Q2 2021 Amicus Therapeutics Inc Earnings Call
[music].
Good morning, ladies and gentlemen, and welcome to the Amicus Therapeutics second quarter 2021 financial results conference call and webcast.
Operator: Welcome to the Amicus Therapeutics Second Quarter 2021 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 Tor and then zero on your touchstone telephone. As a reminder, this conference call is being recorded. I would now like to turn the conference over to your host, Mr. Andrew Fawnen. Head of Investor Relations, you may begin.
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.
As a reminder, this conference call is being recorded.
I'd now like to turn the conference over to your host Mr. Andrew Faughnan.
Net of Investor Relations you may begin.
Andrew Faughnan: Operator. Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics' second quarter 2021 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 Cweeney, Chief Financial Officer, and Dr. Jeff Costelli, Chief Development Officer.
Thank you operator.
Good morning, Thank you for joining our conference call to discuss amicus Therapeutics second quarter 2021 financial results and corporate highlights speaking on today's call. We have John Crowley, Chairman and Chief Executive Officer, Bradley Campbell, President and Chief Operating Officer, Daphne Queenie, Chief Financial Officer, and Dr. Jeff Castelli, Chief Development Officer joining from.
Andrew Faughnan: Joining for Q&A, we'll have Dr. Mitchell Goldman, Chief Medical Officer, as well. As referenced on slide two, we may make forward-looking statements within the meaning of the Private Security's Litigation Reform Act of Medicine.
Andrew Faughnan: Litigation Reform Act of 1995 relating to our business as well as our plans and prospects. Our forward-looking statements should not be regarded as representations by us that any of our plans will be achieved. Any or all 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.
Andrew Faughnan: You are cautioned not to place undue reliance on any forward-looking statements which speak only to the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this presentation and conference call to reflect events or circumstances after the date hereof. For a full 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 10K for the year-ended December.
Andrew Faughnan: 31st, 2020, and the quarterly report on Form 10Q for the quarter ended June 30, 2021, to be 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?
John Crowley: Great. Thank you, Andrew, and welcome everyone to our second quarter 2021 results conference call. Before we begin, I just want to note yesterday the sudden death of Dr. Tachi Yamada. Tachi, as many of you know, is a giant in our industry, and among his many contributions, he was a passionate advocate for research for people living with rare diseases. And I came to know Tachi well as he chaired the Orkin Disease Center with Jim Wilson at the University of Pennsylvania, where I had the honor to serve with him. And our deepest condolences to his family.
Among as many contributions he was a passionate advocate for research for people living with rare diseases and I came to know touchy well as he chaired the orphan disease Center with Jim Wilson at the University of Pennsylvania, where I had the honor to serve with him and our deepest condolences to his family.
I am pleased to report for Amicas that the second quarter of 2021 reflects execution across our strategic priorities that we outlined at the beginning of the year as.
As we did and this morning's press release, so I'd like to highlight several key accomplishment.
First gallop old continues and strong performance and remains the cornerstone of our success.
John Crowley: I'm pleased to report for Amicus that the second quarter of 2021 reflects execution across our strategic priorities that we outlined at the beginning of the year. As we did in this morning's press release, I'd like to highlight several key accomplishments. First, Gallifold continues its strong performance and remains the cornerstone of our success. With $77 million in second quarter revenue, we are very pleased with the continued momentum of Gallifold adoption globally.
With $77 million and second quarter revenue. We are very pleased with the continued momentum of the Gallup old uptake globally.
The number of patients on Gallup hold at the end of the quarter continues to exceed our external or internal expectations. As we continued to add new patients from both switch and naive populations throughout the quarter.
And exciting news for Gallas hold on Monday. This week, we announced that the European Commission has approved approved the expanded Y-you label for Gallo hold for the longterm treatment of February disease, and adolescents, aged 12 and older with and amenable mutation.
John Crowley: The number of patients on Gallifold at the end of the quarter continues to exceed our internal expectations as we continue to add new patients from both switch and naive populations throughout the quarter. And exciting news for Gallifold.
This is a transformative moment for the February community and the European Union, where pediatric patients now aged 12 and above have a new treatment option for the first time and over 15 years.
Yeah.
Second are key regulatory initiatives for a tee GAA remain on track and our R&D pipeline of gene therapies continues to advance and.
John Crowley: On Monday this week, we announced that the European Commission has approved the expanded EU label for Gallifold for the long-term treatment of February disease in adolescents aged 12 and older with an amenable mutation. This is a transformative moment for the February community in the European Union, where pediatric patients now aged 12 and above have a new treatment option for the first time in over 15 years. Second, our key regulatory initiatives for ATGA remain on track, and our R&D pipeline of gene therapies continues to advance.
I'm pleased to share today that the rolling BLA and the NDA submission for a T. G E. A R. A novel next generation 2 components therapy for Pompeii disease have been completed.
We are also pleased to share that following a positive repertory and co rep for tour meeting the E. U regulators are supportive of marketing authorization application or MAA submissions for a tee GAA we.
We expect to complete these EAA submissions and the second half of this year.
We continue to have great confidence and a tee GAA to benefit people living with Pompeii disease, and we intend to move it rapidly through these regulatory submissions towards approval.
John Crowley: I am pleased to share today that the rolling BLA and the NDA submissions for ATGA, our novel next-generation two-component therapy for Pompeii disease, have been completed. We are also pleased to share that following a positive rapporteur and co-raperture meeting, the EU regulators are supportive of marketing authorization application, or MAA, submissions for ATGAA. We expect to complete these EMA submissions in the second half of this year
Within our gene therapy pipeline, we continue to further lead batten programs for <unk>, 3 and seal and 6 as well as our most advanced preclinical gene therapy programs.
And through our broad research collaboration with Doctor, Jim Wilson, and the University of Pennsylvania. We are highly encouraged by the continued preclinical data and the progress we have seen thus far from our February and pump a disease.
Gene therapy clinical candidates.
And third the Amicas cash position is sufficient to achieve self sustainability under the current plan without the need for any future dilutive financings.
John Crowley: We continue to have great confidence in ATGA to benefit people living with Pompeii disease, and we intend to move it rapidly through these regulatory submissions toward approval. Within our gene therapy pipeline, we continue to further lead batten programs for CLN3 and CLN6, as well as our most advanced pre-clinical gene therapy programs. And through our broad research collaboration with Dr. Jim Wilson and the University of Pennsylvania, we are highly encouraged by the continued preclinical data and the progress we have seen thus far from our Febre and Pompeii disease gene therapy clinical candidates. And third, the Amicus Cash position is sufficient to achieve self-sustainability under the current plan without the need for any future dilutive financing.
Our continued revenue growth prudent expense management and growth potential has allowed us to reach this important milestone as we continued to realize our vision of delivering groundbreaking.
And potentially cured of new medicines for people living with rare diseases around the world.
Turning to slide for we are well on track to achieve our 5 key strategic priorities for 2021.
These include.
Yellow folder precision medicine for February disease.
We will continue to drive <unk> availability to more people living with February disease, with amendable variance and existing and new markets.
Look to achieve global product revenue of $300 million to $315 million this year, which reflects strong momentum and demand behind this precision medicine and globally.
Second we remain steadfast and passionate and our commitment to advancing a T. G E..2 global regulatory submissions for the benefit of as many people living with Pompeii disease and as quickly as possible.
John Crowley: Our continued revenue growth, prudent expense management, and growth potential have allowed us to reach this important milestone as we continue to realize our vision of delivering groundbreaking and potentially curative new medicines for people living with rare diseases around the world. Turning to slide four, we are well on track to achieve our five key strategic priorities for 2021. These include Gallifold, our precision medicine for February disease. We will continue to drive Gallifold's availability to more people living with February disease with amenable variants in existing and new markets.
Third we are advancing are industry, leading rare disease gene therapy portfolio.
Stemming from a global research and gene therapy Center of excellence and Philadelphia, we will be advancing the clinical development manufacturing and regulatory discussions across multiple programs and our gene therapy pipeline.
4 and addition, we are progressing our manufacturing capabilities and capacity to build world class technical operations to support all Amicas gene therapy programs.
And finally again, we continue to maintain a strong financial position as we carefully manage our expenses and investments again and we remain fully funded through all major milestones. So with that introduction. Let me go ahead, now and hand, the call over to Bradley Campbell, our President and Chief operating officer to further highlight the <unk> performance.
John Crowley: We look to achieve global product revenue of 300 million to 315 million this year, which reflects strong momentum and demand for this precision medicine globally. Second, we remain steadfast and passionate in our commitment to advancing ATGA through global regulatory submissions for the benefit of as many people living with Pompeii disease as quickly as possible. Third, we are advancing our industry-leading rare disease gene therapy portfolio. Stemming from our global research and gene therapy Center of Excellence in Philadelphia, we will be advancing the clinical development, manufacturing, and regulatory discussions across multiple programs in our gene therapy pipelines.
Darling.
Thanks, John and good morning, everyone.
As John mentioned, all now walk you through and more detail on girlhood performance for the quarter on slide 6 we give our global snapshot of the Gallup hold commercial progress for the quarter total product revenue was $77.4 million driven by strong patient demand new patient starts and business continuity.
Global compliance and adherence rates continue to exceed 90 per cent.
The geographic breakdown of revenue during the quarter was 53.7 million or 69% of revenue generated outside the United States with the remaining $23 and 7 million or 31% coming from within the United States.
And we've mentioned before this is in line with the roughly 70.30 split that we expect to see as we continue to grow both parts of the global business.
Now turning to slide 7 Q2 was another great quarter for our global commercial efforts. The business continues to be incredibly resilient with patients added and all of our major markets.
John Crowley: In addition, we are progressing our manufacturing capabilities and capacity to build world-class technical operations to support all amicus gene therapy programs. And, again, we continue to maintain a strong financial position as we carefully manage our expenses and investments.
In fact, the month of June was the second best month, and net new patient starts and our international markets since launch and globally. The rolling 3 months average patient starts is greater than the 6 month and 12 month average respectively, which should include building momentum.
Going into the second half of the year.
Second quarter revenue reflected increased patient demand and also benefited slightly from the timing of borders and ex uf's geographies and and continuing FX tailwind.
John Crowley: Again, we remain fully funded through all major milestones. So with that introduction, let me go ahead now and hand the call over to Bradley Campbell, our president and chief operating officer, to further highlight the Gallifold performance.
And we highlighted and their queue 1 call. We continue to see some sporadic COVID-19 related slowdowns and new patient starts due to delays at the point of care between patient identification and initiation of treatment importantly, though our supply chain remains fully into our customers have confidence they can access gal fold and our field team has been.
And able to achieve a substantial majority of their pre COVID-19 touch points through a combination of in person digital telephonic and other means of interacting with physicians.
Bradley L. Campbell: Thanks, John, and good morning, everyone. As John mentioned, I'll now walk you through in more detail our Gallifold performance for the quarter. On slide six, we give our global snapshot of Gallifold commercial progress. For the quarter, total product revenue was $77.4 million, driven by strong patient demand, new patient starts, and business continuity. Global compliance and adherence rates continue to exceed 90%. The geographic breakdown of revenue during the quarter was 53.7 million, or 69% of revenue generated outside the United States, with the remaining 23.7 million or 31% coming from within the United States.
We continue to monitor the depend on impact, but the good news is we observed improvements and the second quarter, so that and many of our field personnel and so on increased ability to interact with physicians in person.
The first 2 quarters of 2021 had been in line with our expectations and as we've mentioned on previous calls while we continue to see and expect growth vs. The same quarter last year due to a variety of factors the rate of growth from quarter to quarter within the year is typically non linear so usually and a given year <unk> and <unk> are stronger quarters.
And first quarter and third quarter and that pattern is continued so far this year with a great performance now here and the second quarter.
Based on this momentum and continuing to anticipate a second half recovery from Covid or confident and meeting our full year 2021 guidance of $300 million to $315 million and global revenue.
Now on slide 8 we called out several of the drivers and metrics, which lay the foundation for the growth and 2021 and beyond and.
As we've mentioned we ended 2020 with 1400 plus patients on Gallup holds globally and we're now at about a 49% global market share of treated amendable patients.
Bradley L. Campbell: As we've mentioned before, this is in line with the roughly 70-30 split that we expect to see as we continue to grow both parts of the global business. Now turning to slide seven, Q2 was another great quarter for our global commercial efforts. The business continues to be incredibly resilient, with patients added in all of our major markets. In fact, the month of June was the second best month for net new patient starts in our international market since launch, and globally, the rolling three-month average patient starts is greater than the six-month and 12-month average, respectively, which should include building momentum going into the second half of the year.
So while we're achieving higher market shares and countries, where we've been approved the longest there's still plenty of opportunity to continue to switch patients over the gal hold.
We also know that there are significant numbers of diagnosed and treated patients who have remained who have a mental variance and while the global mix remains about 60% switch and 40% naive patients on Gallup hold and many markets, we're starting to see stronger uptake and naive patients as we've said previously over the next few years, we'd expect to see that right of switching.
E patience to be about 50, 50, and and the long term, we expect to see that percentage reverse in favor of naive patients, reflecting further market growth for gallophone.
All of that is underpinned by compliance and adherence rates that continued to exceed 90 per cent reiterating our belief that those patients who go on Gallup hold tend to stay on Gallophone.
Bradley L. Campbell: Second quarter revenue reflected increased patient demand and also benefited slightly from the timing of orders and ex-US geographies and a continuing FX tail line. As we highlighted in our Q1 call, we continue to see some sporadic COVID-related slowdowns and new patient starts due to delays at the point of care between patient identification and initiation of treatment. Importantly, though, our supply chain remains fully intact.
The value of Gal holds us also been recognized by payers with nearly 100 per cent of insurance Reauthorizations granted and 2021 with us pairs and a very strong track record of successfully negotiating and now renegotiating reimbursement outside of the United States.
A relentless focus on ensuring access to Gal hold continues to be a strength.
And as John just briefly touched upon and thrilled to announce that the European Commission approved the expanded European label of Gal fold and the and lessen population. So now for the first time and over 15 years individuals aged 12 to 16 years old with and amenable mutation have a new treatment option with Gal fold, which is of course the <unk>.
Only oral therapy approved for the longterm treatment of February disease.
And his approval will allow us to expand the eligible treatment population, a further 5% to 10% and Europe and will continue to work very closely with regulatory authorities and other geographies to secure access to gallop old for eligible patients as quickly as possible.
Bradley L. Campbell: Our customers have confidence they can access Gallifold, and our field team has been able to achieve a substantial majority of their pre-COVID touch points through a combination of in-person, digital, telephonic, and other means of interacting with physicians. We continue to monitor the pandemic's impact, but the good news is that we observed improvements in the second quarter so that many of our field personnel saw an increased ability to interact with physicians. The first two quarters of 2021 have been in line with our expectations, and as we've mentioned on previous calls, while we continue to see and expect growth versus the same quarter last year, due to a variety of factors, the rate of growth from quarter to quarter within So usually, in a given year, 2Q and 4Q are stronger quarters than the first quarter and third quarter.
Finally to reiterate another important driver of growth for Gallup hold US continued geographic expansion and 2021, we expect to secure reimbursement at least 5 new countries as we look to expand access to fabric patients with a medical variance around the globe and we expect to see continued traction and our newer markets and the Latin America and Asia Pacific elsewhere.
As a reminder, Gallup hold has received regulatory approval and now over 40 countries and we have commercial sales and over 30 of those today.
So despite the recent COVID-19 related headwinds and certain geography's demand for Gaelic hold worldwide has never been stronger with some cues a potential new gal fold patients building and multiple geographies, we are confident and our guidance of $300 million to $350 million and full year global sales and.
As part of that guidance as a reminder, we project net new patient starts with this year will be even greater than and 2020, and we do expect growth and the number of patients and corresponding revenue to be weighted to the second half of the year as the Covid impact continues to us.
On slide 9 with several years and performance and a track record behind US. We can confidently say we are on a path and the next approximately 2 years to be important milestone of $500 million and annual global revenue per Gallup hold.
Bradley L. Campbell: And that pattern has continued so far this year with a great performance now here in the second quarter. Based on this momentum and continuing to anticipate a second half recovery from COVID, we're confident in meeting our full year 2021 guidance of $300 to $315 million in global revenue. Now on slide eight, we've called out several of the drivers and metrics which lay the foundation for growth in 2021 and beyond. As we've mentioned, we ended 2020 with 1,400 plus patients on Gallifolds globally, and we're now at about a 49% global market share of treated amenable patients.
The exact timing of this milestone will be dependent on how quickly things return to normal posts COVID-19.
But we continue to expect to generate $1 billion and cumulative revenue over the next 3 years, which will continue which will contribute a significant amount towards funding, our RMB and opex over that period.
We also have even further confidence and the over billion dollars peak revenue opportunity for Gallup hold us we continue to see significant growth and the fab gray market globally, driven by continued diagnosis from high risk screening newborn screening and other diagnostic initiatives, which we continue to support and invest and as well.
And finally as a reminder, we have orphan and exclusivity and the US and Europe. In addition to our 26 Orange book listed patents that gave us IP coverage into the late twenties thirties, 13 of which provide protection and through 2038.
So a lot of opportunities to continue to provide access to gallop hold globally for a long time to come.
So with that and let me know turn the call over to Dr. Jessica Stelly, Our Chief Development Officer, Jeff will highlight R. A T J a program and our gene therapy pipeline Jeff.
Bradley L. Campbell: So while we're achieving higher market shares in countries where we've been approved the longest, there's still plenty of opportunity to continue to switch patients over to Gallifold. We also know that there are significant numbers of diagnosed, untreated patients who have amenable variants.
Thanks, Brad and good morning, everyone.
Moving on to our art and the updates on slide 11, and we wanted to start with a T. J a R. A novel next generation therapy for punk day disease.
First we believe it's always important to recognize the significant unmet needs that remains for people living with Pompeii, Despite having 1 of true therapy on the market.
Pompeii disease is a severe and stayed on neuromuscular disease and 1 of the most prevalent license almost disorders.
Bradley L. Campbell: And while the global mix remains about 60% switch and 40% naive patients on Gallifold, in many markets, we're starting to see stronger uptake in naive patients. As we've said previously, over the next few years, we expect to see that rate of switch in naive patients to be about 50-50. And in the long term, we expect to see that percentage reverse in favor of naive patients, reflecting further market growth All of that is underpinned by compliance and adherence rates that continue to exceed 90%, reiterating our beliefs that those patients who go on Gallifold tend to stay on Gellif.
In addition to the numerous individual human tragedy, we've seen multiple publications and natural history studies of Pompeii patients that highlight the initial benefit of treatment being followed by continued long term decline on key measures of disease for many patients.
As a reminder, that sustaining high unmet need for Iraqi experienced patients and only ever been and studied and a controlled setting and our phase III propelled clinical trial all other controlled laid on Sept, Pompey disease, studying and have been and participants naive to treatment.
Moving out of slide 12, we present, a summary of the primary key secondary and biomarker and clients recently presented at the International Congress on neuromuscular disease and May.
As a reminder, propel with a double blind randomized study assessing the efficacy and safety of a T. G E and adult treatment and naive and <unk> experienced participants against the currently approved therapy.
And we enrolled 123 study volunteers at 62 sites and 24 countries.
117 of these patients completed this study and all 117 voluntarily enrolled and the extension study to continue on a T J or transition to a T. J as they are only disease modifying treatment for Pompeii.
Bradley L. Campbell: The value of Gallifold has also been recognized by payers, with nearly 100% of insurance reauthorizations granted in 2021 to U.S. payers and a very strong track record of successfully negotiating and now renegotiating reimbursement outside of the United States. A relentless focus on ensuring access to Gallifold continues to be a strength.
Here on the Sly and gripping these endpoints and to domains and motor function muscle strength pulmonary function patient reported outcomes and Biomarkers you can see that the vast majority of and points across all of these domains, Dave on a T. J over Africa's today's alpha and both the overall and Iraqi experienced populations.
We believe this consistency of effect across the key disease manifestations of Pompeii highlights the potential impact on a T. G. A to address unmet needs per patient living with Pompeii.
Bradley L. Campbell: And as John just briefly touched upon, I'm thrilled to announce that the European Commission approved the expanded European label of Gallifold in the adolescent population. So now, for the first time in over 15 years, individuals aged 12 to 16 years old with an amenable mutation have a new treatment option with Gallifold, which is, of course, the only oral therapy approved for the long-term treatment of February. This approval will allow us to expand the eligible treatment population a further 5 to 10% in Europe, and we will continue to work very closely with regulatory authorities and other geographies to secure access to Gallifold for eligible patients as quickly as possible. Finally, to reiterate, another important driver of growth for Gallifold is continued geographic expansion.
As a reminder, on the primary and point of 6 minute walk. This study patients on a T J outperformed those and Aglukkaq today's alpha and the overall population with a difference between groups of 14 meters, which did not reach statistical significance for superiority.
However on the first tee secondary endpoint per cent predicted force vital capacity or S. B C.
And the overall population atg issue and nominally statistically significant and clinically meaningful difference for superiority vs. Aglukkaq today's alpha with a difference of 3% and a P value and 0.023.
This is and unimpressive finding as progressive blossom pulmonary function is a leading cause of mortality and Pompeii and this was the main and point upon which Aglukkaq today's alpha was initially approved.
We remind everyone and that to propel study tested for superiority vs approved therapy, not placebo and at the bar for approval on the second generation product generally requires demonstration only of Noninferiority vs approved therapy.
And shown on prior and calls and post hoc Noninferiority analyses for the primary endpoint of 6 minute walk and the first key secondary endpoint and SEC. These non authority analyses were highly significant.
Bradley L. Campbell: In 2021, we expect to secure reimbursement in at least five new countries as we look to expand access to Fabry patients with the Medival variants around the globe. And we expect to see continued traction in our newer markets in Latin America and Asia Pacific and elsewhere. As a reminder, Gallupold has received regulatory approval in now over 40 countries, and we have commercial sales in over 30 of those today. So, despite the recent COVID-related headwinds in certain geographies, demand for Gallifold worldwide has never been stronger, with some cues of potential new Gallifold patients building in multiple geographies. We are confident in our guidance of 300 to 350 million in full-year global sales.
Moving on and now to slide 13, we have our next steps surrounding development regulatory and manufacturing strategy for a T J.
First as John mentioned, the Rolling BLA submission for ATB, 200, and and a new drug application from MC with that the 2 components that make up a T. J have both been submitted to F. D. A the.
The clinical modules were completed and submitted to F. D. A and the second quarter CMC module was completed and submitted and July which completed the last parts of the rolling DLA and to F. D. A.
We are pleased to also share the following a positive Emma rapid tour and co rapid toward meeting E regulators are supportive of marketing authorization.
Vacation submissions and <unk> and the second half of this year.
And we expect additional global filings to follow.
And June we announce ATCA was granted a positive scientific opinion through the early access to medicine scheme or <unk> by the Uk's NHRA.
This positive opinion recognizes the high unmet medical need faced by the pump a community and from its eligible adults living with late onset Pompeii disease could received <unk> offer for at least 2 years to switch and have access to a G. A T G. A prior to marketing authorization and the and the U K.
For the younger pump a community we will look to expand our ongoing open label adolescence study and 12 to 18 year olds and are clinical study for pump 8 patients with infantile onset disease is expected to begin this year.
Bradley L. Campbell: As part of that guidance, as a reminder, we project net new patient starts this year will be even greater than in 2020, and we do expect growth in the number of patients and corresponding revenue to be delayed to the second half of the year as the COVID impact continues to ease. On slide nine, with several years of performance and a track record behind us, we can confidently say we're on a path in the next approximately two years to the important milestone of $500 million in annual global revenue for Gallifold.
At this point, we're pleased to report that over 150 patients worldwide are now being treated with a T. G E and to put that figure into perspective. This is close to 5% of people with Pompeii disease today currently on treatment.
And finally in response to the many requests for expanded access will be received for children living with infantile on Sept. Pompeii are expanded access programs for both those with infantile on set and adult onset we continued to expand from multiple individuals.
Moving on now to slide 15.
To briefly highlight our industry, leading portfolio of gene therapies per rare diseases.
We continued to follow the first 13 participants and the 4 participants and our sales on 6 and seal and 3 phase 2 studies, where data from both studies continue to show the potential to stabilize disease progression and these children and when compared to natural history really excited about this result, as these are devastating.
Bradley L. Campbell: The exact timing of this milestone will be dependent on how quickly things return to normal post-COVID, but we continue to expect to generate a billion dollars in cumulative revenue over the next three years, which will contribute a significant amount towards funding our R&B and OPEX over that period.
Neurodegenerative diseases.
With early mortality.
Remain focused on the manufacturing activities and regulatory discussions for both programs to enable dosing additional patients with GNP clinical grade material and future registration directed studies.
Jeffrey P. Castelli: We also have even further confidence in the over billion dollar peak revenue opportunity for Gallifold as we continue to see significant growth in the Fabry market globally, driven by continued diagnosis from high-risk screening, newborn screening, and other diagnostic initiatives, which we continue to support and invest in as well. And finally, as a reminder, we have orphan exclusivity in the U.S. and Europe, in addition to our 26 orange book listed patents that give us IP coverage into the late 2030s, 13 of which provide protection through 2038.
The head on Slide 16, we continue to make progress across our preclinical gene therapy programs with pen and particularly regarding our fab ray and pump agent and therapy programs.
Data from these programs further validate our innovative approach to gene therapy, I, combining amicas us protein engineering expertise with pens gene transfer technologies.
We continued to build out manufacturing capabilities for both programs ahead of the expected and enabling studies.
We believe this is just the beginning as part of this collaboration Amicas has rights to over 50 diseases, including 7 currently and active preclinical programs.
With that I would like to now turn the call over to Daphne, claiming to review our financial results guidance and outlook Daphne.
Thank you Jack and good morning, everyone. Our financial overview begin on slide 18, with our income statement for the second quarter ending June 30th 2021.
Jeffrey P. Castelli: So a lot of opportunities will continue to provide access to Gallifold globally for a long time. So with that, let me now turn the call over to Dr. Jessica Steli, our chief development officer. Jeff will highlight our ATGA program and our gene therapy pipeline.
And a quarter, we achieved California revenue at 77.4 million, which is a 24 per cent increase over the second quarter of 2020 distant.
This increase year over year operational growth 17.2 per cent measured at constant currency exchange rates.
Total operating expenses of 107.9 million and the second quarter of 2021 were relatively flat as compared to $107 million and the second quarter of 2020.
Jeffrey P. Castelli: Thanks, Brad, and good morning, everyone. Moving on to our R&D updates on slide 11, we wanted to start with ATGA, our novel Next Generation Therapy for Pompey Disease. First, we believe it's always important to recognize the significant unmet need that remains for people living with Pompeii, despite having one approved therapy on the market. Pompei disease is a severe and fatal neuromuscular disease and one of the most prevalent lysosomal
On a non-GAAP basis total operating expenses were 93.5 million and the second quarter of 2021 as compared to $95.9 million and the second quarter of 2020.
Decreased and research and development costs reflects the timing of investments and our pipeline.
Officially the timing of manufacturing batches, and a T G I, a and other research costs, which we expect to incur and subsequent quarters.
And we define non-GAAP operating expense ex research and development and SG&A expenses, excluding share based compensation expense changes and fair value a contingent consideration and depreciation.
Jeffrey P. Castelli: In addition to the numerous individual human tragedies, we've seen multiple publications in natural history studies of Pompeii patients that highlight the initial benefit of treatment being followed by continued long-term decline on key measures of disease for many patients. As a reminder, the sustaining high unmet need for E or T experienced patients has only ever been studied in a controlled setting in our phase three propel clinical trial. All other controlled late-onset pumpy disease studies have been in participants naive to treatment.
Net loss for the second quarter of 2021, 51.2 million are and 19 cents per share as compared to a net loss at 52.5 million or 20 cents per share for the prior and year period.
As of June 32021, and we had approximately 266 million shares outstanding.
Turning now to slide 19, with our current cash position and we are on a path yourself sustainability without the need for any future due to financing. We have achieved this milestone on through our revenue growth with counsel as well as driving efficiencies cost savings and continued expense management.
And the third year straight Yeah, we expect total non-GAAP operating expenses and 2021 to remain relatively flat and we leverage the global commercial infrastructure and it's already in place for the a T G and orange and other products and our pipeline true.
Jeffrey P. Castelli: Moving now to slide 12, we present a summary of the primary, key, secondary, and biomarker results recently presented at the International Congress on Neurumuscular Diseases in Maine. As a reminder, Propel was a double-blind randomized study assessing the efficacy and safety of ATGAA in adult treatment naive and ERT experience participants against the currently approved therapy. We enrolled 123 volunteers at 622 sites in 24 countries.
In addition costs associated with the development and a T. G. I a true multiple gene therapy program and on pipeline and maintain financial discipline and while meeting on our taxes.
To reiterate all high priority research programs and gene therapy, and moving ahead and we continue to support the work with the world from that and pain.
A few comments about our cash position and 2021 financial guidance.
Cash cash equivalents and marketable securities with 383.1 million on June 30th 2021 and compare too.
Jeffrey P. Castelli: 117 of these patients completed the study, and all 117 voluntarily enrolled in the extension study to continue on ATGA or transition to TGA as their only disease-modifying treatment for Pompeii. Here, the slide grouping these endpoints into domains of motor function, muscle strength, pulmonary function, patient-reported outcomes, and biomarker. You can see that the vast majority of endpoints across all of these domains favor ATGA over Albuquerada's alpha in both the overall and ERT experience population.
$183 and 3 million at December 31, 20 <unk>.
We are reiterating a full year galiform revenue guidance at 300, and 315 million and are non-GAAP operating expense guidance at 410 million to $429.
And with that I can try and call back over to John and for clothing comments.
Great. Thank you Daphne and also Jeff and Bradley as you can see we have been relentlessly focused on execution and performance across the business.
Driven by a global team of passionate entrepreneurs at Amicas, who had led and will continue to lead us on our patient focused mission I am confident that as the world emerges from this COVID-19 pandemic amicus will emerge even stronger so with that operating we're happy to turn the call over to questions.
Jeffrey P. Castelli: We believe this consistency of effect across the key disease manifestations of Pompeii highlights the potential impact of ATGA to address unmet needs for patients living with Pompeii. As a reminder, on the primary endpoint of six-minute walk distance, patients on ATJA outperform those on Aglocococidase alpha in the overall population, with a difference between groups of 14 meters, which did not reach statistical significance for superiority. However, on the first key secondary end point, percent predicted force vital capacity, or SVC, in the overall population, ATGA showed a nominally statistically significant and clinically meaningful difference for superiority versus Aglicosity Alpha, with a difference of 3% and a P value of 0.03. This is an impressive finding as progressive loss of pulmonary function is a leading cause of mortality in Pompeii. And this was the main endpoint upon which Agucusidae's alpha was initially approved.
Thank you ladies and gentlemen, if you have questions. Please press star.
And then the number 1 key on your Touchtone telephone at this time, we asked 50 on the ask 1 question. If you have any additional questions. Please enter back into the queue. You to your question has been answered or you wish to remove yourself from the queue. Please press the pound key. Thank you. Your first question comes from the line a free too.
From Colin your line is open.
Good morning, guys. Thanks for taking the question. So I wanted to ask about how we should think about expectations for the review period, 1 B a T G a BLA and and <unk> our access all the.
I guess priority with you as an option.
That is there anything that's either and necessitates priority review what would trigger priority review.
Or standard curfew, and then I have and very quick follow up on that and find it too. Good. Good morning, Yes, So now and the BLA and the and yeah with the BLA and NDA submission is complete and into F. D. A will expect to hear back hopefully this quarter, we would expect to hear back from the FDA on their acceptance.
Jeffrey P. Castelli: We remind everyone that the Propel study tested for superiority versus approved therapy, not placebo, and that the bar for approval of a second-generation product generally requires demonstration only of non-infriority versus approved therapy, as shown on prior calls. Post hoc non-inferiority analyses for the primary endpoint of the six-minute walk and the first key secondary endpoint of FEC, these non-inferiority analyses were Moving on now to slide 13, we have our next steps surrounding the development, regulatory, and manufacturing strategy for ATGA.
That is part of the submission US you know we did request a priority review.
Reminder, to everybody, we do have breakthrough therapy designation for this product.
It also and part recognizes the significant unmet need and the community as well as the uniqueness of the day to here. So we're hopeful that the FDA will look favorably upon that but we'll find out Wednesday, hopefully accept the filing of the submission with the priority review of course that would trigger a 6 month review time line, but we don't know that yet that's up to the regs.
Jeffrey P. Castelli: First, as John mentioned, the rolling DLA submission for ATB 200 and the new drug application for MIGLSTAT, the two components that make up ATGA, have both been submitted to FDA. The clinical modules were completed and submitted to FDA in the second quarter. The CMC module was completed and submitted in July, which completed the last parts of the rolling DLA to FDA. We are pleased to also share that, following a positive EMA Rapporteur and co-rapporteur meeting, EU regulators are supportive of marketing authorisations of ATGA in the second half of this year. And we expect additional global filings to follow.
Later, so we'll see.
Got it and then on the same application moving to Europe, and your the competitor Uhm, Hi, I believe sort of we petitions and.
And for novel chemical status.
And from D O T a and I'm, assuming that's sort of us updated for exclusivity can you remind us true you wrap the tours are and and how is this sort of.
Chemical novelty uhm sorted out with an E Bay.
Yeah. It is it's first of all I don't want to speculate on discussions for a competitor, but I'll just remind everybody per the E. M. A definition in order for a product to qualify as a new active substance it has to either be a biological substance not previously authorized.
Jeffrey P. Castelli: In June, we announced ATGA was granted a positive scientific opinion through the early access to medicines team or EMS by the UK's NHRA. This positive opinion recognizes the high unmet medical needs faced by the Pompeii community and permits eligible adults living with late-onset Pompeii disease who have received Aglicosity Alpha for at least two years to switch and have access to ATGA prior to marketing authorization in the UK. For the younger Pompeii community, we will look to expand our ongoing open-label adolescent study in 12 to 18-year-olds, and our clinical study for Pompeii patients with infantile onset disease is expected to begin this year.
Or a biological substance previously authorized but differing significantly and safety and efficacy due to the differences in 1 or a combination of its molecular structure. The nature of the source material, where the manufacturing process. So you'll just remind everybody with respect to a T G I a and.
We know it's differentiated we believe it's differentiated on key structural differences, it's differentiate it on a dual components therapy and it has a differentiated clinical efficacy.
So again I'm not going to comment on the denial of that for the neo product and.
We're very confident of the uniqueness of our product and certainly and the data that we have.
Jeffrey P. Castelli: At this point, we're pleased to report that over 150 patients worldwide are now being treated with ATGA. And to put this figure into perspective, this is close to 5% of people with Pompeii disease today currently on treatment.
Got it thanks.
Did you have a quick follow up or to or to recapture it.
Oh that that what's the follow up actually on now it was good okay variety yep.
Yeah, Yeah, so very very interesting dynamics at play for sure.
Your next question comes from the line of Randy miles from you and you'd be S share line is open.
Jeffrey P. Castelli: And finally, in response to the many requests for expanded access that we've received for children living with infantile onset Pompeii, our expanded access programs for both those with infantile onset and adult onset have continued to expand for multiple individuals. Moving on now to slide 15 to briefly highlight our industry-leading portfolio of gene therapies for rare diseases. We continue to follow the first 13 participants and the four participants in our CLN6 and CLN3, Phase 1,2 studies, where data from both studies continue to show the potential to stabilize disease progression in these children when compared to natural history.
Mmm Yeah. Thanks for taking my question just another 1 on the bank and I try interaction Uhm curiosity, you know and the discussions with the email on and off hanging on that be on I.
And there have you seen any different guys and term as you know how that you ask for something yesterday and feeling the submission any difference as in terms of you know how they're getting the clinical data are and plaint. Thanks.
Yeah, it's actually been remarkably similar early and and again I'll remind everybody. This pump type program and the Amicas Palm Pray program. We were the first company to utilize the joint scientific advice.
So that began in the fall of 2018 US we were proposing to propel study.
So we had received prior to study start harmonized feedback from both E M. A and F. D. A together on the trial design and the statistical analysis plan and we've also seen.
Jeffrey P. Castelli: We're really excited about this result as these are devastating neurotogenicernerative diseases with early mortality. We remain focused on the manufacturing activities and regulatory discussions for both programs to enable dosing additional patients with GMP clinical grade material in future registration-directed studies. As shown on slide 16, we continue to make progress across our pre-clinical gene therapy programs with Penn, particularly regarding our Fabre and Pompeii therapy programs. Data from these programs further validate our innovative approach to gene therapy by combining Amicus's protein engineering expertise with Penn's gene transfer technologies.
Very very similar feedback now from E M, a and F. B a on the regulatory path forward and so we think we hope that bodes well.
Mmk, Inc.
Okay.
From the line.
Okay and yourself.
Hi, Good morning, everyone. Thanks for taking my question. So as there are multiple specialist often involved for the treatment of both Pompeii and fabric diseases I wanted to ask of your existing commercial team for Galiform, what the overlap and is for centers that also cheap for Pompeii disease and then if.
And you were to expand into more territory. It's a T. T. A is approved how do you believe this could also benefit the footprint for Galiform. Thank you.
Jeffrey P. Castelli: We continue to build out manufacturing capabilities for both programs ahead of the expected I&D enabling studies. We believe this is just the beginning. As part of this collaboration, Amicus has rights to over 50 diseases, including seven currently in active preclinical programs. With that, I would like to now turn the call over to Daphne Queenie to review our financial results, guidance, and outlook.
Yeah. Thank you, Chris and I'll have Bradley answer. This I'll just highlight that we think there is great leverage from the global commercial infrastructure that we built for Gallo fold. We think we can lever that significantly for a tee GAA and again, we're already expanding the gala fold footprint to all the geographies that would be necessary and appropriate for.
Daphne E. Quimi: Thank you, Jeff, and good morning, everyone. Our financial overview begins on slide 18 with our income statement for the second quarter ending June 30, 2021. For the quarter, we achieved Gallifold revenue of 77.4 million, which is a 24% increase over the second quarter of 2020. This includes year-over-year operational growth of 17%.
<unk>, so great synergies, there, but Friday and I'll, let you comment further.
Yeah. Thanks, John and you you definitely hit the highlights so the good news is lots of leverage between the gullible commercial organization and what will be the a T. G. A commercial organization and in fact right now no intention to add further field support for the launch of a T. J, which is great. Just a few more details help explain that so.
So in terms of sort of a specialty that typically treat these these february and pump and patients. There is some overlap in particular and genetics and metabolic specialists and there are some other some other specialties that ended up trading both patience how're.
Daphne E. Quimi: of 17.2% measured at constant current
Daphne E. Quimi: Total operating expenses of $107.9 million in the second quarter of 2021 were relatively flat as compared to $107 million in the second quarter of 2020. On a non-gap basis, total operating expenses were 93.5 million in the second quarter of 2021 as compared to 95.9 million in the second quarter of 2020. The decrease in research and development costs reflects the timing of investments in our pipeline, specifically the timing of manufacturing batches of ATGA and other research costs which we expect to incur.
However, there is high degree of overlap in terms of the centers that that followed these patients and so.
Typically.
Academic center or LSD treating center will be following multiple patients with different like several storage disorders, including February and Pate and that's really what gives you the leverage to be able to use 1 commercial organization to support both programs and we've also taken the approach of really higher and more key account managers. So some.
He's used to supporting and entire academic center not just 1 specific physician in terms of how they help support the understanding and utilization of these products. So we feel really good about that leverage and again see very little increase and the commercial infrastructure that will need to support the launch it a D J.
Daphne E. Quimi: expect to incur in subsequent quarters. We define non-gap operating expense as research and development and SGNA expenses, excluding share-based compensation expense, and changes in fair value of
[noise] and have your next question coming from the line cause I knew from now I'm not from J P. Morgan.
Daphne E. Quimi: The net loss for the second quarter of 2021 was $51.1.19 per share, as compared to a net loss of $52.5 million or $0.20 per share for the prior year period. As of June 30, 2021, we had approximately 266 million shares outstanding.
Go ahead.
Hi, guys. Thanks, so much for taking the question quick 1 on Gallup hold on the new patient International add in June being the second best since launch is this really being driven by deeper penetration into the region or maybe new country ads that you saw and the first half and.
Daphne E. Quimi: Turning now to slide 19, with our current cash position, we are on a path to self-sustainability without the need for any future dilutive financing. We have achieved this milestone through our revenue growth with Galfold, as well as driving efficiencies, cost savings, and continued expense management. For the third straight year, we expect total non-gap operating expenses in 2021.
2021, thanks, so much.
Yeah on a pump. Thanks for the question good to hear your voice alternative Bradley just to highlight you had mentioned June it was actually it through that it was and the second quarter that we have seen the most significant number of new patient starts second most significant since product launch, but Bradley and you want to comment further children on a little bit on the dynamics that we're seeing that's driving that.
Yeah. So on a palm good question again and get the good to hear from you. So I think it's a few things first of all it is some new countries.
Daphne E. Quimi: to remain relatively flat as we leverage the global commercial infrastructure that is already in place for the ATGA launch and other products in our pipeline. Transition costs
Central and Eastern Europe. For example have been later on the reimbursement cycle and we're starting to see those countries come on I think we talked about Poland as an example.
Daphne E. Quimi: associated with the development of ATGA
Which is a great opportunity for patients there, but it is also seeing patients come on in our existing larger markets, Germany, or the UK et cetera. During COVID-19. There were some small queues here and there are patients that were disrupted and their ability to get into the hospital and so I think there are some some of that going on but.
Daphne E. Quimi: to multiple gene therapy programs in our pipeline and maintain financial discipline while meeting our objective. To reiterate, all high priority research programs in gene therapy are moving ahead, and we continue to
But it is also increasing penetration into naive patient and so it's reflective of market growth. So we think those are all great sides of of the underlying business and it's a combination of number of factors that again, and we think should continue as we go into the back half of this year, but but beyond as well.
Daphne E. Quimi: to support the work with the Wilson Lab at Penn. A few comments about our cash position and 2021 financial guidance.
Daphne E. Quimi: Cash, cash equivalents, and marketable securities were $3 808.
Thank you and we have here and next question from data on Hassan <unk> per line is open.
Daphne E. Quimi: 83.1 million on June 30, 2021 as compared to 483.3 million at December 31, 2020. We are reiterating our full year Gallifold revenue guidance of 300 million to 315 million and our non-GAP operating expense guidance of 410 million to 420 million. And with that, I will turn the call back over to John for closing comments. Great, thank you, Daphne, and also Jeff and Bradley. As you can see, we have been relentlessly focused on execution and performance across the business, driven by a global team of passionate entrepreneurs at Amicus who have led and will continue to lead us on our patient-focused mission. I am confident that as the world emerges from this COVID pandemic, Amicus will emerge even stronger.
Good morning, and thanks for taking my questions just 1 follow up to and earlier question and another follow up for me regarding the regulatory side on a T. G E. A I I'm not too familiar with the early access to medicine and scheme and the U K, but I'm just looking at the the language and a press release. So I was just wondering if there is so much.
<unk> I guess overlap between the EU and FTA can you maybe speak to how we should think about the exposure to alcohol cause today's alpha for 2 years or more and then secondly, I guess going back to the biology of ATB 206, P modification and maybe a question for Jeff can you speak to the degree of M 60 modification.
On this biologic and whether you think having a better modification profile were produced and even better clinical outcome. Thank you [noise].
Sure dig on thank you I'll, just comment that to remind everybody back and May we announced that the NHRA hit granted early approval to a tee GAA for patients.
Who were treatment experience too had been on for more than 2 years that was the initial indication and again through the process. We would expect that to be for all of us which patients as well as for naive patients as well. The reality is the vast majority of patients and the United Kingdom on the current approved your T have been on.
John Crowley: So with that operator, we're happy to turn the call over to us. Thank you. Ladies and gentlemen, if you have questions, please press TAR and then the number one key on your touchstone telephone. At this time, we ask that you only ask one question. If you have any additional questions, please re-enter the queue. If your question has been answered or you wish to remove yourself from the queue, please press the pound key. Thank you. Your first question comes from the line Vituberal from Cowan. Your line is open.
And for 2 or more years. So we think there's significant opportunity prior to full and final approval and the United Kingdom to offer a tee GAA as requested to many many patients on the current standard of care, we've seen great momentum and enthusiasm since that announcement, just a couple of months ago and they're really continues.
To be a significant unmet need for that particular pump pump a population.
That is now working its way through the local health authorities.
Operator: Good morning, guys. Thanks for taking the question.
And based on feedback and proactive request from physician, who would expect in the months ahead to see significant numbers of requests for patients to be switched from current D. R. T to the a T. J a product with respect to M..6 P modification I'll I'll ask Jeff to comment as well, but Ah.
Ritu Subhalaksmi Baral: So I wanted to ask about how we should think about expectations for the review period once the ATGA, BLA, and NDA are accepted.
Ritu Subhalaksmi Baral: I guess priority review is an option. Is there anything that either necessitates priority review or would?
John Crowley: or standard review, and then I have a very quick follow-up on... That's fine, Ritu. Good morning.
Again, what we've developed at Amick us as a novel biologic known as ATB 200. It is a naturally occurring cell line that we have selected cultivated and now with our partners and Wuxi biologics scaled to commercial scale, maintaining the proper gluck constellation, we think optimal like constellation.
John Crowley: Yeah, so now with the BLA and NDA submissions complete and into FDA, we'll expect to hear back, hopefully this quarter, we would expect to hear back from the FDA on their acceptance of that. As part of the submission, as you know, we did request a priority review. Reminder to everybody, we do have a break.
And very high levels of naturally occurring mental 6 phosphate, including the carbohydrate structure that is this phosphorylated and and all of our in vitro studies preclinical studies and now consistent with the clinical data we've.
John Crowley: through therapy designation for this product. That also, in part, recognizes the significant unmet need in the community, as well as the uniqueness of the data here. So we're hopeful that the FDA will look favorably upon that, but we'll find out once they hopefully accept the filing of the submission. With priority review, of course, that would trigger a six-month review timeline. But we don't know that yet. That's up to the regulators, so we'll see. Got it. And then, on the same application, moving to Europe,
We've seen significantly higher uptake of the protein and again a protein with that naturally occurring carbohydrate structure that is highly phosphorylated that has the ability to not only get and to get into multiple muscle cells and from what we've seen preclinically certainly we see that and I think continued down with the clinical data.
Ritu Subhalaksmi Baral: The competitor has, I believe,
Into.
Ritu Subhalaksmi Baral: sort of repetitioned EMA for novel chemical status for Neo-GAA. I'm assuming that's sort of a bid for exclusivity.
Cardiac tissue, we see it and to skeletal muscle smooth muscle diaphragm and with that we think it's a very differentiated product again and with the enzyme stabilizer D and the chaperone product. So together, we think that's a very novel novel.
John Crowley: remind us who your rapporteurs are and how this sort of chemical novelty is sorted out.
Novel product for people living with Pompeii, and we think it has an optimal level of and 6 P.
John Crowley: sorted out within EMA. Yeah, it is.
John Crowley: Yeah, it is, you know, first of all, I don't want to speculate on discussions for a competitor, but I'll just remind everybody, per the EMA's definition, in order for a product to qualify as a new active substance, it has to either be a biological substance not previously authorized or a biological substance previously authorized but differing significantly in safety and or efficacy due to the differences in one or a combination of its mo So, you'll just remind everybody with respect to ATGAA, we know it's differentiated, we believe it's differentiated on key structural differences, it's differentiated on its dual component therapy, and it has differentiated clinical efficacy.
And not post translational modified but with the cell line that we've selected and cultivated Jeff.
Jeff if I missed anything please feel free to add.
Yeah sure diet on and as John.
<unk> said, when we know that ATB 200 has significantly improved phosphorylation and and 6 P and in fact, the vast majority of all the protein molecules have and M..6 P on average.
Least 1 disc and <unk> for every single ATB 200 molecules. So we think that we've really managed to create something that is pretty optimally targeted.
We don't think that having additional and <unk>.
Notably meaningfully changed our uptake.
And we sort of Max out.
Alright.
Great. Thank you day huh.
Thank you and your next question is from thousand Mr from Goldman Sachs and Caroline is open.
Thanks for taking my question is Andrea on for South and maybe 1 on your bat and disease program. Just wondering when we can expect the next day and what would give us hope.
John Crowley: So, you know, again, I'm not going to comment on the denial of that for the neoproduct, but we're very confident of the uniqueness of our product and certainly in the data that we have. Got it. Thanks. Did you have a quick follow-up or two, or did we just capture it?
Give us to understand from that and release.
Yeah, Jeff do you on a comment on the Batten program.
Yeah. So the the last time, you provided and update on the 13 kids treated with sale and fix and the 4 treated with sale and 3 it was at the World license on what disease meeting earlier this year.
That data set included 8 of the patients who had reached 2 years and follow up and sale and 6 and it was 1 year data and the first 3 kids treated with steel and 3.
Ritu Subhalaksmi Baral: Oh, that was the follow-up, actually, on the... It was good. Okay, very good. Yep.
And that data is continue to show disease stabilization on the Hamburg motor language, and feeling 6 and that you'd be interested physical demand for sale and 3 compared to what we've seen and natural history, which is really encouraging.
John Crowley: It was good. Okay, very good. Yes. Yeah, very interesting dynamics at play, for sure.
Operator: Your next question comes from the line of early Merle from UBS. Your line is open. Hey, thanks for taking the question.
We will continue and we do continue to follow those kid.
We will have additional updates as we have significant amount of new data.
Eliana Rachel Merle: Hey, guys, thanks for taking the question. Just another one on the regulatory interactions. I'm curious, you know, in the discussions with the EMA and then also, you know, the BLA filing, have you seen any differences in terms of, you know, how the U.S. versus the EU is sort of viewing the submission, any differences in terms of, you know, how they're viewing the clinical data or endpoints? Thanks.
That could happen you know, we have and specifically said when but typically key venues, where we do provide updates or either of the child Neurology Society meeting and the fall or at the World meeting early and the year. So those are 2 and places where you can expect potential updates as we have more data to share, but we're really excited by what we've seen and continue to see and that's good.
Okay. Thanks, so much.
Thank you and we have a question coming from the line a few songs from D. T I G.
Line is open.
Great. Thank you and good morning. Thanks for taking my question. So excuse me on H G. A I think you mention that and some patients. After longterm treatment was a luminous items. They start to decline, losing response and I believe the reason is still not well understood. So I wonder I know, it's too early to tell but.
John Crowley: Yeah, it's actually been remarkably similar, Ellie. And again, I'll remind everybody, this Pompeii program, the Amicus Pompeii program, we were the first company to utilize joint scientific advice. So that began in the fall of 2018, as we were proposing the propel study. So we had received, prior to study start, harmonized feedback from both EMA and FDA together on the trial design and the statistical analysis plan. And we've also seen very, very similar feedback now from EMA and FDA on the regulatory path forward. So we think, and we hope that that bodes well.
<unk> and evidence to suggest that.
This.
Observation might not happen to a T G and and your clinical study and the pump a study did you by any chance and row any patients who might have entered the declining face from receiving and zoom in on treatment.
Yeah. Thank you, yeah, and maybe Jeff if you want to feel that and and Mitch after jazz or do you have any color to add please.
Sure, Thanks, John and and.
And what we've seen here now and multiple studies is that there's initial benefit on treatment with the current standard of care, we see patients improve on on 6 minute walk hats and improvements on that day and other muscle function and is it.
Really for about the first 2 years and and even that response can be somewhat variable, but what's dancing and after 2 years and.
Operator: and your next question up from the line at Chris and Smooth, and you're out here, oh, man, don't know. Hi, good morning, everyone. Thanks for taking my question.
Much continued decline almost looking in many cases like it was pre treatment.
We do believe with Atg ay and addressing the the improved and <unk> binding and uptake and also the stability in the blood with the stabilizer that that will hopefully help to better penetrate deeper into tissues and clear glycogen that might not have been cleared and.
Kristen Brianne Kluska: Hi, good morning everyone. Thanks for taking my question. So as there are multiple specialists often involved in the treatment of both Pompeii and Fabry diseases, I wanted to ask your existing commercial team for Gallifold, what the overlap is for centers that also treat Pompeii disease. And then if you were to expand into more territories, if ATGAA is approved, how do you believe this could also benefit the footprint for Gallifold? Thank you.
That could be the cause of that continued progression.
We are really encouraged by the results, we've seen and or ear T experienced patients both and our phase and once you study and now here and propel where those patients had been on treatment for on average over 7 years and well into that.
<unk>, where they're nearly all expected to be declining and we see that we can stabilize the SEC and actually lead to improvements and and 6 minute lock and those patients.
John Crowley: Yeah, thank you, Chris, and I'll have Bradley answer this. But I'll just highlight that we think there is great leverage from the global commercial infrastructure that we built for Gallifold. We think we can leverage that significantly for APGAA. And again, we're already expanding the Gallifold footprint to all the geographies that would be necessary and appropriate for ATGAA. So, great synergies there, but Bradley, I'll let you comment further. Yeah, thanks, John. And you definitely hit the highlights.
So we do think that that not only goes really well for those <unk> experienced patients to offer them something that could change the trajectory of their disease, but hopefully that read through too naive patients longer term when they become more akin to us switch patient and we hopefully will see it continue and sustained benefit and those patients.
Yeah. This is miss I'll, just add that I think with just during the call was important.
To reiterate the majority of 115 patients went on to the access your study will continue to follow them like a regular day to catch and release those at appropriate venues and.
And and she pointed out individual heterogeneity. We also think that's important and again, we look at every patient that's ongoing and they'll trial and the look to find out how they're doing and I gave up report that data on an ongoing basis.
Bradley L. Campbell: So the good news is lots of leverage between the Gallifold commercial organization and what will be the ATGA commercial organization. In fact, right now, there is no intention to add further field support for the launch of ATGA, which is great. Just a few more details to help explain that. So in terms of the sort of the specialties that typically treat these Faberine pont-pate patients, there is some overlap, in particular among genetics and metabolic specialists.
Thank you and next time you have question from a skilled news a line from you'd have a companion your.
And your line is open.
Thank you for taking my questions on your on congratulations on the execution maybe.
And maybe a bit of a question on the patterns and news program Ah do you guys have any thoughts about this upcoming ask Congress September which will discuss.
And a benign therapeutics. Thank you.
Yeah, great. Thank you Gill, we think that'll be and important meeting I'll, just remind everybody that for a battens program for seal and 6 we've dose and.
Bradley L. Campbell: and there are some other specialties that end up treating both patients. However, there is a high degree of overlap in terms of the centers that follow these patients. Typically, an academic center or LSD treating center will be following multiple patients with different lysosomal storage disorders, including Fabri and Pompeii.
Now 13 children all of them more than 2 years post that dosing and we have seen no safety.
Safety events whatsoever with those children of any concern and then also for children would seal and 3 and again.
1 of them, even at a higher dose so with the interests equal delivery of the low dose of AAV 9 that we're using their we've not seen any safety considerations and I was just remind everybody to that part of the premise for Amicas US involvement with gene therapy was the notion that with our protein engineering technologies together with and.
Bradley L. Campbell: And that's really what gives you the leverage to be able to use one commercial organization to support both programs. And we've also taken the approach of really hiring more key account managers. So somebody's used to supporting an entire academic center, not just one specific physician, in terms of how they help support the understanding and utilization of these products. So we feel really good about that leverage and, again, see very little increase in the commercial infrastructure that we'll need to support the launch of ATGA.
Next generation technologies, and gene therapy coming from Jim Wilson's lab that our collaboration looks to address exactly the problem of safety, particularly with these AAV campuses. So we think actually will be very favorably positioned we continue to have concerns and the field broadly around high dose systemic.
Operator: And we have your next question coming from the line of Anupam Rama from J.P. Morton. Please go ahead.
Anupam Rama: Hi guys, thanks so much for taking the question. A quick one on Gallifold on the new patient.
[noise] AAV, we think that does present complications and dangers for patient and so if we think it's terrific that the F. D. A is taking this on to review and we would expect to be actively engaged and in those discussions.
Anupam Rama: on the new patient international ads in June being the second best since launch. Is this really being driven by deeper penetration into the region or maybe new country ads that you saw in the first half of 2021? Thanks so much.
But I think again very very differentiated from the Amicas approach here.
Jeff from Mitch feel free to comment as well.
John you covered it pretty well I don't have anything specific that.
John Crowley: Yeah, Anapum, thanks for the question. Good to hear your voice. I'll turn it to Bradley, just to highlight what you'd mentioned June.
This is Jeff.
And I'll, just say that and we welcome and you have to be a advisory.
Committee to look into these things to provide.
John Crowley: It was actually through this, it was in the second quarter that we saw the most significant number of new patient starts, second most significant since product launch. But Bradley, do you want to comment further, drill down a little bit on the dynamics that we're seeing that's driving that? Yeah, so Anapom, good question, again, good to hear from you.
Color to the field us evolves and gives us output that we can incorporate into art and for a clinical thinking and the safety of patience is paramount. So we really look forward to that and put it on the.
The meeting.
Create and again give I'll just direct you to some of the work that we've published and discuss now publicly with respect to our February and pump pay gene therapy programs utilizing Avi technologies and systemic delivery, we think again with our unique ability to engineer the trans genes that we not only may have been.
Bradley L. Campbell: So I think it's a few things. First of all, it is some new countries. You know, central and eastern Europe, for example, has been later in the reimbursement cycle, and we're starting to see those countries come on board. I think we talked about Poland as an example, which is a great new opportunity for patients there. But it's also seeing patients come on in our existing larger markets, Germany, the UK, etc. During COVID, there were some, you know, small cues here.
Sensually better efficacy and much better ability to manufacture those gene therapies, we actually hope that we can deliver them at a much safer dose as well lower dose and that's what we've demonstrated preclinically.
Okay. Thank you we have your next question from she was a smart from SBB, leaving your line is open.
Hi, Andrea and for John Thank you for taking our questions. So assuming a T G E a N.
Bradley L. Campbell: and there are patients that were disrupted in their ability to get into the hospital. And so I think there's some of that going on. But it's also increasing penetration into naive patients, so it's reflective of market growth. So we think those are all great signs of the underlying business, and it's a combination of a number of factors that, again, we think should continue as you go into the back half of this year and beyond as well.
Oh, how eager R and patients currently on therapy and looking for alternative treatments.
And you believe can be early adopters and and T J, a and how old London or the opportunities for these patients to switch to therapy switch to switch therapy and now that there could be several treatment options available for for these patients any color and that would be helpful. Thank you.
Sure on day, I'll ask Bradley to comment as well from us to kind of a patient demographic and market perspective, but just to remind everybody people living with Pompeii disease, whether they're instance, all the way through late adulthood for 15 years have had the option of only 1 approved and as a replacement therapy. So we think it's terrific that they'll have the potential.
Operator: Thank you, and we have your next question from De Gonha from Cipolls. Your line is open.
Salveen Jaswal Richter: Good morning, thanks for taking my questions. Just one follow-up to an earlier question.
Salveen Jaswal Richter: up to an earlier question and another follow-up for me. Regarding the regulatory side of ATGAA, I'm not too familiar with the early access to medicine scheme in the UK, but just looking at the language and the press release, so I was wondering if there is so much, I guess, overlap between the EU and FDA. Can you maybe speak to how we should think about the exposure to glucose to alpha for two years or more?
<unk> ahead very shortly to have potentially multiple options for new next generation therapies again, we think a T. G. E. A is demonstrated overwhelmingly clinically meaningful and significant benefits for these patients. So you'll see a hunger and a great need and the patient community and with the physicians as well.
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Bradley if you want to talk about how we see the the patient population demographics speak to the early adopters that would be very helpful.
Salveen Jaswal Richter: And then secondly, I guess going back to the biology of ATB-200, the M6P modification, maybe a question for Jeff. Can you speak to the degree of M-6-P modification on this biologic and whether you think having a better modification profile will produce an even better clinical outcome? Thank you.
Sure. Thank you day.
Sorry about that switching over so we can avoid the echo there Hum John I think you've hit the important highlights from from a need for alternative treatments for the patient population.
Couple of other important points to make 1 thing of course is that the market today is about about.
About 3000, or so patients treated today, we estimate and that's the 1.1 billion dollar.
Revenue globally for women's on so that's a huge population of potential switch patients. It is growing at high single digit rates the market's so's continuing to bring on some naive patients as well.
John Crowley: I'll just comment that to remind everybody, back in May, we announced that the MHRA had granted early approval to ATGA for patients who were treatment experienced who had been on for more than two years. That was the initial indication, and again, through the process, we would expect that to be for all switch patients, as well as for naive patients as well. The reality is that the vast majority of patients in the United Kingdom on the current approval of ERT have been on it for two or more years.
Importantly, too we have today over 150 patients on a T. G E and we would expect as we depending on the utilization through various extended access and further clinical development program that number could even grow and so when we launch this product or we anticipate launching this product you will have a significant basic clinical trial patients.
To convert over and we think that gives you a lot of of momentum coming into the launch and and the last thing I'll I'll offer us something with John touched earlier. Unlike when we launched scallop hold which was a very successful launch, but we were building our commercial organization from scratch at that time here, we have a ah well experienced global commercial team.
John Crowley: So we think there's significant opportunity prior to full and final approval in the United Kingdom to offer ATGAA as requested to many, many patients on the current standard of care. We've seen great momentum and enthusiasm since that announcement just a couple of months ago. There really continues to be a significant unmet need for that particular Pompeii population, which is now working its way through the local health authorities.
Already as I mentioned before covering many of the same centers and many of the same physicians who are treating pompeii disease.
And will present in the.
The major markets around the world and so we have a great experience team of commercial and and medical.
Passionate entrepreneurs focused on getting our products out to patients. So we think we've got a great basis to start from and and should have great momentum going to watch.
Thank you and our last question comes from the line of free to put on some college and your line is open.
John Crowley: And based on feedback and proactive requests from physicians, we would expect in the months ahead to see significant numbers of requests for patients to be switched from current DRT to the ATGA product. With respect to the M6P modification, I'll ask Jeff to comment as well. But again, what we've developed at Amicus is a novel biologic known as ATB 200. It is a naturally occurring cell line that we have selected. cultivated, and now, with our partners at Wushi Biologics, scaled to commercial scale, maintaining the proper glycosylation, we think optimal glycosylation, and very high levels of naturally occurring manosix phosphate, including the carbohydrate structure that is bisphosphorylated.
Hi, guys. Thanks for taking a follow up.
Back on sale and I want the battens program and general.
I wanted to round back on FTA interactions I believe you are going to have a meeting.
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Yeah of course for 2 again, we continue to put great effort into the lead programs that we expect to enter a pivotal study us for both C. L and 6 and of course, the very large battens indication and C O and 3 it's been a series of interactions focus both on CMC again, a lot of our activities for the last year and a half really.
John Crowley: And in all of our in vitro studies, preclinical studies, and now consistent with the clinical data, we've seen significantly higher uptake of the protein, and again, a protein with a naturally occurring carbohydrate structure that is highly phosphor-related, that has the ability to not only get into multiple muscle cells, but from what we've seen preclinically, certainly we see that, and I think it will continue now with the clinical data, And with that, we think it's a very differentiated product again with the enzyme stabilizer, the, the, the, the, um, chaperone product.
And have been focused on manufacturing, including the tech transfer from the University the scale up and with now completed GMP manufacture with our partners at grammar thermal Fisher lot of work on the assays, including the potency assay is to make sure that we have all the analytical tools, we saw the field trending toward that and the regs.
Elaters and particular, leading people in that direction as early as the fall of 2019. So I think we were ahead of the curve there with that and we've also had interactions on our clinical data and on our thinking and the regulators feedback on future studies those continue to be ongoing.
But maybe Jeff and Mitch if you want to give us some idea of broadly how we're thinking about those pivotal studies for the lead batten programs. Please.
John Crowley: So together we think that's a very novel, novel product for people living with Pompeii, and we think it has an optimal level of M6P, again, not post-translationally modified, but with the cell line that we've selected and cultivated. Jeff, if I missed anything, please feel free to add. Yeah, sure. Hi, Dagon.
Sure I'll start first mentioned and higher too so.
John mentioned, we continue to be focused on all of the enabling CMC work and and our regulatory interactions across different topics as per the current thinking on the next clinical studies first day would be done with the thermo G&P commercial process material for.
Brazil and fix we believe it would be a study similar to what we did and phase 1 too and in terms of.
General Sigh US single arms study vs natural history focused on Hamburg Motor language. So we feel really confident about our ability to.
Jeffrey P. Castelli: As John said, we know that ATB 200 has significantly improved the fossil relation in M6P. In fact, the vast majority of all the protein molecules have an M6P molecule. On average, there's at least one bis M6P for every single ATB 200 molecule. So we think that we've really managed to create something that is pretty optimally targeted. We don't think that having additional M6P would notably or meaningfully change our uptake. We sort of maxed out. Oh, sorry.
And succeed on that study in terms of design power based on what we've seen already and our phase 1 too.
And for sale on 3 of course, our initial phase 1 to study was only the 4 patients that was all the material and we had from nationwide.
We would expect a much larger next study.
And the several dozens of patients again, we believe it should be a single arm study.
Compared to the very robust natural history data that exists and sale and 3 we believe that you'd be near us physical domain and the likely primary endpoint.
We think there's a lot of data with that and point and available.
But again of course discussions and agencies about all those key components control arm and points size of study et cetera.
Operator: Thank you, and your next question is from Salvin Richter of Goldman Sachs. Your line is open.
Mitch anything else to add a from a high level from your perspective.
Operator: Thanks for taking the question. This is Andrea on behalf of Selveen.
No just thanks, I think you're covered and well.
Great. Thank you you too.
Operator: Maybe one on your Baton disease program. I'm just wondering when we can expect the next data and what you would look to understand from that data release. Yeah, Jeff, do you want to comment on the Baton program?
Yeah of course.
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Yes that is all the questions I would now like to try and the conference maps and Mister John Crowley, Chairman and C O for closing remarks.
Jeffrey P. Castelli: Yeah, so the last time we provided an update on the 13 kids treated with CLN6 and the four treated with CLN3 was at the World Licizizome Disease Meeting earlier this year. That data set included aid patients who had reached two years of follow-up in CLN6, and it was one-year data for the first three kids treated with CLN3. That data continued to show disease stabilization, the Hamburg Motor Language, in CLN6, the UBDRX physical domain for CLM3, compared to what we've seen in natural history, which is really encouraging.
Great. Thanks, again, operator, and thank you all for listening and did a great question. It was and incredibly strong second quarter for Amicas and you can see across the execution of all of our programs and we look forward to a very full and exciting second half of the year. Thank you everybody have a good day.
Ladies and gentlemen, Complicities conference call. Thank you and have a great day.
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Jeffrey P. Castelli: We will continue, and we do continue to follow those kids. We will have additional updates, you know, as we have a significant amount of new data. That could happen, you know; we haven't specifically said when, but typically, key venues where we do provide updates are either at the Child Neurology Society meeting in the fall or at the World Neurology meeting early in the year. So those are two places where you can expect potential updates as we have more data to share. But we're really excited by what we've seen and will continue to see in this kid.
Operator: Thank you, and we have a question coming from the line of Yom Song from DTIG. Her line is open.
Operator: Thank you, and good morning. Thanks for taking the time to ask me a question. So, excuse me, on HGAA, I think you mentioned that some patients, after long-term treatment with lumazine, start to decline, losing response. And I believe the reason is still not very well understood, so I wonder, I know it's too early to tell, but is there any evidence to suggest that this observation might not happen to ATGA. And in your clinical study in the Pompeii study, did you, by any chance, enroll any patients who might have entered that declining phase after receiving an Zuma-Dine treatment?
John Crowley: Yeah, thank you, Yun. Maybe Jeff, if you want to field that, and Mitch, after Jeff, if you have any color to add, please.
Jeffrey P. Castelli: Sure, thanks, John. And, you know, what we've seen here now in multiple studies is that, you know, there's initial benefit on treatment with the current standard of care. We see patients improve on the six-minute walk, have improvements on FEC, and on their muscle function. It's really for about the first two years.
Jeffrey P. Castelli: And even that response can be somewhat variable. But what's then seen after two years is a pretty much continued decline, almost looking, in many cases, like it was pre-trial treatment.
Jeffrey P. Castelli: We do believe that with ATGA, addressing the improved M6P binding and uptake and also the stability in the blood with the stabilizer, that that will hopefully help to better penetrate deeper into tissues and clear glycogen that might not have been cleared, and that could be the cause of that continued progression. We are really encouraged by the results we've seen in our ERT-experienced patients, both in our Phase 1-2 study and now here in Propel, where those patients had been on treatments for, on average, over seven years and well into that stage where they're nearly all expected to be declining, and we see that we can stabilize their SEC and actually lead to improvements in the six-minute walk in those patients.
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Jeffrey P. Castelli: So we do think that that not only goes really well for those ERT experience patients to offer them something that could change the trajectory of their disease, but hopefully, that reads through to naive patients longer term when they become more akin to a switch patient. We hopefully will see it continue to stay and benefit those patients. Yeah, this is Mitch. I'll just add that I think what Jeff said during the call was important to reiterate the majority, 117 patients went on to the extension study. We'll continue to follow them, look at regular data cuts, and release those at appropriate venues.
Jeffrey P. Castelli: And to point out individual heterogeneity, we also think that's important. And again, we look at every patient that's ongoing in that trial and look to find out how they're doing. And again, we'll report that data on an ongoing basis.
Operator: Thank you. And next, we have a question from Gil Bloom's line from Needham Company. Your line is open.
Operator: Thank you for taking my questions and congratulations on the execution. Maybe a bit of a question on the Baton disease program. Do you guys have any thoughts about this upcoming ADCOM in September, which will discuss safety and AV-9 therapeutics? Thank you. Yeah, great.
John Crowley: Thank you, Gil. We think that'll be an important meeting. I'll just remind everybody that for our Batten's program, for CLN6, we've dosed 13 children, all of them more than two years post-dose, and we have seen no safety events whatsoever with those children of any concern, and then also four children with CLM3. And again, one of them was even at a higher dose.
John Crowley: So with the intracadical delivery of the low dose of AAB 9 that we're using there, we haven't seen any safety considerations. Now, just remind everybody, too, that part of the premise for Amicus' involvement in gene therapy was the notion that with our protein engineering technologies, together with the next generation technologies and gene therapy coming from Jim Wilson's lab, our collaboration looks to address exactly the problem of safety, particularly with these AAV capsids.
John Crowley: So we think it will actually be very favorably positioned. However, we continue to have concerns in the field broadly around high-dose systemic AAV. We think that does present complications and dangers for patients. So we think it's terrific that the FDA is taking this on to review it, and we would expect to be actively engaged in those discussions. But I think this is, again, very differentiated from the amicus approach here. Jeff or Mitch, feel free to comment as well.
Jeffrey P. Castelli: John, you covered it pretty well. I don't have anything specific to add. This is Chats.
Jeffrey P. Castelli: And I'll just say that we welcome the FDA Advisory Committee to look into these things to provide, you know, color to the field as it evolves and gives us output that we can incorporate into our clinical thinking, and the safety of patients is paramount. So we really look forward to the output of the meeting. Great, and again, Gil, I'll just direct you to some of the work that we've published and discussed publicly with respect to our Febre and Pompeii gene therapy programs, utilizing AIV technologies and systemic delivery.
Jeffrey P. Castelli: We think, again, with our unique ability to engineer the trans genes, that we not only may have potentially better efficacy and a much better ability to manufacture those gene therapies, but we actually hope that we can deliver them at a much safer dose as well, a lower dose. That's what we've demonstrated preclinically.
John Crowley: Thank you. We have your next question from Joseph Swartz from SBB Learing. Your line is open. Hi, I'm Jerry Daly and this is for Joe.
Operator: Thank you for taking our question. So, assuming ATGA is approved, how eager are the patients currently on therapy looking for alternative treatment? Who do you believe could be earlier adopters of ATGA, and how abundant are the opportunities for these patients to switch, to switch therapies now that there could be several treatment options available for these patients? Any color and that would be helpful. Grange, I'll ask you.
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Operator: Sure, Andre. I'll ask Bradley to comment as well from a kind of patient demographic and market perspective, but just to remind everybody, people living with Pompeii disease, whether in their infants all the way through late adulthood, for 15 years have had the option of only one approved enzyme replacement therapy. So we think it's terrific that they'll have the potential ahead very shortly to have potentially multiple options for new, next-generation therapies.
Operator: Again, we think ATGA has demonstrated overwhelmingly clinically meaningful and significant benefits for these patients, so you'll see a hunger and a great need in the patient community and with the physicians as well for new, novel next-generation therapies. So Bradley, if you want to talk about how we see the patient population demographics, speak to the early adopters, that would be very helpful. Sure, yeah, John, I think you've had a, Sorry John, I think you hit the important highlights from a need for alternative treatments for the patient population. There are a couple of other important points to make.
Operator: One thing, of course, is that the market today is about 3,000 or so patients treated today, we estimate, and that's the $1.1 billion revenue globally for Lumazine. So that's a huge population of potential switch patients. It is growing at high single-digit rates, and the market will continue to bring on some naive patients as well. Importantly, too, we have today over 150 patients on ATGA, and we would expect, as we, depending on the utilization through various expanded access and further clinical development programs, that number could even grow.
Operator: And so when we launch this product, or we anticipate launching this product, you'll have a significant base of clinical trial patients to convert over, and we think that gives you a lot of momentum going into the launch. And the last thing I'll offer is something that John touched earlier, you know, unlike when we launched Gallifold, which was a very successful launch, but we were building our commercial organization, you know, from scratch at that time.
Operator: Here we have a well-experienced global commercial team already, as I mentioned before, we're covering many of the same centers and many of the same positions who are treating Pompeii disease, and we're present in the major markets around the world. And so we have a great experience team of commercial and medical, passionate entrepreneurs focused on getting our products out to patients. So we think we've got a great base to start from and should have great momentum going into the long run.
Bradley L. Campbell: Thank you, and our last question comes from the line of Rita Burrell from Koweth. Your line is open. Hi, guys, thanks for taking the follow-up. Just back on CLN,
Operator: Well, the Batons program in general. I wanted to round back on FDA interactions. I believe you were going to have a meeting recently,
Ritu Subhalaksmi Baral: or soon, and can you just address the current thinking on the strategy for proposed
Ritu Subhalaksmi Baral: for proposed pivotal trials around CLN6, CLN3 and the other batons that your Yeah, of course, for two, again, we continue to put great effort into the lead programs that we expect to enter pivotal studies for both CLN6 and, of course, the very large indication for CLN3. It's been a series of interactions focused both on CMC. Again, a lot of our activities for the last year and a half, really, have been focused on manufacturing, including the tech transfer from the university, the scale up, and we've now completed GMP manufacturing with our partners at Brammer. thermal fissure
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John Crowley: A lot of work on the assays, including potency assays, to make sure that we have all the analytical tools. We saw the field trending toward that, and the regulators, in particular, leading people in that direction as early as the fall of 2019. So I think we were ahead of the curve there. With that, we've also had interactions with our clinical data and our thinking, and the regulators' feedback on future studies.
John Crowley: Those continue to be ongoing. But maybe Jeff and maybe Jeff and if you want to give some idea broadly how we're thinking about those pivotal studies for the lead baton programs, please. Sure, I'll start first, Mitch and higher two.
Jeffrey P. Castelli: As John mentioned, we continue to be focused on all the enabling CMC work and our regulatory interactions across different topics. As for the current thinking on the next clinical studies, first, they would be done with the thermo-GMP commercial process material. For CLN6, we believe it would be a study similar to what we did in phase 1-2 in terms of general size, single-arm standard, versus natural history focused on Hamburg Motor Language.
Jeffrey P. Castelli: So we feel really confident about our ability to succeed in that study in terms of design and power, based on what we've seen already in our case 1-2. For CLN3, of course, our initial phase 1-2 study was only four patients. That was all the material we had from nationwide.
Jeffrey P. Castelli: We would expect a much larger neck study in several dozens of patients. Again, we believe it should be a single-arm study compared to the very robust natural history data that exists in CLM3. We believe the UBDRS physical domain is the likely primary endpoint because we think there's a lot of data with that endpoint available. But again, of course, discussions with agencies about all those key components, the control alarm, endpoints, size of the study, et cetera.
Jeffrey P. Castelli: Mitch, anything else to add from a high level from your perspective? No, Jeff, thanks. I think you covered it well. Great, thank you, Richard. Yeah, of course. So, operator, I think that's all the questions we show in the queue, correct?
Operator: Yes, that's all the questions. I would now like to turn the conference back to Mr. John Crowley, Chairman and CEO, for closing remarks.
John Crowley: Great, thanks again, Operator. Thank you all for listening and for the great questions. It was an incredibly strong second quarter for Amicus, and you can see the execution of all of our programs, and we look forward to a very full and exciting second half of the year. Thank you, everybody. Have a good day.
Operator: Ladies and gentlemen, this concludes today's conference call. Thank you, and have a great day. Theeus
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