Q2 2020 Amicus Therapeutics Inc Earnings Call

Right and wall constant mucus curve to your second quarter 2020 financial results Conference call and look at this time all participants are in listen only mode. Later, we'll conduct a question answer session.

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

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Operator: As a reminder, this conference call is being recorded. I'd now like to turn the conference over to your host, Mr. Andrew Faughnan, Director. Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics' second quarter 2020 financial results and corporate highlights. Speaking on today's call are John Crowley, Chairman and Chief Executive Officer, Bradley Campbell, President and Chief Operating Officer, Daphne Quimi, Chief Financial Officer, and Dr. Jeff Castelli, Chief Development Officer.

A reminder, this conference call is being recorded I'd now like to turn the conference over to your host Mr., Andrew fauna director of Investor Relations you may begin.

Good morning, Thank you for joining our conference call to its Scott Amicus Therapeutics second quarter 2020 financial results in corporate highlights.

Speaking on today's call, we have John Crowley, Chairman and Chief Executive Officer, Bradley Campbell, President and Chief Operating Officer, Dr., Mcqueeney, Chief Financial Officer, Dr. Jeffs Castelli, Chief Development Officer as referenced on slide two we may make forward looking statements within the meaning of the private Securities Litigation Reform Act of 1995.

Andrew Faughnan: As referenced on slide two, we may make forward-looking statements within the meaning of the Private Security 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 on this call may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. You are cautioned not to place undue reliance on any forward-looking statements, which speak only to the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this presentation and conference call to reflect events or circumstances after the date hereof.

Relating to our business as well as our plans and prospects.

<unk> looking statements should not be regarded that's your presentation by us, but any of our plants will be achieved any or all the forward looking statements made on this call may turn out to be wrong. It can be affected by inaccurate assumptions, we might make whereby known or unknown risks and uncertainties.

You are cautioned not to place undue reliance on any forward looking statements, which speak only to the date hereof. All forward looking statements are qualified in their entirety by this cautionary statement and we undertake no obligation to revise or update this presentation and conference call to reflect events or circumstances. After the date hereof.

Andrew Faughnan: For a full discussion of such forward-looking statements and the risks and uncertainties that may impact them, we refer you to the forward-looking statements and risk factors section of our annual report on Form 10-K for the year ended December 31, 2019, and the quarterly report on Form 10-Q for the quarter ended June 30, 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 Jon Crowley, Chairman and Chief Executive Officer. Jon?

For full discussion such forward looking statements and the risks and uncertainties that that may impact them. We refer you to the forward looking statements and risk factor section or annual report on form 10-K pretty year ended December 31st 2019, and the quarterly report on form 10-Q quarter ended June Thirtyth 2020 beef.

Filed later today with the Securities and Exchange Commission.

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

Great. Thank you Andrew Good morning, and welcome everyone to our second quarter Twentytwenty results Conference call as we did last quarter, let me start by stating that I hope everybody and their families are well one thing.

John Crowley: Great, thank you Andrew. Good morning, and welcome everyone to our second quarter 2020 results conference call. As we did last quarter, let me start by stating that I hope everybody and their families are well and safe. Our leadership team at Amicus continues to emphasize a range of programs and initiatives to protect and support our global workforce during this ongoing pandemic. While adapting to all of the changes brought about by the global pandemic, for Amicus, the first half of 2020 has been a period of excellent growth and execution across all aspects of our business, including science, clinical, regulatory, and, as you see, our commercial efforts, as we continue to build one of the next great global biotechnology companies poised to impact people around the world living with rare diseases.

Our leadership team it and it just continues to emphasize a range of programs and initiatives.

To protect and support our global workforce during this ongoing pandemic.

While adapting to all of the changes brought about by the global pandemic parameters. The first half was Twentytwenty has been a period of excellent growth and execution across all aspects of our business, including sign clinical regulatory and as you see our commercial effort.

As we continue to feel one at the next three local biotechnology companies poised to impact people around the world living with where diseases.

As we did in this mornings press release I'd like to highlight several key accomplishment.

John Crowley: As we did in this morning's press release, I'd like to highlight several key accomplishments. First, Yalopold continues its strong launch performance and remains the cornerstone of our success. With $62.4 million in second quarter revenue, the Gallup Hold launch continues to exceed expectations. Second quarter revenue represents the performance across the global business, including new patient starts from both switch and treatment naive patients throughout the quarter in all major regions, including those hardest hit by COVID-19. Second, our R&D timelines remain on track. We continue to expect phase three of the ATGAA propel study in late-onset Pompe disease to read out in the first half of 2021. Additionally, the rolling VLA submission for ATGAA remains on schedule, and we expect the first submission later this year. Within our gene therapy pipeline, we continue to move forward our lead batten disease programs with CLN6 and CLN3, as well as our most advanced preclinical programs in gene therapy, and third, following our strategic financing in July. The Amicus cash position is sufficient now to achieve profitability without the need for any future diluted financing.

First Galafold continues its strong launch performance and remains the cornerstone of our success.

$62.4 million in second quarter revenue.

I will hold launch continues to exceed expectations.

Second quarter revenue represents the performance across the global business, including new patient starts from both switch and treatment naive patients.

Ralph quarter in all major regions.

Moving those hardest hit like hoping thinking.

Second jar R&D time lines remain on track, we continue to expect series phase three propel study of 80 G.A. in late onset pompei disease to read out in the first how lucky 21.

Additionally, the rolling via Lasik mission for a C.G.A. remains on schedule and we expect the first submission later this year.

We did our gene therapy pipeline, we continue to move forward our lead batten disease programs, we're seeing in six and CLM three.

Well as our most advanced preclinical programs in gene therapy.

And third following our strategic financing in July.

And it gets cash position is sufficient now to achieve profitability without the need for any future completed financing.

Our continued revenue growth.

John Crowley: Our continued revenue growth, prudent expense management, and growth potential have allowed us to reach this important milestone of self-sustainability as we continue to realize our vision of delivering groundbreaking and potentially curative new medicines for people living with rare diseases around the world. Turning now to slide four, we are well on track to achieve our five key strategic priorities for 2020, including first, Yellowfold, our precision medicine for febrile disease. We will continue to drive Gallup Polls to more people living with febrile disease with minimal variance in existing and new markets around the world. We look to achieve global product revenue.

I wouldn't expense management and growth potential has allowed us to reach its important milestones. So sustainability as we continue to realize our vision of delivering groundbreaking and potentially curative new medicines for people living with rare diseases around the world.

Turning now to slide four we're well on track to achieve our five key strategic priorities for Twentytwenty.

Including first.

Yes, awful our precision medicine for Fabry disease.

We will continue to drive yellow hold to more people living with fabry disease, with a minimum ovarian and existing and new markets around the world.

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

John Crowley: $250 million to $260 million. Second, we are increasing the clinical regulatory manufacturing and pre-commercial activities and planning surrounding our Pompe program for ATGAA as we move this important therapy toward approval. Third, again, we are advancing our industry-leading rare disease gene therapy portfolio stemming from our new Global Research and Gene Therapy Center of Excellence in Philadelphia. We will be advancing the clinical development, manufacturing, and regulatory discussions for both our CLN-6 and CLN-3 BATN programs. Fourth, in addition, we are progressing our Pompe gene therapy towards IND, and we plan to disclose up to two additional IND candidates this year. A lot of work is underway with our manufacturing partners for the manufacture and scale up of the Pompe gene therapy, as well as our other potential IND candidates. So again, we look forward to sharing the additional IND candidates from our PEN collaboration later this year. Finally, we will continue to maintain a strong financial position as we carefully manage our expenses and our investments, and we remain fully funded now for profitability.

Second we are increasing.

The clinical regulatory manufacturing and pre commercial activities and planning surrounding our campaign program for 80 GA here as we move this important therapy toward approval.

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

Stemming from our new Global research in Gene therapy Center of excellence in Philadelphia, we will be advancing the clinical development manufacturing and regulatory discussion for both our steel and B and C. L. Three baton program.

Fourth in addition, we are progressing our clumping gene therapy towards I envy and we plan to disclose <unk> to two additional hi, Andy Kennedy This year.

A lot of work is underway with our manufacturing partners for the manufacturing scale up of the Pompey gene therapy, as well as or other potential I envy candidate.

So again, we look forward to sharing the additional R&D candidates from Merck and collaboration later this year.

Yeah again, we will continue to maintain a strong financial position as we carefully manage our expenses.

Our investment.

And we remain fully funded now to profitability.

So.

With that before I turn call over to our teams to provide more detail on the strong quarter I just wanted to take a moment to acknowledge dr. Ted lugs retirement Neonic his board of directors.

John Crowley: With that, before I turn the call over to our team to provide more detail on the Strong Quarter, I just wanted to take a moment to acknowledge Dr. Ted Love's retirement from the Amicus Board of Directors. Ted, as many of you know, is a remarkable leader, a great friend of mine, and a fierce advocate for innovation for patients. Ted and I will continue to work closely together as the Chair and Vice Chair of the Emerging Companies Section of the BIO Board, and I would very personally like to thank him for his nearly decade of service on our board. He has helped to put Amicus in a much stronger position, so thank you, Ted. With that, let me turn the call now over to Bradley Campbell, our President and Chief Operating Officer, to further highlight the strong Gallup Hold performance. Bradley

And as many of you told me as a remarkable leader a great friend of mine and a fierce advocate for innovation for patients.

Ted and I will continue to work closely together is the chair and Vice chair of the emerging companies section on the buy a board.

I would in a very personally like to thank him for his nearly a decade of service on our board. He has helped to put amick isn't a much stronger position. So thank you Ted.

With that let me turn the call now over to Bradley Campbell, our President and Chief operating Officer further highlights the strong Galafold performance right now.

Great. Thanks, John Good morning, everyone.

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

As John mentioned I'll now walk you through in more detail, our galafold performance for the quarter and let me start on the continued growth of Galafold revenue with the second quarter of 2020 here on slide six.

Bradley L. Campbell: As John mentioned, I'll now walk you through in more detail our Gallup Hold performance for the quarter. And let me start on the continued growth of Gallup Hold revenue in the second quarter of 2020 on slide six. And this is where we give our global snapshot of Galliford commercial progress. For the second quarter, as John mentioned, total product revenue was $62.4 million, driven by strong patient demand, favorable reimbursement dynamics, and business continuity, even during the worst quarter of COVID so far. And importantly, our global compliance and adherence rates continue to exceed 90%. The geographic breakdown of revenue during the quarter was $41.5 million, or 67% of revenue generated outside the United States, and the remaining $20.8 million, or 33%, coming from within the United States. On a sequential basis, you'll note that XUS quarterly revenue did decline slightly from Q1 to Q2 due to the timing of orders.

And this is where we give our global snapshot of the Galafold commercial progress.

For the second quarter as John mentioned total product revenue was $62.4 million driven by strong patient demand favorable reimbursement dynamics at business continuity, even during the worst quarter of covert so far and importantly, our global compliance and adherence rates continue to exceed 90%.

The geographic breakdown of revenue during the quarter was 41.5 million or 67% of revenue generated outside the United States and the remaining 20.8 million or 33% coming from within the United States.

On a sequential basis, you'll note that ex us quarterly revenue did decline slightly from Q1 Q2 due to the timing of orders. However, we continue to see strong broken patients added during second quarter and that strength has continued now into the first month of Q3. So we expect to finish the year around 70, 37 X U.S. to U.S. sales.

Bradley L. Campbell: However, we continue to see strong growth in patients added during the second quarter, and that strength has continued now into the first month of Q3, so we expect to finish the year around 7030 XUS for U.S. sales. As a reminder, we now have over 40 countries around the world with regulatory approvals and commercial sales now in over 30 of those countries. And this expanding global footprint is important not just to support the continued growth of patients with access to Galafold, but it really lays a strong foundation that is highly leverageable to support the potential launch of ATGAA for Pompeii and our future products as well. One other interesting aspect of the expanding market access for Galliford, which is highlighted on this slide, and this is quite unique, is that we have now added over 1,000 additional amenable mutations to the European The previous 300 mutations were all associated with specific patients who'd been identified with those mutations. These new mutations represent nearly all of the possible missense mutations in the GLA gene that are amenable to Gallifold as determined by our amenability assay.

As a reminder, we have now over 40 countries around the world with regulatory approvals and commercial sales now in over 30 of those countries.

And this expanding global footprint is important not just to support the continued growth of patients with access to galafold, but it really lays a strong foundation that it's highly leverageable to support the potential launch of 80, GA for pump pay and our future products as well.

[noise] one other interesting aspect to be expanding market access to Galafold, which is highlighted on the slide and this is quite unique is that we now have added over 1000 additional amenable mutations to the Europeans level.

The previous 300 mutations refer all associated with specific patients who had been identified with those mutations.

These new mutations represent nearly all of the possible missense mutations in the GE laid genes that are amenable to galafold as determined by our men ability assay.

Bradley L. Campbell: So when a physician now diagnoses a new patient with one of these mutations, they will no longer have to wait for us to characterize the mutation and run it through the amenability assay, a process which could take several months. Now the physician can simply refer to the website and check the amenability of the mutation, and the patient can get immediate access to Galpol.

The physician now diagnosis, the new patient with one of these mutations so no longer have to wait for us to characterize the mutation and run it through the men ability assay a process, which previously could take several months.

Now the physician can simply refer to the website to check the amount ability of the mutation and the patient can get immediate access to galafold.

Bradley L. Campbell: Again, a really exciting application and a really unique application of the pharmacogenetic aspects of this medicine, and one that we're looking to apply to other geographies as well. Turning now to slide 7, we had a very strong quarter even in the face of the COVID-19 pandemic. Our global supply chain remains fully intact, our customers have great confidence that they can access Galliford, and our field team has been able to achieve a significant portion of their pre-COVID touchpoints through digital, telephonic, and other means of interacting with their physicians. All of this has led to continued uptake with new patient starts all throughout the crisis in both switch and treatment naive patients. Even in the hardest-hit countries like the UK, France, Italy, Spain, Japan, the U.S., and many others, we were able to bring patients to Galliford throughout the quarter.

Again, it really exciting application in a really unique application of the pharmacogenetic aspects of this medicine and one that we're looking to apply to other geographies as well.

Turning now to slide seven we've had a very strong quarter, even in the face of the cobot 19 per dollar pandemic.

Our global supply chain remains fully intact, our customers have great confidence that they can access galafold and our field team has been able to achieve a significant portion of their pre cobot touch points through digital telephonic and other means interacting with their positions. All of this has led to continued uptake with new patient starts all throughout the crisis.

In both switch and treatment naive patients even in the hardest hit countries like UK, France, Italy, Spain, Japan, U.S. and many others, we were able to bring on patients to galafold throughout the quarter.

And it's important to note that in a country by country basis local conditions allow in in a very carefully prescribed manner. We're now, allowing our field organization to go back out to visit their positions in key accounts.

Bradley L. Campbell: And it's important to note that on a country-by-country basis, as local conditions allow and in a very carefully prescribed manner, we're now allowing our field organization to go back out to visit their physicians on key occasions. We believe that these dynamics may continue to provide some tailwinds even as the evolving global pandemic inevitably causes some headwinds, which we'll continue to closely monitor as the full impact of the pandemic currently remains unknown. From a numbers perspective, you can see that the second quarter sales increased 41% from the second quarter of 2019, which does include a 2% negative impact from foreign exchange. So from a true operational perspective, sales increased by 43% compared to last year.

We believe that these dynamics may continue to provide some tailwind even as the evolving global pandemic inevitably has caused some headwinds which will continue to closely monitor as the full impact the pandemic currently remains unknown.

From a numbers perspective, you can see that the second quarter sales increased 41% from second quarter 2019, which does include a 2% negative impact from foreign exchange. So from a true operational perspective sales increased by 43% compared to last year.

On the left hand side, we show a quarterly performance.

Bradley L. Campbell: On the left-hand side, we show our quarterly performance over the past several quarters. And as we've mentioned on previous calls, while we continue to expect strong growth quarter-to-quarter, due to a variety of factors, the rate of growth is typically nonlinear. Looking to the back half of the year, as we've seen in many years past, we expect higher quarter-on-quarter growth in Q4 as compared to Q3, which means we expect a larger portion of the revenue in the second half of the year will fall into the fourth quarter. But overall, as we've just finished the seventh month of the year here in July, we continue to be very confident in our guidance of $250 million to $260 million in full-year global sales.

Over the past several quarters.

And as we've mentioned on previous calls, while we continue to expect strong growth quarter to quarter due to a variety of factors the rate of growth is typically nonlinear.

Looking to the back half of the year as we've seen in many years past, we expect higher quarter on quarter growth.

For as compared to Q3, which means we expect a larger portion of the revenue in the second half of the year will fall into the fourth quarter.

But overall as we've just finished the seventh month seventh month of the year here in July we continue to be very confident in our guidance of 250 million to 260 million in full year global sales.

Now on slide eight.

Bradley L. Campbell: Now on slide eight, with several years of performance behind us and now launched in most major markets around the world, we can confidently say we are on a path to that $500 million sales opportunity in 2023. As I've outlined previously, to get to that $500 million, we expect a five-year average CAGR of about 40% from 2018 to 2023, and we expect to generate $1 billion in cumulative revenue between 2020 and 2022 alone, which goes a long way towards funding our R&D and OPEX over that period. We also have even more confidence now in the $1 billion revenue opportunity at peak as we continue to see significant growth in the Fabre market globally, driven by continued diagnosis from high-risk screening, newborn screening, and other diagnostic initiatives, in addition to penetration into the diagnosed untreated population.

Well years and performance behind US now launched at most major markets around the world confidently say, we're on a path to that 500 million dollar sales opportunity in 2023.

As I've outlined previously used to get to that $500 million. We expect a five year average CAGR of about 40% from 2018 to 2023, and we expect to generate $1 billion cumulative revenue between 2020, and 2022 alone which goes a long way towards funding our R&D and open.

It's over that period.

We also have even further confidence now in the 1 billion dollar revenue opportunity at peak as we continue to see significant growth in the fabry market globally, driven by continued diagnosis from higher screening newborn screening and other diagnostic initiatives. In addition to penetration into the diagnosed and treated population.

As we have orphan exclusivity in the U.S. in Europe. In addition to our multiple Orange book listed patents that give us an IP converts into the late twenties thirtys. So we have lots of opportunity to provide access to galafold for a long period to come.

Bradley L. Campbell: As we have orphan exclusivity in the U.S. and Europe, in addition to our multiple Orange Book-listed patents that give us IT coverage into the late 2030s, we have lots of opportunity to provide access to Galliford for a long period. With that, let me hand the call now over to Dr. Jeff Castelli, our Chief Development Officer, who will further highlight our LEAD pipeline programs. Thanks, Bradley, and good morning, everyone.

With that let me hand, the call now over to Dr., Jeff because selling our Chief development Officer, who will further highlight our lead pipeline programs Jeff.

Yeah, Thanks, Bradley and good morning, everyone.

Moving onto our R&D updates on slide 10, we wanted to remind everyone of our highly differentiated Pompey therapy, 80, GA and its mechanism of action.

Jeffrey P. Castelli: Moving on to our R&D updates on slide 10, we wanted to remind everyone of our highly differentiated Pompe therapy, ATGAA, and its mechanism of action. ATJA is our novel, next-generation therapy consisting of ATB200, or Cytoglucosidase Alpha, an investigational human recombinant GAA enzyme designed to target muscle cells throughout the body, administered by IV infusion, combined with AT2221, an orally-ad AT2221 is administered shortly before the infusion of ATB200 begins and is intended to bind and stabilize ATB200 in circulation, allowing more active enzymes to be taken up into cells and delivered to life. The combination of ERT and an enzyme stabilizer is one major distinction from the standard of care and other treatments in development for Pompeii. The other, and we believe potentially more impactful..., is the unique carbohydrate profile of

Hey, TJ is our novel next generation therapy, consisting of 80 be 200 or simple couldn't possibly alpha an investigational human recombinant G.A. enzyme designed to target muscle cells throughout the body.

Administered.

The IB infusions combined with 80 20 to 21 and orally administered enzyme stabilizer.

80, 20 to 21 is administered shortly before the infusion that ATP 200 begins and is intended to bind and stabilize ATP 200 in circulation, allowing more active enzyme to be taken up into cells and delivered to license items.

The combination of E. R. T. An enzyme stabilizer is one major distinction from the standard of care and other treatments in development for pumping.

Other and we believe potentially more impactful distinction is the unique carbohydrate profile of the enzyme itself.

Dr. hung go our Chief Science Officer, and his team had been working over the past decade to develop a pump eight year tea, which improved binding to target receptors for fishing uptake into cells, while importantly, retaining the ability of the your team to be processed by themselves after taken up to the mature more active.

Jeffrey P. Castelli: Dr. Hung Do, our chief science officer, and his team have been working over the past decade to develop a Pompe ERT that improves binding to the target receptors for efficient uptake into cells, while importantly retaining the ability of the ERT to be processed by those cells after it's taken up into the mature, more active form of GA.

Form of GA.

With that in mind and now turning to slide 11, he would like to highlight recent publication in the molecular therapy methods in clinical development Journal for Dr. Neenah Raven team at the National Institute of Health publish findings from an independent preclinical study building upon previous work with 80 GA.

Jeffrey P. Castelli: With that in mind, and now turning to slide 11.

Jeffrey P. Castelli: We would like to highlight a recent publication in the Molecular Therapy Methods and Clinical Development Journal, where Dr. Nina Raven's team at the National Institute of Health published findings from an independent preclinical study building upon previous work with ATJA that showed additional improvements with longer-term treatment in knockout mice. The study showed that ATGA was successful in completely reversing excess glycogen assimilation.

It showed additional improvements but longer term treatment knockout mice.

The study show for the aging GA was successful in completely reversing excess quite a good glycogen simulation restoring muscle strength and significantly improving cascade of secondary abnormality, including the elimination or reduction of audit project built in muscle fibers improvement.

Jeffrey P. Castelli: Restoring Muscles.

Jeffrey P. Castelli: and significantly improving a cascade of secondary abnormalities, including the elimination or reduction of autophagic buildup in muscle fibers, improvements in markers of lysosomal damage, and in self-signaling. These are particularly exciting findings as these impacts had not been observed in previously disclosed studies. Finally, on slide 12, we want to remind everyone of the integrity of our POMPEI Phase 3 PROPEL clinical trial.

Markers of lenses are more damage and in self said million.

These are particularly exciting finding the disease impacts had not been observed in previously.

Disclosed studies.

Finally on slide 12.

He wants to remind everyone of the integrity of our pump safety three propel clinical trial.

We really need reiterate that he timeline remain on track with data expected in the first half of 22 of the first half of 2021.

Jeffrey P. Castelli: We reiterate that study timelines remain on track with data expected in the first half of 2021. To date, more than 97% of the 2,800-plus planned infusions and all study assessments for the ongoing PROPEL study have been completed on schedule. Additionally, we continue to enroll patients in our pediatric studies and advance manufacturing to support a 2021 BLA and MAA. We want to express thanks to all our POMPEI clinical study participants and their families, to all our investigators and their staff, and to our cross-functional Amicus Pompeii team for their collective, unwavering commitment and supportive efforts and for Unprecedented Time. Moving on now to slides 14 and 15.

To date more than 97% of the 2800, plus planed infusion and all study assessment for the ongoing propel studies have been completed on schedule.

Additionally, we continue to enroll patients in our pediatric studies and advanced manufacturing to support the 2021 B.L.A. and anyway.

You want to express thanks.

All our pump a clinical study participants and their families to all our investigators and their staff.

And who are cross functional and the good pump a team for their collective unwavering commitment and supportive efforts for.

I mean this unprecedented time.

Moving on now to slide 14, and 15 I'll briefly highlight here our industry, leading portfolio of gene therapies for rare diseases.

Jeffrey P. Castelli: I'll briefly highlight here our industry-leading portfolio of gene therapies for rare diseases. During this time of COVID, we've been able to maintain our critical science and lead programs across the gene therapy portfolio, including CLN6 and CLN7.

During this time of Kogut, we've been able to maintain our critical science.

And we programs across the gene therapy portfolio, including selling six and seal and three batten disease as well the Palm Bay in Fabry Gene therapy program with the University of Pennsylvania.

Starting with our batten disease franchise and steel in six we previously reported positive interim data and our clinical study that demonstrate meaningful impact of our 80 gene therapy in this extremely devastating form of batten disease.

Jeffrey P. Castelli: Patent Disease, as well as the Pompeii and Fabry gene therapy programs with the University of Pennsylvania. Starting with our BATN disease franchise in CLN6, we previously reported positive interim data in our clinical study that demonstrates the meaningful impact of our AAV gene therapy in this extremely devastating form of BATN disease.

In October at the virtual child Neurology Society annual meeting, we're expecting additional data from the C. On six phase one two study.

We continue to advance regulatory discussions to finalize the clinical and regulatory path and expect to provide an update in early 2021.

Jeffrey P. Castelli: Good night.

Jeffrey P. Castelli: At the Virtual Child Neurology Society Annual Meeting, we're expecting additional data from the CLN6 Phase 1-2 study. We continue to advance regulatory discussions to finalize the clinical and regulatory path and expect to provide an update in early 2021. We believe that the initial CLN6 data provide important research for our clinical study in CLN3 patent disease, which is the most common form of child

We believe this initial seal and six.

Data provide important reason for clinical study and see on three batten disease, which is the most common form of childhood nerve regeneration.

We plan to report initial data from the ongoing phase one to see on free study early next year based on scheduling changes for some of the major gene therapy conferences concurrent with the data early next year, we will provide the expected regulatory path for the key on three program.

We continue to make great progress on all of our commercial manufacturing process.

Jeffrey P. Castelli: The Most Common Form of Childhood

For both sealants Exensio, one three and remain on track to begin dosing additional patients in both of these programs next year.

Jeffrey P. Castelli: I plan to report initial data from the ongoing Phase 1, 2, CLN3 study early next year, based on scheduling changes for some of the major gene therapy concepts. Concurrent with the data, early next year, we will provide the expected regulatory path for the CLM3 program. We continue to make great progress on all of our commercial programs.

Moving on to slide 16, I would like to remind everyone of the research collaboration with the University of Pennsylvania, which will be an important driver of growth for and if it's in the future. This collaboration with the Wilson lab, and Penn combined and the consumer protein engineering and glycol biology expertise.

With pens gene transfer technology and expertise to develop novel gene therapies that are designed for optimal similar uptake targeting dosing safety and Manufacturability.

Jeffrey P. Castelli: For both CLN-6 and CLN-3.

Jeffrey P. Castelli: of these programs next year. Moving on to slide 16, I would like to remind everyone of our research collaboration with the University of Pennsylvania.

As part of this collaboration and it gets has right to over 50 different diseases. In addition to the active ongoing preclinical programs.

Jeffrey P. Castelli: be an important driver of growth for Amicus in the future. This collaboration with the Wilson Lab at Penn combines Amicus's protein engineering and glycobiology expertise with Penn's gene transfer technologies and expertise to develop novel gene therapies that are designed for optimal cellular uptake, targeting, dosing, safety, and manufacturability. As part of this collaboration, Amicus has rights to over 50 different diseases in addition to the active, ongoing preclinical program. Several Amicus presentations were given at the American Society of Gene and Cell Therapy Annual Meeting back in the second quarter.

Several and good presentations were given at the American Society of gene and cell therapy meeting back in the second quarter.

This data was highlighted by our pumping gene therapy results would show the Energous engineered G protein had improved targeting and clearance of glycogen storage pumped into the bias.

Additionally, preliminary data and non human primate suggested therapeutically relevant expression levels and target, Oregon, which is a key factor as you move to larger speech.

We remain very encouraged by the state. It continued to progress are pumping gene therapy towards an ion beam.

We also continued to make progress across our preclinical gene therapy program and look to disclose up to two additional Andy candidates later this year.

With that I would like now to turn the call over to Daphne to review our financial results guidance outlook I think.

Jeffrey P. Castelli: This data was highlighted by our Pompe gene therapy results, which show the Amicus-engineered GAA protein had improved targeting and clearance of glycogen storage in Pompe disease mice. Additionally, preliminary data in non-human primates suggested therapeutically relevant expression levels in target organs, which is a key factor as we move to larger species.

Thank you Jack and good morning, everyone.

Our financial overview begins on slide 18, with our income statement for the quarter ended June 30, 2020, <unk> second quarter, we achieved Galafold revenue 62.4 million, which is a 41% increase over the second quarter of 2019. This includes year over year operational revenue growth measured at.

Jeffrey P. Castelli: We remain very encouraged by...

Daphne E. Quimi: and continue to progress our Pompe gene therapy towards an IND. We also continue to make progress across our preclinical gene therapy programs and look to disclose up to two additional IND candidates later this year. With that, I would like now to turn the call over to Daphne to review our financial results, guidance, and outlook.

Constant currency exchange rate of 43% offset by negative currency impact of 2%.

Cost of goods, so as a percentage of net sales was 10.8% in the second quarter as compared to 12.1% for the prior year period.

Cost of goods sold as a percentage revenue was favorable as Galafold revenue continues to grow in the United States, where we do not old royalties as well as in other countries, where we are subject to lower royalties.

Daphne E. Quimi: Thank you, Jeff, and good morning, everyone. Our financial overview begins on slide 18 with our income statement for the quarter ended June 30, 2020. For the second quarter, we achieved Galliford revenue of $62.4 million, which is a 41% increase over the second quarter of 2019. This includes year-over-year operational revenue growth measured at constant currency exchange rates of 43%, offset by a negative currency impact of 2%. Cost of goods sold as a percentage of net sales was 10.8% in the second quarter as compared to 12.1% for the prior year period. Cost of goods sold as a percent of revenue was favorable as gallifold revenue continues to grow in the United States, where we do not owe royalties, as well as in other countries where we are subject to lower royalties.

Total operating expenses were 170 million in the second quarter, 2020, which decreased as compared to 115.2 million in the second quarter 2019.

On a non-GAAP basis total operating expenses were 95.9 million in the second quarter of 2020 as compared to 103.6 million in the second quarter of 2019.

The decrease in retreat research and development costs reflects increased travel and third party color.

Got it by continued investments to support to propel study and then in our gene therapy pipeline.

All right you got plenty research and development includes the impact as the implementation of the cost reduction measures that were previously it now.

Daphne E. Quimi: Total operating expenses were $107 million in the second quarter of 2020, which decreased as compared to $115.2 million in the second quarter of 2019. On a non-GAAP basis, total operating expenses were $95.9 million in the second quarter of 2020, as compared to $103.6 million in the second quarter of 2019. The decrease in research and development costs reflects decreased travel and third-party costs, offset by continued investments to support the PROPEL study and in our gene therapy pipeline. Our investment in research and development includes the impact of the implementation of the cost reduction measures that were previously announced, as well as the decrease in selling, general, and administrative expenses. Due to the timing of expenses, the non-GAAP operating expense is expected to increase in the third quarter of 2020 as compared to the second quarter of 2020. We define non-GAAP operating expense as research and development and SG&A expenses, excluding share-based compensation, changes in fair value of contingent consideration, and depreciation. The net loss for the second quarter was $52.5 million, or $0.20 per share, as compared to a net loss of $84.6 million, or $0.36 per share, for the prior year period.

Does the decrease in selling general and administrative expenses.

Due to the timing of expenses non-GAAP operating expense is expected to increase in the third quarter of 2020 as compared to the second quarter of 2021.

We did fine non-GAAP operating expenses as research and development and S. T. In any expenses, excluding share based compensation changes in fair value of contingent consideration and depreciation.

Net loss for the second quarter was 52.5 million for 20 cents per share as compared to net loss at 84.6 million or 36 cents per share for the prior year period.

As of June 30, 2020, we had approximately 257 million shares outstanding.

Turning now to slide 19, as John mentioned, following our 400 million dollar senior secured term loan facility. We have now on a clear path to profitability without the need for any future given your financing.

And we initially laid out last year at our analyst day, we have achieved this milestone by our continued revenue growth was cancelled.

What was driving efficiencies cost savings and careful expense management.

Security mid financing with market setting tenants gives us a strong financial platform to advance both patient advocates shareholder interest.

Going forward again to emphasize we expect total non-GAAP operating expenses in 2020 to remain relatively flat. Some 2019 as we further leverage the commercial infrastructure that is already in place <unk>, TJ and Ross and other products and our pipeline.

Daphne E. Quimi: As of June 30, 2020, we had approximately 257 million shares outstanding. Turning now to slide 19, as John mentioned, following our $400 million Senior Secured Term Loan Facility, we are now on a clear path to profitability without the need for any future dilutive financing. As we initially laid out last year at our Analyst Day, we have achieved this milestone through our continued revenue growth with Gallup polls, as well as driving efficiencies, cost savings, and careful expense management. Securing the financing with market-setting terms gives us a strong financial platform to advance both patient and amicus shareholder interests. Going forward, again to emphasize, we expect total non-GAAP operating expenses in 2020 to remain relatively flat from 2019 as we first leverage the commercial infrastructure that is already in place for the ATGAA launch and other products in our pipeline. Secondly, we transition the costs associated with the development of ATGAA to multiple gene therapy programs in our pipeline, and third, maintain financial discipline while meeting our objectives. To reiterate, all high-priority research programs in gene therapy are moving ahead on schedule, especially CLN3, CLN6, Pompeii, and Fabry.

Secondly, we transition costs associated with the development H.T.V. and any multiple gene therapy programs and our pipeline.

Third maintain financial discipline, while meeting our objectives.

To reiterate all high priority research program in gene therapy, or moving ahead on schedule.

Actually in Seattle, and three feel I'd say.

Okay and fabric and we continue to fully supports to work with Jim Okay and Pat.

A few comments about our cash position and 20 plenty financial guidance.

Cash cash equivalents and marketable securities for 309.6 million at June 30, 2020, compared to 452.7 million at December 31 2019.

Following the receipt of net proceeds from the July that facility.

Retiring the prior term loan at 150 million advocates has a cash position over 530 million as of July 31st.

We are reaffirming our full year Galafold revenue guidance of 250 million to 260 million. In addition to our non-GAAP operating guidance of 400 intend to 420 million.

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

Great. Thank you that Daphne, Jeff and Bradley Phil as you can see we have been relentlessly focused on performance across the business. Despite all of the unprecedented change and challenges that had been brought about by the local pandemic. We have a great global team are passionate entrepreneur.

Daphne E. Quimi: And we continue to fully support the work with Jim Wilton and Penn. Here are a few comments about our cash position and 2020 financial guidance. Cash, Cash Equivalents, and Marketable Securities were $309.6 million at June 30, 2020, compared to $452.7 million at December 31, 2019. Following the receipt of net proceeds from the July Debt Facility and retiring the prior term loan of $150 million, Amicus had a cash position of over $530 million as of July 31. We are reaffirming our full-year Gallup Gold revenue guidance of $250 million to $260 million in addition to our non-GAAP operating expense guidance of $410 to $420 million. And with that, I will turn the call back to John for closing remarks.

Or is it abacus, who have led and we'll continue to lead us through this I'm confident that as the world emerges from this crisis Energous will emerge even stronger so with that operator, we're happy to take any questions.

Ladies and relevant if you have question.

Piece first part and into number one.

And your Touchstone telephone at this time, yes, Q. That's you only ask ones question. If you have any additional questions be center back into the queue for your question has been answered for you wish to remove yourself from the Q piece person Duncan.

And your first question Punch line, that's I know from Roma from JP Morgan.

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

For lines Hilton.

Hey, guys. Thanks, so much for taking my question just a quick question on the propel study.

No small number of patients who did not yet and on timing fusion.

Scheduling infusion unique patient ultimately getting using outside of the window or was it just to skip.

And then they got them excellent. Thanks, so much.

Sure, Jeff maybe I'll ask you to field that question again on apologists state that even in preparing for this study as with most clinical studies in the statistical analysis plan, we expected.

Operator: Ladies and gentlemen, if you have questions, please press star and then the number 1 on your touchtone telephone. At this time, we ask you only to ask one question. If you have any additional questions, please enter back into the queue. If your question has been answered, or you wish to remove yourself from the queue, please press the down arrow. And your first question, contraline, is Anupam Rama from J.P. Morgan. Her line is now open. Hey guys, thanks so much for taking the question. Just a quick question on the Propel study: for those small number of patients who did not get an on-time infusion, scheduled infusion, did these patients ultimately get an infusion?

You know more than 3% that infusions would be missed that just kind of commented and he said he certainly in rare disease studies, so even with Covidien 19, we're performing better than we expected from an infusion and an assessment standpoint, but maybe can you give speak to.

Be missed in the very very few missed infusions.

Sure. Thanks, Jonathan Thanks.

You know that that 3% number could refer to either entirely missed infusion or it could be an infusion that was slightly out of every two week window.

John Crowley: Sure, Jeff, maybe I'll ask you to field that question. Again, Anupam, I'll just state that even in

But importantly, as part of the initial study design, we had allowed for individual study subjects to have some small number of missed infusions and just still remain in the primary now season in the study and everything we've seen since within what we have even better than what we had planned pre cobot for potentially having patients aneurysm.

Operator: [inaudible]

Jeffrey P. Castelli: Sure, thanks John and thanks Anupam.

Jeffrey P. Castelli: So that 3% number could refer to either entirely missed infusions, or it could be an infusion that was slightly out of the every two weeks window. But importantly, as part of the initial study design, we had allowed for individual study subjects to have some small number of missed infusions and to still remain in the primary analyses and in the study. And everything we've seen, you know, fits within what we had planned pre-COVID for potentially having patients miss an infusion.

Fusion.

That's actually a remarkable on upon that even through the height of the early days of the crisis in March and April we had so very few missed infusions that I think it was a real testament to patient motivation desire to participate in this study.

John Crowley: It's actually remarkable, Anupam, that even through the height of the early days of the crisis in March and April, we still had very few missed infusions. I think that was a real testament to patient motivation, desire to participate in the study, desire to access these medicines, investigator efforts, and just some remarkable patient outcomes.

Our desired accessories medicine. This medicine investigator effort can just some remarkable efforts by our team again, if you remember back then.

Really came up with a patient by patient site by site plan to maintain the integrity of that study. So we're really pleased where we are today.

Your next question comes from the line are free to borrow from Holland Airlines now open.

Operator: https://www.kenhub.com

Operator: Again, if you remember back then, we really came up with a patient-by-patient, site-by-site plan to maintain the integrity of that study. So we're really pleased where we are today.

Good morning, guys. Thanks for taking the question I'll take the the flip side to honor Palms question asked about the assessments specific lean six minute walk [laughter] final time point necessary for the primary endpoint can you talk about how many of those specifically have been completed.

John Crowley: Your next question comes from the line of Ritu Baral from Holland. Your line is now open. Good morning, guys.

John Crowley: Thanks for taking the question. I'll take the flip side to Anupam's question and ask about the assessment, specifically the six minute walk at the final time point necessary for the primary end point. Can you talk about how many of those specifically have been completed? You know, how many of them might have been out of spec and the timing of the number and timing of those that are planned for Q3. Since we don't know how COVID's going to go. I'd love to know how many of them are, so to speak, in danger.

You know how how many of them might have been out back and the timing of.

The number and timing of those that are planned for Q3 since we don't know how cold it is going to go.

I'd love to know how many of them are so to speak in danger.

Yeah, we probably can't provide that level of granularity to in terms of who is due for which the assessments on which date or which quarter, but I'll, just and I'll ask Jeff to comment here virtually all of the six minute walk test test assessments were done and completed on.

John Crowley: Yeah, we probably can't provide that level of granularity, Ritu, in terms of who's due for which assessment on which date or in which quarter, but I'll just, and I'll ask Jeff to comment here. Virtually all of the six-minute walk test assessments were done and completed on time within the window, particularly those, again, those 12-month assessments. You know, we were in the March and April timeframe where that was a real concern. We went to extraordinary lengths to make sure that those assessments took place. So, very, very few, I think maybe one or two patients may have fallen out of that window. So, it should have no impact on the study, and again, as expected in a study like this.

Time within that window, particularly those again those 12 months assessments you know it was we were in the March and April timeframe, where that wasn't real concerned we went into the extraordinary linked to make sure that those assessments occurred. So very very few I think you know maybe it was warner to patient.

They have fallen out of that window. So it should have no impact on study and again as expected in a study like this is Jeff at any color get like please.

Yeah, John you covered it very well.

I'll just remind everyone.

We plan to study into statistics as you do with any clinical trial, you assume some amount of.

Jeffrey P. Castelli: Yeah, John, you covered it very well. I'll just remind everyone that, you know, as we plan the study and the statistics, as you do with any clinical trial, you assume some amount of missing data due to dropouts, missed, you know, visits. And we are well within what we had planned even before we over-enrolled the trial from 100 to 123. And as John said, you know, that 97% plus refers not only to infusions but also to scheduled study assessments. And in particular, the six minute walk is the primary.

Missing data due to dropouts missed you know visits and we are well within what we had planned even before we over all the trial from 100 123, and as John said, you know that 97% plus implies not only to infusion, but also just scheduled study assessments and in particularly six minute walk as the primary end.

Point and that month well.

Everyone is really pitch and you've got to make sure those are going on track. So so we're in a great place in terms of available data in patients for the final propel analysis.

Jeffrey P. Castelli: , and everyone is really pitching in to make sure those are going off on track. So we are in a great place in terms of available dates and patience for the final.

Yeah Importantly, Richard we've had virtually no dropouts in this study.

And again you typically in the statistical plan, we will plan for up to 10% to study participants are more to drop out so that will certainly help us a great deal as well and also gives us a great margin for it goes or acute patients.

Jeffrey P. Castelli: and all.

John Crowley: Propel Analysis

John Crowley: Yeah, importantly, Ritu, we've had virtually no dropouts in the study. And again, you typically in the statistical plan will plan for up to 10% of study participants or more to drop out. So that will certainly help us a great deal as well, and also gives us a great margin for those very few patients who may have missed an assessment.

They missed assessment.

Your next question Punch line of Debjit Chattopadhyay young from here.

In North Carolina <unk>, Okay.

Hey, good morning, and thanks for taking my question. So as you think through the gene therapy program for a bumpy any thoughts on the relative contribution so the becomes XP glycosylation versus bad stuff stabilization with me, let's stop and.

Operator: And your next question, call to line is Debbie Shadow-Podjai from H.C. Wainwright. Your line is now...

Would you consider keeping patients as amended gene therapy program goes in the clinic on a sort of a stable background dose on my list out based on the propel experience. Thank you.

Operator: Salveen Richter, Eliana Merle, Hey, good morning!

John Crowley: and thank you for taking my question. So as you think through the gene therapy program for Pompeii, any thoughts on the relative contributions of bisM6B glycosylation?

Yeah debt just so we looked very carefully and did some pretty exhausted studies pre clinically to look at the relative contribution are the new enzyme that we had developed with the enhanced like constellation and you recruit targeting with the high levels and then a six phosphate together.

John Crowley: versus stabilization with megalostat. And would you?

John Crowley: Consider keeping patients as and when the gene therapy program goes into the clinic on a sort of stable background dose of megalostat based on the Propel experience. Thank you.

With a shop around and again remember the mechanism here is that the improved quite constellation and a high levels of benefits phosphate our whats necessary for the enzyme to move from the blood to move from the plasma targets those receptors on muscle and to be internalizing absorbing all key muscle tissues.

John Crowley: Yeah, Devjit, so we looked very carefully and did some pretty exhaustive studies pre-clinically to look at the relative contribution of the new enzyme that we had developed with enhanced glycosylation and improved targeting with high levels of mannose 6-phosphate together with the chaperone. And again, remember the mechanism here is that the improved glycosylation and the high levels of mannose 6-phosphate are what's necessary for the enzyme to move from the blood, move from the plasmid, target those receptors on muscle, and then be internalized and absorbed into all key muscle tissues. So that would be skeletal, that would be diaphragmatic, and in patients with cardiac involvement, cardiac absorption as well.

So that would be skeletal that would be tire dramatic that would be in patients with cardiac.

Involved in cardiac absorption as well and we think that the great majority of the benefit 80, 90% I think is but hung is characterized.

The benefit is due to the enhanced nature of the absorption of the enzyme. It's the classical biology, we do think the chaperone plays an important function and in all of our preclinical studies we saw.

John Crowley: And we think that the great majority of the benefit, 80-90%, I think is what Hong has characterized of the benefit, is due to the enhanced nature of the absorption of the enzyme. It's the glycobiology. We do think the chaperone plays an important function, and in all of our pre-clinical studies, we saw Enhanced Breakdown of Glycogen. So we think the function of the chaperone is to enhance the stability of the protein in plasma, potentially to make it better tolerated, but also, when it does deliver it to the lysosome, it delivers it in a more active form, which again contributes to even greater glycogen breakdown. So again, that's part of the novelty of the combination therapy here. But again, we think the great benefit for patients, the preponderance of the benefit, is in the glycosylation of the enzyme.

Enhanced breakdown of glycogen. So we think the function of the chaperone is to enhance the stability of the protein and plasma potentially to make it better tolerated, but also when it does deliver it to the lysosome delivers it in a more active form which again contributes to either.

In greater like it shouldn't breakdown. So again, that's part of that novelty of the combination therapy here, but again, we think the great benefit for patients at the preponderance of the benefit is in the clay constellation of the enzyme.

Hi, This is Jeff I'll, just add basically to that is as you would fly that towards gene therapy, which I think the question also might have been asking about similarly say TJ. We think you know majority of the benefit there is the engineered transgene with well targeted GA.

Jeffrey P. Castelli: This is Jeff. I'll just add.

Jeffrey P. Castelli: Similarly to ATGAA, we think the majority of the benefit there is the engineered transgene with the well-targeted GAA. A difference with gene therapy versus enzyme replacement therapy is that rather than only giving a dose of enzyme every two weeks, with gene therapy, the enzyme is sort of constantly secreted out from the transduced cells into the blood. So it's not quite as simple to think how a chaperone might help with the gene therapy. So right now, our vision for the Pompe gene therapy is just as a gene therapy, not combined with a stabilizer. But there is some theoretical potential where you could look to use a stabilizer, but right now, that's not our plan.

A difference, but gene therapy versus the enzyme replacement therapy is that rather than only giving a dose of enzyme every two weeks, but gene therapy. The enzyme is sort of constantly secreted out from the trends do cells in the blood. So it's not quite simple to think how is chaperone might help with the gene therapy. So right now are.

Vision for the pumping gene therapy is just as a gene therapy, not combined with our stabilizer, but there is some theoretical potential way you could look to you the stabilizer, but right now that's not our plan.

Got you also could in some diseases that we're looking at worthy enzymes are even more inherently a stable and plasma.

John Crowley: Tedra, you also could, in some diseases that we're looking at where the enzymes are even more inherently unstable in plasma, you could potentially engineer stability into those transgenes, and that's one aspect of the technology that we're exploring for some other disorders.

You could potentially engineer stability into those trends genes and that's one aspect of the technology that we're exploring for some other disorders.

Your next question comes along with smoking Tonsil from Citigroup. Your line is now open.

Operator: In your next question, the control line is Mohit Ghansal from SIPI Group. Your line is now open.

John Crowley: Great, thanks for taking my question and congrats on all the progress despite the pandemic here. Maybe one question on Sanofi's side, because now that we have data from the Neo-GAA program, one thing, at least in my mind, is getting clear is how inadequate the existing treatments are for Pompe disease, especially lumizine, which was the control here. So with that in mind, do you have any updated thoughts about the control arm for propel and the powering assumptions here? Because, if I'm not mistaken, you're assuming control to be relatively stable here. Is that fair for treatment experience?

Great. Thanks for taking my question and congrats on all the Providence, Despite bend that Mccann.

[noise] VB, one question of and Sanofi side because.

Now that we have data from the Neo G April.

One thing keys in my mind is getting to yet is that how indicted UK. The existing treatments are sort of on BDC specially lumizyme, which was to controlling here. So that you might do you have any updated thoughts.

About the control arm for propel study and the following resumption sets because if I'm not mystic and that assuming continued to be relatively stable here.

Is that fair for the FID for Piedmont expedient species at this point.

John Crowley: Thank you, Mohit. I'm not going to comment on any of the NEO data. I think that the data will speak for itself. You did reference the lumizyme control arm. That was a surprising finding in that study, that at the 12-month endpoint, the control arm, the lumizyme, again, in treatment-naive patients, showed, if I recall, just maybe it was a 2-meter improvement, which is less than has been seen in any previous studies. The approval study, the late-onset study, or LOP study, showed, I think it was a 20- or 25-meter improvement. That's what we built into our powering assumptions for the cohort of patients, the treatment-naive patients in our study. We assumed lumizyme would perform much better than it did in that comet study, and that's what we built into our stats. If it does worse, of course, that will only enhance our ability to show a distinction with ATGAA and Jeff. Add any color or comments.

Thank you Mohit I I'm not going to comment on any of that neo data I think that data will speak for itself.

You did reference to the Lumizyme control arm that was a surprising finding in that study that at the 12 month end point the control arm. The lumizyme again in treatment naive patients showed day, if I recall, just maybe was it two meter improvement.

Which is less than has been seen in any previous studies. The approval study. The laid off that study were locked studies showed I think it was a 20 or 25 meter improvement that's what we built into our powering assumptions for the cohort of patients the treatment naive patients in our study.

We assumed bloomed design would perform much better than it did in my comments study.

So and that's what's built into our staff if it does worst of course that will only enhance.

Enhance our ability to show the distinction with 80 GA.

And just add any color on.

John Crowley: No, John, you covered that perfectly.

Huh.

No John you covered up perfectly.

And your next question comes from the line of selling Merlin from Cantor Fitzgerald. Your line is now open.

Operator: And your next question comes from the line of Ellie Merle from Cantor Fitzgerald. Your line is now open. Hey guys, thanks so much for taking the question and congratulations on all the progress. Okay.

Hi, Thanks, so much for taking the question and congrats.

Oh, Hey.

John Crowley: Just a little on Fabry disease, just in terms of, I know you mentioned for Galliford, you're seeing sort of, you know, improvements in the diagnosis rate and treatment rate. I'm curious, you know, about the current diagnosis and treatment rate that you're seeing broadly in your addressable markets as well as just based on the recent trends you're seeing, what sort of, you know, a realistic assumption to think about for long-term penetration in this market given sort of the, sort of, pretty, you know, underdiagnosed and undertreated currently right now. So how do we think about kind of peak penetrance and, you know, in light of potential gene therapy competitors, how do you see this disease landscape evolving? Thanks.

Yes.

And just in terms of I know you mention for Galafold, you're seeing sort of you know incrementing the diagnosis rate and treatment rates I'm curious you know I guess, but the current diagnosis and treatment that you're seeing broadly in your Oh, My gosh polished just based on the recent trends you're seeing what sort of you know a realistic assumption to think about for long term penetration.

Ms market given sort of that's.

<unk>, you know under diagnosed and under she had.

Now currently right now so how we think about kind of peak penetrance and in light of potential gene therapy competitors, how you see that disease landscape evolving. Thanks.

Yeah again, it remains I think a great opportunity to help a lot of people living with fabry disease as more and more experience is being had with Galafold and importantly, more and more patients are being identified we continue to believe that fabry is one of the most if not the most under or misdiagnosed.

John Crowley: Yeah again, it remains, I think, a great opportunity to help a lot of people living with febrile disease as more and more experience is being had with Galifold and, importantly, more and more patients are being identified. We continue to believe that febrile disease is one of the most, if not the most under or misdiagnosed human genetic diseases in existence.

The genetic diseases.

In existence, so Bradley I'll, let you comment specifically about growth potential here and what we're planning for.

Yeah. Thanks for the question I think they're sort of two pieces of your question first is.

John Crowley: So Bradley, I'll let you comment specifically about the growth potential here and and what we're planning for.

Penetration of treatment into the diagnose population in the second is.

Percentage of potential fabry patients, who are diagnosed I'll I'll take the first one first I think it's a little bit easier. There I think we will see high penetration of treatment into diagnosed population I think as we've been able to introduce a small molecule into the space you know we've been able to grow.

Bradley L. Campbell: Yeah, Ellie, thanks for the question. I think there's sort of two parts to your question. First is the penetration of treatment into the diagnosed population. And then the second is the percentage of, you know, potential Fabry patients who are diagnosed. I'll take the first one first.

The treated market, even since we've been on the Mark and I think the end of last year, we shared that of the thousand plus patients on Galafold 300 of them were diagnosed but previously untreated and I think you see that analogy play out in other markets as well if you look at the M.S. space with the introduction of texture, there or the other small mom.

Bradley L. Campbell: I think it's a little bit easier there, and I think we'll see high penetration of treatment into the diagnosed population. I think as we've been able to introduce a small molecule into the space, you know, we've been able to grow the treated market even since we've been on the market. I think at the end of last year, we shared that of the thousand-plus patients on Galafil, 300 of them were diagnosed but previously untreated. And I think you see that analogy play out in other markets as well. If you look at the MS space with the introduction of Texedera, and other small molecules, even if you look further back and at PAH with the introduction of the Embracentin class of molecules, you see significant growth in treated patients.

Fuels, even if you look further back in at ph.

With the introduction of the 10% 10 class of molecules.

You see significant growth of treated patients.

Of the diagnosed population.

Separately I mean, we think we'll see that galafold as well.

Separate from that.

You asked the question of how much you know what percentage of potential fabry patients will be diagnose we know that number is growing.

Seeing no ourselves other manufacturers in the space.

Bradley L. Campbell: of the diagnosed population, separate, and we think we'll see that in Gallup as well. Separate from that, if you ask the question of what percentage of potential Fabre patients will be diagnosed, we know that number is growing. We've seen it ourselves, other manufacturers in the space.

Spend a lot of time, both looking at newborn screening, but also looking at high risk population screening and we've talked a lot about that before in.

Kidney populations in cardiac populations.

In M.S. and pain clinics. So we see that a lot of efforts are going into that that being said if you know if you look at newborn screening and you see that this is a you know potentially one in 1500 to one in 3000 wide berth incidents.

Bradley L. Campbell: I would suspect that, unfortunately, it will continue to be an underdiagnosed disease for years to come. You know, that could imply a global incidence of 100,000 CABRE patients. So I think we would do a great job to continue to chip away at that diagnosis rate. But I think you'll, you know, that would be a journey that all of us go on over the next decade or so.

I would suspect that unfortunately, and we'll continue to be under diagnosed disease for years to come you know that that could back could imply a global incidents of 100000 fabry patients. So I think we would do a great job to continue to chip away at that diagnosis rate.

But I think deal.

It would be a journey that all of US go on over the next decade or so.

Operator: And your next question comes from the line of Mike Oltz from Baird. Your line is now open.

And your next question personal line. This Michaels from Baird. Your line is now open.

Operator: Hey guys, thanks for taking the question. Good morning, Mike. Good morning.

Hey, guys. Thanks for taking the question.

Morning, like quick morning, just a quick question on H.P.G.A. and you're you're rolling deal. A are you still on track to start that in the second half of this year I'm wondering if you can give us the sense of what you plan to file this year I'm just curious if you think.

John Crowley: Just a quick question on ATGAA and your rolling BLA. You're still on track to start that in the second half of this year. I'm wondering if you can give us a sense of what you plan to file this year. And I'm just curious if you think that you'll be in a position to kind of have the majority of the BLA filed prior to Phase 3. Thanks.

You'll be in a position to kind of have the majority of the BLE filed.

Prior to the phase three.

Thanks.

John Crowley: Yeah, as you know, there are really three key sections to the Biologic License Application, or BLA. The first is the preclinical module, second is the CMC module, and third will be the full clinical. We expect that the full preclinical, including all the toxicology work that has been completed, will form the foundation for the first part of the filing this year. We then expect the second filing for the CMC section. And again, we've completed all of the PPQ runs successfully, so a significant part of the work, and frankly, a significant part of the risk, has been taken out of that. So we feel really good about where manufacturing is, and our partners at WUSHI Biologics, with our tech ops and quality teams, have really just done a remarkable job at the commercial scale up here.

Yeah. As you know there were really three key sections to the biologic license application or be a lay the first is the preclinical module second is the CNC module and third will be the full clinical.

We expect full preclinical, including all the toxicology work.

Completed that will form the foundation for the first part of filing.

This year, we expect then the second filing for the CMC section and again, we've completed all of the Bbq runs successfully so a significant part of that work and frankly, a significant part of the risk has been taken out of that so we feel really good about where manufacturing is then.

Our partners that we see biologics with our tech ops quality teams that really just on a remarkable job at the commercial scale up here. So we expect that then to fill we ended the first half of next year and on the last section to go in wholesale in the first half of next year would be the full clinical so.

John Crowley: So we expect that then to go in during the first half of next year, and then the last section to go in also during the first half of next year would be the full clinical trial. Hopefully, that answers your question, Mike, so we'll be in really good shape with that rolling BLA beginning this year and completed in the first half, the full BLA in 2021, followed very quickly thereafter, we hope, by the MAA filing in Europe.

Hopefully that answers your question, Mike. So, we'll we'll be it really good shape with that rolling B.L.A., beginning this year and and completed in the first half the full be allay in 2021, followed very quickly thereafter, we hope so I'd and the a filing in Europe.

And again, that's your if any question. Please press star and the number one key on your such there and telephone during next question, calling from a line. This can be to Gupta from Guggenheim Securities. Your line is smelting.

Operator: And again, if you have any questions, please press star and then the number one key on your touch-tone telephone. Your next question comes from the line of Andita Gupta from Guggenheim Securities. Your line is now open. Hi guys, congrats on the quarter and hope you all are doing well.

Hi, guys. Congrats on the culture and hope fuel on the doing well my question is on the fee and upon the Bakken programs. What is the extent of the additional data from the she had insects that can be able to see into CNS, saying till there and if you could maybe talk about how many patients worth of data and how long of a follow up does the FDA.

Jeffrey P. Castelli: My question is on the BATN program. What is the extent of the additional data from CLN6 that we will be able to see at the CNSA in October? And could you maybe talk about how many patients' worth of data and how long of a follow-up does the FDA want to see in your discussions for the regulatory path forward for both CLN6 and CLN3? Thank you.

I want to seeing a discussions fidelity make any thoughts on forward in bullet CNN six inch entry I can't.

John Crowley: Yeah, I'll ask Jeff to comment for a moment on the data that we'll see at Child Neurology this fall, and again, we are engaged in discussions both for CLM 6 and CLM 3 with the US FDA. We expect to complete those this year and now very early next year to be able to share that. I'm not going to comment on the ongoing discussions.

Yeah, I'll ask just to comment on a moment on the David will see a child neurology this fall Nvidia.

And again, we are engaged in discussions both for CLM Zixone CLM three with the U.S.F.D.A., we expect to complete the this year and now very early next year, we'll be able to share that.

I'm not going to comment on the ongoing discussions we've always said that we know we want to the need to treat.

Jeffrey P. Castelli: We've always said that we know we want to, but we need to treat additional patients with our commercial-scale material. That material will be available in the first half of 2021, so we expect it will be a relatively small number of patients, and I don't yet have an answer for what the duration of that treatment period would be that would be sufficient then for the BLA filing, but we continue to have great confidence in this program being on a really good path for people living with both CLM 6 and CLM 3 with those two respective gene therapies. Jeff, do you want to comment on the data?

Additional patients with our commercial scale material that material will be available in the first half of 2021. So we expect it will be a relatively small number of patients and I don't yet have an answer for with the duration of that treatment period would be that would be sufficient then whether it be a late filing.

We continue to have great confidence in this program.

Being on a really good path for people living with both sealant, six and sealant three but those two respective gene therapies.

Jeff do you want to comment on that data for sealant six the updated data that we'll see.

Sure. Thanks, John So just as a reminder, last year at the CNS annual meeting we presented data on that first eight patients that had been treated and that was with anywhere from one to two years of follow up. So this year, we'll have a one to two years a follow up data in all 13 treated patients and the majority of that will be.

Jeffrey P. Castelli: So just as a reminder, last year at the CNS Annual Meeting, we presented data on the first eight patients that had been treated, and that was with anywhere from one to two years of follow-up. So this year, we'll have one to two years of follow-up data on all 13 treated patients, and the majority of that will be year two of follow-up. In addition, we'll have more natural history data and comparisons to also put the data from these 13 kids into context as to what would have been expected based on natural history.

Year to follow up.

In addition, we'll have more natural history data and comparisons to also put the data from these 13 kids into context as to what would have been expected based on the natural history.

And your next question parcel on the speaking hall from Viki Aichi. Your line is now open.

Operator: And your next question, call from the line of Dagan Hough from BTIG. Your line is now open. Great.

John Crowley: Thanks very much for taking the questions. Sorry to miss this, John, but in the press release when you talk about the 1,000-plus mutations included in the EU label for Galifold, can you maybe help us understand what percentage of the Fabry patients that covers? I think the previous number was 35 to 50 percent. And concurrently, any updates to the FDA label as it pertains to the number of mutations? And then the second question is, as we look toward your Pompe gene therapy that everyone's looking forward to, I was wondering if you could provide a little bit of context or maybe some insights as to your internal discussions with regard to, say, goals such as, you know, less than 10 E14 vector genomes per KG or trying to avoid AAV8 as a vector outright. Thanks.

Great. Thanks, very much for taking my questions I'm, sorry, I missed this I'm, John but when in the press release when you talk about now a thousand plus mutations included into your label for Galafold.

Can you maybe help us understand what percentage of the fabry patients that encompasses I think the previous number was 35% to 50% and concurrently any updates to the FDA label as it pertains to the a the number of mutations and then the second question is as we look toward your pumping gene therapy that that everyone's looking forward.

Too I was wondering if you can provide a little bit of contacts or maybe some insights as to your internal discussions with regards to say goal such as you know less than 10 knee 14 vector genomes per check or trying to avoid aviate as a vector outright. Thanks.

John Crowley: Sure. All right, Dagan. You squeezed three questions in there, so let me break them apart. On the first part for the mutations, the addition here, I would not expect that this increases the market size or the relative percentage of patients with amenable mutations. It will just help us accelerate treatment for those patients once they are identified.

Sure Alright, big on your screen three questions in there so let me break those apart.

On the first part with a mutation. The addition here I would not expect that this increases the market size, where the relative percentage of patients with amenable mutations. It will just help us accelerate treatment to those patients. Once they are identified so we still believe up to about half.

John Crowley: still believe up to about half of people living with Pompe disease are living with an amenable mutation. So, again, this completes the work necessary to expand that label. Bradley, or Jeff, would you like to comment on, or Jeff, on the FDA when we would expect any label update there with those additional mutations?

People living with pump a disease are living within a minimal mutation. So again this completes the the work necessary to expand that label Bradley do you want to comment on where Jeff on the FDA. When we would expect any label update there with the additional mutations.

Yeah, John the FDA has a slightly different process. So.

Bradley L. Campbell: Yeah, John, the FDA is a slightly different process.

We are.

Bradley L. Campbell: We are, Essentially, as we had been doing in Europe, essentially adding batches of additional mutations that we identify. So, this year, we'll send in a quite large batch of new mutations that have been identified in patients to the FDA, but it's a different regulatory process. So, we'll continue to share that as we have those updates. We are looking to do a similar kind of global update as we did in Europe with some other geographies outside the United States, and that will take place over the course of this year and into next as well. But, as John said, I think the most important thing is these updates really provide facilitated access for patients. So, rather than having to go through the individual characterization of a new mutation and then getting it into the label or onto the website, this kind of batch process really allows that to be much faster for patients.

Essentially as we had been doing a Europe, where essentially adding batches of additional mutations that we identified so.

This year will send in a quite large batch of new mutations that had been identified with patients into the FDA, but the different regulatory process.

So we'll continue to share that as as we have those updates we are looking to do a similar kind of global update as we did in Europe with some other geographies outside the United States and that'll take place over the course of this year and into next as well, but as John said I think the most important thing is these updates really provide facility.

Got it access for patients so rather than having to go through the individual characterization of them you knew mutation and then getting it into the into the label or onto the website. This kind of batch process really allows that to be much faster for patients and physicians.

And then relative to your question around pumping gene therapy. Other continues to progress very well, we've said all along that gene therapy and pay disease is much needed, but it's also an incredibly complicated given the nature of this disease.

John Crowley: And then, relative to your question around Pompe gene therapy, you know, that continues to progress very well. We've said all along that gene therapy in Pompe disease is much needed, but it's also incredibly complicated given the nature of this disease. We built our gene therapy work in Pompe disease around our collaboration with Jim Wilson, and you, Ben, if you remember going back about two years, it was one of the original three programs that formed the foundation of our initial collaboration with Dr. Wilson. One key element of that collaboration was our belief that we needed to not only deliver properly targeted and ultimately effective gene therapies, but we needed to make sure we could do it safely, particularly in a disease like Pompe, given the compromised nature of the patient and the need likely for systemic delivery of an AAV.

We.

Bill our gene therapy work in pump a disease around our collaboration with Jim will fit in EWP and if you remember going back about two years. It was one of the original three programs that form the foundation of our initial collaboration with Dr. Wilson, one key element to that collaboration with our belief that we need it.

Not only deliver properly targeted and ultimately effective gene therapies, we needed to make sure we could do it safely.

Meticulously in it disease like Tom paid to the compromise nature of the patients.

And the need likely for systemic delivery of the Navy.

So we would always been focused on enhancing the targeting true.

John Crowley: So we have always been focused on enhancing the targeting through the novel transgenes that we have, enhancing the targeting of the enzyme that's produced by the gene therapy vector so that we can have a low or as low a dose as possible. So with that, maybe Jeff, do you wanna comment on what we and Jim believe about this? Obviously, there's been a lot of difficult news in this space from others in the gene therapy field that I think only highlight the importance of proper targeting and safety. So Jeff, I'll turn it to you.

Through the novel Transgene that we have enhancing the targeting of the enzyme that produced by the gene therapy vector. So that we can have a low four as low it goes as possible. So with that maybe Jeff do you want to comment around where we and Jim believe around this obviously, there's been a lot of.

Difficult news in this space from others in the gene therapy field that I think only highlight the importance of copper targeting and safety.

So Jeff I'll turn it to you.

Sure Thanks, John and as John noted from the beginning our approach in gene therapy has been to design more potent gene therapies based on either stabilization or better targeting for uptake to keep systemic doses in a range that are as low as possible.

Weve said previously that you know as we went into things based on the guidance from our colleague that and we had an upper ceiling in the range of Fiveg 13 per kilogram based on all the existing data pre clinically that's been reported wells clinically.

Jeffrey P. Castelli: Sure, thanks, John. And as John noted, you know, from the beginning, our approach in gene therapy has been to design more potent gene therapies based on either stabilization or better targeting for uptake to keep systemic doses in a range that is as low as possible. But we remain committed to trying to make more potent gene therapies that we can keep, you know, hopefully systemic dosing in that, you know, 1 to 5E13 per kg range that we think has some established safety.

We still think that that's a likely ceiling I think there will be more and more learnings as people process through the recent data from the all done to study the very high dose systemic gene therapy.

To figure out how you really scale across weight than ages of Kid.

But we remain committed to trying to make more potent gene therapy. So we can keep hopefully systemic dosing in that.

One to 513 for Keurig range that we think has some established safety.

Your next question cultural on the free to borrow from Cowen. Your line is now open.

Operator: Your next question comes from the line of Ritu Baral from Cohen. Your line is now open. Hey guys, thanks for taking the follow up. I wanted to ask, maybe I'm reading too much into it, but in the CMC portion of your presentation, John, did you imply that there are still outstanding CMC activities to be completed before the BLA filing? And also, where are you on additional manufacturing sources outside China? Thanks.

Hi, guys. Thanks for taking the follow up I want to ask maybe I'm reading too much into it but in this season.

Portion of your presentation John.

I did you imply that they're still.

Outstanding.

CMC activities to be completed for the BLE filing and also where are you on additional manufacturing sources outside China. Thanks.

Bradley L. Campbell: Yeah, I'll let you, we're in a really good place on both of these, but I'll let Bradley comment. Brad, do you want to go first? to get those?

Yeah, I'll, let we're really good place on both of these but I'll, let friendly comments I've read you want it.

Bradley L. Campbell: Yeah, sure. On the first one, we had previously said that all the upstream and manufacturing portions of the PPQ sections were complete, and that drug product was released. That was all according to spec and to our analytical compatibility agreements. We do have the drug product PPQ runs. That's the lyophilization process.

You get those.

Yeah sure on the first one we had previously said that the all the upstream.

And manufacturing portion of the PDQ sections were complete and ER Amber drug product was released that was all according to spec and to our analytical comparability agreements, we do have the drug product.

He runs up the wild basin process, that's a much more straightforward process is much faster process that's underway.

Bradley L. Campbell: That's a much more straightforward process. It's a much faster process that should be complete here shortly. That's kind of the last manufacturing step. Of course, you have to pull together the package and submission, and that's all on track and per the protocols that we agreed to with the agencies. And I think John answered a question previously on the rough timing for when we hope to complete that section.

And should be complete here shortly that's kind of wire manufacturing step of course, you have to pull together the packaging condition and that's all.

On track and per the protocols that we agreed to with the agencies.

And I think John the answer to question previously on the rough timing for when we hope to complete that that section.

Bradley L. Campbell: In terms of manufacturing capabilities outside of China, as we discussed previously, Wuxi has invested in a similar manufacturing facility in Dundalk, Ireland, and the construction of that facility is well underway. And the intention is for that facility to be licensed right around the time of our anticipated approval so that the first launch material would come out of the Wuxi facility in Wuxi, China. And then as we get into the first couple of years of commercialization, that would be complemented by the Irish material coming out of the Ireland plant. So you'd very quickly have two sources of manufactured goods. I would remind you, though, that we've also already begun the process of moving product outside of China into our various regional hubs, and then down even to the site level where appropriate. That was done in part to make sure we had product in various regions, but it has also been very important from a continuity of supply perspective for the challenging times of COVID. And that's how we've really been able to maintain that uninterrupted supply chain, even through the worst of COVID. [inaudible]

In terms of the manufacturing capabilities outside of China.

As we discussed previously.

She has invested in a similar manufacturing facility in Dundalk, Ireland, and the manufacturer of that facility as well underway and the intention is for that facility to be license right around the time of our anticipated approval. So that you would the first launch material would come out of the Lucy.

Facility in Wuxi, China, and then as we get into the first couple of years of commercialization that would be complemented by the Irish other material coming out of the Ireland plant. So we'd very quickly have two sources of of manufactured goods I would remind you. Though we have also already began the process of moving.

Product outside of China into our various regional hubs and then down even to the site level, where appropriate that was done in part to to make sure. We had product in various regions. But also has has been very important from a continuity of supply perspective for for the challenging times of cobot and that's how we've really been able to maintain that.

Uninterrupted supply chain, even through the worst of the cobot.

John Crowley: So hopefully, Ritu, thank you, Bradley. Hopefully, that makes it clear that everything remains on the timeline that we've laid out previously. And so far, everything remains on track, both for the activities to support the CMC section of the BLA, as well as the scale-up, and now we're continuing to build commercial inventory so that, hopefully, at launch, we'll be able to provide the medicine to all patients in need. And part of that is the capacity planning to make sure that we have dual sources of manufacture. And again, as Brad noted, that remains on track with the Ireland plant moving forward as well. I was supposed to be in Ireland next week, actually, to visit that plant. Thank you. Thank you.

The impact.

So hopefully reduce your thank you Bradley hopefully that's clear that everything remains on the timeline because we've laid out previously and so far everything remains on track both for yeah activities to support the CMC section of the B.L.A.

As well as this scale up and now we're continuing to build commercial inventories. So that hopefully at launch will be able to provide the the medicine to patients need and part of that is the capacity planning.

To make sure that we have dual source as a manufacturer and again as Brad noted that remains on track with the higher than planned moving forward as well.

I was supposed to be in Ireland next week actually to visit that plant.

Soon.

And your next question gone from on this on beats a group tough from Guggenheim Securities. Your line is now open.

Operator: And your next question comes from Alinas Anbita Gupta from Guggenheim Securities. Your line is now open. Hi guys.

Hi, guys. Thanks for the following a follow up just following up a bit. This question back on manufacturing on the team terrorists decide and apologies if I missed this earlier if I have could you comment on your efforts in building the in house.

Operator: Thanks for the follow-up. Just following up on Ritu's question, but on manufacturing on the gene therapy side. And apologies if I missed this earlier; if I have. Could you comment on your efforts in building the in-house gene therapy manufacturing capabilities and whether we might see a hybrid manufacturing model first, and then probably completely in-house? Thank you.

Gene typing manufacturing capabilities, and if you might see a hybrid manufacturing model for us and then probably a completely in house. Thank you.

John Crowley: Thanks, Anvita, and that's exactly what we're evolving to. You know, as we began working two years ago in the gene therapy space, we relied for our initial materials in batons on materials supplied by Nationwide Children's. We'd then gone through an exhaustive review of the entire supply chain, including plasmid producers and ultimately the contract manufacturers. We, as you know, work very closely with our partners at Grammar, ThermoFisher.

Thanks, Andy to and that's exactly what we're evolving to you know is we began in that working two years ago in the gene therapy space, we relied for our initial materials in battles on material supply, but nationwide children's we then gone through an exhaustive review at the entire supply chain, including plasmin producers and ultimately to.

Contract manufacturers, we as you know worked very closely with our partners. It forever Thermo Fisher I have been enormously impressed with their capabilities in a growing capacity they will supply the material for us on a commercial basis for the sealant 60 of them three buttons programs. We're also working with them now.

John Crowley: I have been enormously impressed with their capabilities and their growing capacity. They will supply the material for us on a commercial basis for the CLN6, and CLN3 baton programs. We're also working with them now on other programs, including, for instance, our Pompei gene therapy manufacturing. While we're doing that, in parallel, as you know, we are evaluating sites for our own in-house manufacturing. We're actually designing now a Phase I-II manufacturing facility. We'll have more to say about that in the months ahead, as well as looking forward, beginning the basis of design for a larger commercial-scale facility. I continue to believe that if you're going to be one of the world's leading companies in the field of genetic medicine and gene therapy, you also need to be one of the world's experts in the manufacturing of those gene therapies.

Now on other programs, including for instance, our pump a gene therapy manufacturing, while we're doing that in parallel is you know we are evaluating sites for our own in house manufacturing were actually designing now a phase one two manufacturing facility will have more to say about that.

In the month ahead.

As well as looking forward beginning the basis of design for a larger commercial scale facility I continue to believe that if you're going to be one of the world's leading companies in the field to genetic medicine in gene therapy gene therapies, you also needs to be one of the world experts in the manufacturing of those gene therapies.

John Crowley: And again, this was something Amicus has been uniquely suited for with our, you know, more than 75 people at Amicus working in technical operations, manufacturing, and quality. Having gone through, for the last seven years, the design, scale-up, and manufacture now at commercial scale and quality of ATGAA, one of the most complicated glycosylated proteins ever manufactured. We've done that successfully. So we really think we can translate many of those skills and capabilities at Amicus now to become one of the world's leading manufacturers of gene therapy. So it's been a very carefully thought-out strategy and one that we remain on track for. Thank you.

And again this was something amick as has been uniquely suited for with our.

More than 75 people at AMAK is working in technical operations manufacturing and quality, having gone through for the last seven years, the design scale up and manufacturer now at commercial scale in quality for 80, GA one of the most complicated quite costly proteins ever manufactured.

We've done that successfully.

So we really think we can translate many of those skills and capabilities that abacus now to become one of the world's leading manufacturers for gene therapy. So we've been very carefully thought through strategy and one that we remain on track.

Thank you.

John Crowley: And there are no further questions at this time. I would now like to turn the conference back to Mr. John Crowley, Chairman and CEO, for closing remarks.

And there are no for your question. This time of all like to during the conference back to Mr., John College, Chairman and CEO for closing remarks.

John Crowley: Great, everybody, thank you for the great questions. I hope everybody and your families remain healthy. And again, we had a really strong second quarter and look to finish out a really strong second half of the year. Thanks, everybody, have a great day. Ladies and gentlemen, this concludes today's conference.

Great everybody. Thank you for the great questions I hope everybody in your family's remained healthy and again, we had a really strong second quarter and look to finish out it really strong second half the year. Thanks, everybody have a great day.

Ladies and gentlemen, this concludes today's conference call. Thank you and have a great that.

Operator: Ladies and gentlemen, this concludes today's conference call. Thank you, and have a great day. Thanks for watching!

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