programs to have robust natural history's, Jeff do you want to comment on kind of where we are with both ceilings and steel and three and then more broadly how we think about natural history.

Jeffrey P. Castelli: Yeah, I'll turn it to Jeff. Thank you, Chris. That's a great question. It's going to be very important in all of these neuro programs to have robust natural histories. Jeff, do you want to comment on kind of where we are with both CLN6 and CLN3, and then more broadly, how we think about natural histories as comparator arms for development here?

Parador arms for development here.

Jeffrey P. Castelli: Sure. And hi, Kristen.

Sure on Hi, Kristen.

Jeffrey P. Castelli: So in the Batten diseases, there's pretty, depending on the subtype, the amount of existing natural history data varies. CLN3, for example, actually has the most natural history data already currently available, a lot of it collected prospectively with that UBDRS endpoint.

So in the batten diseases, there's a pretty depending on the subtype.

On a existing natural history data various sales on three they're actually has the most natural.

Natural history data already currently available a lot of it collected prospectively with that you'd be drs endpoint.

Jeffrey P. Castelli: We're going to supplement all of that with natural history. We've already started our own natural history study across the Batten subtypes to collect both retrospective and prospective data. So we think, you know, for Batten diseases, that would be a key part of our development path for CLN6, 3, as well as to get into CLN1 and other subtypes. As you look at some of the earlier programs, you know, CD-Cal5 deficiency disorder, for example, is a relatively new disease.

We're going to supplement all of that natural history, we've already started up our own natural history study across the batten subtypes to collect both retrospective and prospective data. So we think.

For batten diseases that would be a key part of our development path for sale on <unk> III as well as they get into see on line one and other subtypes.

If you look at some of the earlier programs.

<unk> deficiency disorder for example, that's a relatively new disease.

Jeffrey P. Castelli: The natural history data is a little bit less, but we're already looking to start work there, working collaboratively with others in the community to start to work on some of that natural history, and then NPS 3A and 3B. Similarly, there's some data that's out there, and we'll continue to, early on in development, start to supplement those efforts and try to pull together the natural history. We think it's a key part of development for these, you know, ultra-rare diseases.

On the history data is a little bit less but we're already looking to start work there working collaboratively with others on the community to start to work on some of that natural history.

And then <unk> rebate. Similarly, there is some data that's out there and we will continue to on early on in development start to supplement those efforts and tried to pull together natural history. We think its a key part of development for these ultra rare diseases.

As you look at our Fabry Pompe day programs, obviously, there we think that there's lots of data, but we think controlling trials will be needed for new gene therapies. So a little less focus on obviously on that front book for the early programs. It's something that we think it's critical to address early in the development programs building on what the communities already doing.

Jeffrey P. Castelli: You know, as you look at our Fabri-Pompe programs, obviously, there, we think that there is lots of data, but we think controlled trials will be needed for new gene therapies. So, a little less focus, obviously, on that front, but for the early programs, it's something that we think is critical to address early in the development programs, building on what the community is already doing.

Yes.

Yes.

Your next question comes from the line of step cheat channel.

Operator: Your next question comes from the line of Deb Cheat, Chattopadhyay, with Guggenheim Security.

With Guggenheim Securities.

Yeah.

Hi, guys. Good morning, this is Aaron on Snapchat.

Operator: Hi guys. Good morning. This is Aaron on The DevJet.

No.

Okay.

Couple of quick questions any interaction from the agency on propel study and considering the stats plan accounted for evaluation of SBC at second endpoint.

Jeffrey P. Castelli: Any interactions from the agency on the PAPEL study?

Jeffrey P. Castelli: and considering the STATS plan accounted for evaluation.

Jeffrey P. Castelli: This is a question about the evaluation of FDC at a second endpoint. Was the significance level specified any different from the first endpoint?

Was the significance level specified any different than the usual point clarify.

Jeffrey P. Castelli: as usual.

Jeffrey P. Castelli: and the usual.05.

Jeffrey P. Castelli: Yeah, just on the first part again, our plan remains to complete the BLA submission by the end of the second quarter, and we don't comment on ongoing regulatory discussions. And I just want to be clear on your second point. Maybe, Jeff, just remind people of FDC, its importance, and what we saw there, both in the overall population and the switch population.

Yeah, just on the first part again, our plan remains to complete the BLA submission by the end of the second quarter and we don't comment on ongoing regulatory discussions and I just want to be clear on your second maybe Jeff just remind people of FCC it's important.

What we saw there both on the overall population in the switch population.

Jeffrey P. Castelli: Yeah, so, you know, Propel was designed as superiority versus approved therapy, and the primary endpoint was a six-minute walk.

Yes, so propel was designed as superiority versus US approved therapy primary endpoint was six minute walk. The first key secondary hierarchically was that B C. But we demonstrated was nominal superiority with that key value below point on five that we showed.

Jeffrey P. Castelli: The first key secondary hierarchically was FEC. What we demonstrated was nominal superiority with that C value below 0.05 that we showed. We had other endpoints as well in the study. As I mentioned in the slide we covered on post hoc non-inferiority, we'd also pre-specified some non-inferiority analyses on FEC, which we actually did not need to formally conduct as we hit on nominal superiority on that first key secondary of FEC.

You had other endpoints as well on this study as I mentioned in the slide we covered on the post hoc non inferiority, we'd also pre specified some non inferiority analysis on FTC, which we actually did not need to formally conduct as we hit on nominal superiority on that first key secondary efficacy.

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

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

Operator: Great, operator, thank you. So again, 2020 was a great year. We've got a lot of work ahead of us here still in 2021, but I wanna reassure everybody that you've got, again, a passionate team of entrepreneurs at Amicus who remain relentlessly focused on our mission. So thank you, everybody. Have a great day. Ladies and gentlemen, this concludes today's conference call. Thank you, and have a great day!

Great operator. Thank you. So again 2020 was a great year, we've got a lot of work ahead of us Youre still in 2021, but I want to reassure everybody that <unk> got again, a passionate team of entrepreneurs at amicus who remain relentless relentlessly focused on our mission. So thank you everybody have a great week.

Ladies and gentlemen. This concludes today's conference call. Thank you and have a great day you may now disconnect.

Operator: Ladies and gentlemen, this concludes today's conference call. Thank you, and have a great day. You may now disconnect.

Operator: Very well done, everybody.

Yeah.

Okay.

Yeah.