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Amicus Therapeutics Inc (NASDAQ:FOLD)
Q4 2019 Earnings Call
Mar 2, 2020, 8:30 a.m. ET

Contents:

  • Prepared Remarks
  • Questions and Answers
  • Call Participants

Prepared Remarks:

Operator

Good morning, ladies and gentleman, and welcome to the Amicus Fourth Quarter and Full-Year 2019 Results Conference Call and Webcast. [Operator Instructions] As a reminder, this conference call is being recorded.

I would now like to turn the conference over to your host, Mr. Andrew Faughnan, Director of Investor Relations. You may begin.

Andrew Faughnan -- Director of Investor Relations

Good morning. Thank you for joining our conference call to discuss Amicus Therapeutics' full-year 2019 financial results and corporate highlights. Speaking on today's call, we have John Crowley, Chairman and Chief Executive Officer; Bradley Campbell, President and Chief Operating Officer; and Daphne Quimi, Chief Financial Officer. Also joining for Q&A are Dr. Jay Barth, Chief Medical Officer; Dr. Hung Do, Chief Science Officer; and Dr. Jeff Castelli, Chief Portfolio Officer and Head of Gene Therapy.

As referenced on Slide 2, we may make forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business as well as our plans and prospects. Our forward-looking statements should not be regarded as representation by us that any of our plans will be achieved. Any or all of the forward-looking statements made on this call may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. You are cautioned not to place undue reliance on any forward-looking statements, which speak only to the date hereof.

All forward-looking statements are qualified in their entirety by this cautionary statement and we undertake no obligation to revise or update this presentation and conference call to reflect events or circumstances after the date hereof. For a full discussion of such forward-looking statements and the risks and uncertainties that may impact them, we refer you to the forward-looking statements and risk factors section of our Annual Report on Form 10-K for the year ended December 31, 2019 to be filed later today with the Securities and Exchange Commission.

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

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Great. Thanks, Andrew. Good morning, everybody, and welcome to our full-year 2019 results conference call. I'll begin here on Slide 3. I'm pleased to highlight the tremendous progress that we've made at Amicus throughout 2019, and now into the start of 2020. In early January, we laid out several key elements and accomplishments for Amicus through 2019 and ultimately bringing us to where we are today in 2020, building one of the next great biotechnology companies poised to impact people around the world who are living with rare diseases.

For Amicus, 2019 was another significant period of growth and execution across our science, clinical, regulatory and commercial efforts. I'd like to take a moment to highlight just a few of them. I'll begin with Galafold. Galafold continues its strong global launch and remains the cornerstone of our success. With $182 million in full-year revenue for 2019, this is the second year in a row that we've exceeded the top end of our revenue guidance. For AT-GAA, again, that's our next generation enzyme replacement therapy for Pompe disease and the crown jewel of our portfolio. This continues with significant momentum toward completion of the Phase 3 PROPEL trial and the potential approval and to becoming what we believe will be the new standard of care for people living with Pompe disease.

Also after its strategic review of our business in the second half of last year, we continue to carefully shepherd our financial resources and we reiterate our cash runway, leading us well into 2022, as we continue on a path to profitability. And finally, and very importantly, in 2019, we advanced what we believe to be among the most significant collection of programs, capabilities and collaborations in the field of gene therapy.

Including in 2019, the significant extension of our collaboration with Dr. Jim Wilson and the University of Pennsylvania, as well as important clinical data that we shared in our CLN6-Batten disease program. Amicus is the most significant collaborator with the Wilson Lab and UPenn. We believe that this gene therapy partnership with UPenn and Dr. Wilson provides us with a tremendous research engine for the future growth of Amicus.

Turning now to Slide 5, our key strategic priorities for 2020. We have five key strategic priorities this year and we are well on track to achieving all of them. First, with Galafold, our precision medicine for Fabry. We will continue to drive Galafold to more patients living with Fabry disease with amenable variance in existing and in new markets. We look to achieve global product revenue this year of $250 million to $260 million. In 2020, we are off to a strong start thus far and all key metrics are tracking in all key geographies, which give us further confidence in our guidance for this year.

Second, we are increasing the clinical, regulatory, manufacturing and pre-commercial activities surrounding our Pompe program as we advance AT-GAA toward approval.

In 2020, we are set to complete the Pompe Phase 3 PROPEL study. We are set also to enroll the pediatric studies as well as advance manufacturing to support a 2021 BLA and MAA filings. Importantly, this year, we plan to apply for and initiate a rolling Biologics License Application or BLA for AT-GAA with full clinical results in the first half of 2021 to support a full approval under the Fast Track Designation for this breakthrough therapy designated program.

Third, we are advancing our industry-leading rare disease gene therapy portfolio from our new Global Research and Gene Therapy Center of Excellence in Philadelphia. We will be advancing the clinical development, manufacturing and regulatory discussions for both our CLN6 and CLN3 Batten's program.

Fourth, in addition, we are progressing our Pompe gene therapy toward IND and we'll disclose up to two additional IND candidates this year. A lot of work is under way with our manufacturing partners for the manufacturer and scale up of the Pompe gene therapy getting us ready to begin clinical studies we believe in 2021 as well as our other potential IND candidates. And we look forward to sharing the additional IND candidates from our UPenn collaboration again later this year.

And again, we will continue to maintain a strong financial position as we carefully manage our expenses and our investments. Our cash runway is expected to lead us well into 2022, as we continue on a path to profitability, and we are fully funded through all major milestones.

On a final note before I hand it over here in a moment to Bradley, Amicus has been very conscious of our global supply chain and even well before the emergence of the coronavirus. There has been no impact on our supply chain for any of our products and no impact on import or export of our products or essential raw materials.

We will continue to monitor the situation closely, of course. Specifically, Galafold sales remain on track for the year with adequate supply on hand.

With respect to the manufacturer of AT-GAA for Pompe, we and our partners at WuXi Biologics have a very robust risk mitigation plan in place, and there has been no impact to WuXi's operations in China. Importantly, we have enough supply on hand stored at our distribution center in Ireland to support the entire Phase 3 PROPEL study and beyond.

So with that introduction, let me now turn the call over to Bradley Campbell, our President and Chief Operating Officer to highlight the Galafold performance for the year.

Bradley L. Campbell -- President and Chief Operating Officer

Great. Thanks, John. Good morning, everyone. Let me begin by providing more color on the continued growth of Galafold for the full-year of 2019. I'll begin on Slide 7 with a global snapshot of the Galafold launch. For 2019, total product revenue was $182.2 million, which is an increase in revenue of $91 million over full-year 2018. Galafold metrics were driven by exceptionally strong momentum in new countries, including the US and Japan, as well as steady growth in our large early launch countries and our small and mid-sized countries as well.

Specifically, the revenue breakdown last year was $129.8 million or about 71% of revenue generated outside of United States and the remaining $52.5 million or about 29% coming from within the US over the full year.

I do want to take an extra minute here to touch on our global commercial reach that we've been building. We now have over 40 countries around the world with regulatory approvals for Galafold, including Argentina, Brazil, Colombia and Taiwan, which were all approved last year.

We have commercial sales in nearly 30 of those countries with more on horizon this year. And we have an incredibly passionate and experienced team working tirelessly to bring our medicines to patients around the world. This expanding global footprint is important not just to support the continued growth of patients with access to Galafold, but it lays an incredibly strong foundation, which is highly leverageable to support the launch of Pompe and our future products as well.

Turning now to Slide 8. I'll recap the performance over 2019. As John mentioned, on the right hand side of the slide, you can see the 2019 sales nearly doubled, even including a 5% negative impact from foreign exchange. If you look at that from purely an operational performance perspective, not including FX, we actually more than doubled our sales last year, finishing with well over a 1,000 patients on Galafold worldwide.

On the left hand side of this slide, you can see our quarterly performance, which again was very strong in Q4. And again, I think, this illustrates the phenomenon we've seen over the past few years that while the quarter-on-quarter growth is relatively non-linear, it's consistently strong and we'd expect to see a similar pattern this year.

Now on Slide 9. With three years of performance behind us, we can confidently say, we're on a path to that $500 million sales opportunity in 2023. And as I outlined late last year, to get to that $500 million, we expect a five-year compound annual growth rate of about 40% from 2018 to 2023. And we expect to generate $1 billion in cumulative revenue between 2020 and 2022 alone, that goes a long way toward funding our R&D and OpEx over that period.

We also have even further confidence in the $1 billion revenue opportunity at peak for Galafold as we continue to see significant growth in the Fabry market globally. In fact, the global Fabry market for all therapeutics surpassed the $1.5 billion mark in 2019, driven by continued diagnosis from high-risk screening, newborn screening and other diagnostic initiatives, which we're also investing in as well.

And as a reminder, we have orphan exclusivity in the US and Europe, which alone take us to the end of the 2020s, in addition to our Orange Book-listed patents that give us IP coverage into the late 2030s. So a lot of opportunities to provide access to Galafold globally for a long period to come.

And in the meantime, let me just reiterate that 2020 has gotten off to a great start with all of the commercial metrics, including our switch and naive metrics, continued market share growth and continued high adherence and compliance all on track to deliver our guidance of $250 million to $260 million in global Galafold revenue.

So with that, let me turn the call back to John to discuss our program updates for AT-GAA and Pompe. John?

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Great. Thank you, Bradley. I will begin on Slide 11 with AT-GAA for Pompe disease, again, the first ever second generation therapy as well as the first therapy for Pompe disease ever to receive Breakthrough Therapy Designation or BTD. There is tremendous momentum behind for what we believe may be the next standard of care for a broad population of people living with Pompe disease, a product representing a potential $1 billion to $2 billion peak annual revenue opportunity.

We are set to complete the Pompe Phase 3 PROPEL study, enroll the pediatric studies and advance manufacturing to support 2021 BLA and MAA filing. It is our plan to apply for and initiate a rolling BLA in the first half of 2021 to support full approval under the Fast Track Designation.

Two new updates this morning with respect to AT-GAA are, first, in response to the many request for compassionate use that we have received for children with infantile-onset Pompe disease, it is our plan this year to offer an Expanded Access Program to those in need. Second, our PPQ runs are nearing successful completion with our key strategic partner WuXi biologics, and will serve as the foundation for the Chemistry, Manufacturing and Control or CMC module for a Biologics License Application or BLA submission. Importantly, we have successfully completed now all three of the bioreactor or upstream production runs under the PPQ process at WuXi.

This was a huge step forward and a major derisking event for this program.

As a reminder, the pivotal PROPEL study together with additional data that we've collected in the Phase 1/2 study will support, we believe the full approval of AT-GAA. We continue to be extremely excited about AT-GAA as well as our preclinical Pompe gene therapy program to build what we believe could be the largest and most valuable franchise in the industry with the potential to offer solutions to all patients living with Pompe disease globally.

So with that, I'd like to turn the call over now to Daphne to review our financial results, guidance and outlook. Daphne?

Daphne Quimi -- Chief Financial Officer

Thank you, John, and good morning, everyone. Our financial overview begins on Slide 14, with our income statement for the full-year ending December 31, 2019. For 2019, we achieved Galafold revenue of $182.2 million, which is 99% increase over the full-year of 2018. This includes year-over-year operational revenue growth measured at constant currency exchange rates of 105%, offset by a negative currency impact of approximately 5%.

Cost of goods sold as a percentage of net sales was 12.1% in 2019 as compared to 15.8% for the prior year. Cost of goods sold as a percent of revenue was favorable, as Galafold revenue continues to grow in the United States where we do not owe royalties as well as other countries where we are subject to lower royalties.

We continue to make significant investments in R&D and manufacturing with the ongoing pivotal study and commercial scale up in our Pompe program as well as the expansion of our gene therapy portfolio and capabilities. During 2019, we reported $286.4 million in R&D expense as compared to $270.9 million for the prior year period. The increase is primarily due to continued investments in the Galafold launch, Pompe clinical study program and our gene therapy pipeline offset by an upfront payment of $100 million for an asset acquisition that occurred in 2018.

Total selling, general and administrative expense for 2019 was $169.9 million as compared to $127.2 million for the prior-year period. The increase represents the expanded geographic scope of the ongoing Galafold commercial launch, including launch activities in Japan and the United States.

On a non-GAAP basis, total operating expenses were $411.8 million in 2019, which was at the lower end of our guidance. This was as a result of careful expense management, while also advancing all of our key programs. We define non-GAAP operating expense as a research and development and SG&A expenses, excluding share-based compensation expense, changes in fair value of contingent consideration and depreciation.

Net loss for 2019 was $356.4 million, or $1.48 per share as compared to a net loss of $349 million, or $1.88 per share in the prior year period. As of December 31, 2019, we had approximately 255 million shares outstanding.

Turning now to Slide 15. As John mentioned, we are fully funded into 2022 through our major milestones in our portfolio and continued global growth. As part of our normal course of business and taking into account all of the new programs that we have integrated into our organization, last year we completed a strategic business review to identify our top priorities and areas of investment to drive efficiencies and cost savings, while advancing all of our key programs.

We are taking the following actions that extend our cash runway.

We will continue to support Galafold revenue growth and have increased confidence around a higher growth trajectory. Through our internal teams and contract manufacturing partners, we have identified synergies and efficiencies with gene therapy development and manufacturing. We also expect to take a phased approach to build out Amicus facilities, internal manufacturing and other capital expenditures.

We plan to continue our prudent gene therapy portfolio management process to support one to two INDs starting in 2021 and beyond. And as we grow, we are leveraging internal resources and external collaborators for measured internal headcount growth through 2022. Going forward, again, to emphasize, we expect total non-GAAP operating expenses in 2020 to remain relatively flat as we leverage the global commercial infrastructure that is already in place for the AT-GAA launch and other products in our pipeline. And we transition the costs associated with development of AT-GAA to multiple gene therapy programs in our pipeline.

A few comments about our current cash position and 2020 financial guidance. Cash, cash equivalents and marketable securities were $452 million at December 31, 2019 as compared to $504 million at December 31, 2018. Looking at 2020, we are reaffirming our full-year Galafold revenue guidance of $250 million to $260 million, in addition to our non-GAAP operating expense guidance of $410 million to $420 million.

And with that, let me turn the call back over to John for updates on our next-generation gene therapy platform.

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Great. Thank you, Daphne. I'll move now to Slide 18 and highlight here our industry-leading portfolio of gene therapies for rare diseases. Starting with our Batten disease franchise, we have now received US and EU orphan drug designation for both CLN6 and CLN3 Batten disease, which together with our CLN1 and CLN8 programs provide a robust Batten disease franchise that combined may represent $1 billion plus in peak recurring annual revenue.

In CLN6, we have reported positive interim data in our clinical study that demonstrates meaningful impact of our AAV gene therapy in this devastating form of Batten disease. We have just recently initiated the long-term follow-up of these initial participants, these children in the CLN6 Phase 1/2 study, and this year we plan to advance regulatory discussions to finalize the clinical and regulatory path forward for this program.

The initial CLN6 results provide important read through for our clinical study in CLN3 Batten, the most common form of childhood neurodegeneration, where we have safely dosed the initial cohort in addition now to patients in our higher dose cohort. Our plans this year to advance regulatory discussions and to finalize clinical and regulatory paths in CLN3 and report initial data later this year.

On slide 19, I'd like to remind you of the research collaboration with the University of Pennsylvania and the Dr. Jim Wilson Lab, which will be an important driver of growth for Amicus in the future. This collaboration with the Wilson lab at Penn combines Amicus's protein engineering and glycobiology expertise with Penn's gene transfer technologies to develop novel gene therapies designed for optimal cellular uptake, targeting, dosing, safety and manufacturability. As part of this collaboration with UPenn, Amicus has rights to 50 plus diseases, including eight currently in active preclinical programs.

As highlighted on slide 20, our most advanced program from the Penn collaboration is in Pompe disease.

This is moving now into IND-enabling studies with the potential to enter the clinic in 2021. We expect additional preclinical data out of this collaboration this year in multiple programs and we are guiding to the disclosure of up to two additional IND candidates by the end of this year.

So, before we turn the call over to Q&A, I'd just like to focus again on people living with rare diseases. We are fighting to bring new hope and alleviate an enormous amount of suffering for many people with rare diseases and for their families. And we are here to deliver on our mission for these patients, while creating significant value for our shareholders.

So operator, with that, happy to take any questions.

Questions and Answers:

Operator

[Operator Instructions] Your first question is from Ritu Baral with Cowen.

Ritu Baral -- Cowen -- Analyst

Good morning, everyone. Thanks for taking the question. I wanted to ask about what is left to complete data wise before you start -- rolling BLA? Do you have any outstanding preclinical data for the preclinical module or is that pretty much set to go? And as far as the pediatric study and requirements, is that required for approval or is that something that you're aiming for your desired label? Thank you.

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Yes. Thank you, Ritu, to the last part of that question on the pediatrics, that's our own initiative that's not required for approval. That's, we think, important to get this drug into children, we've now been dosing children in the 12 -- age 12 to 17 cohort that pediatric study is under way. We expect now to add additional patients below that age 12, all the way down to the expanded access programs that we will initiate here shortly for the infantile-onset Pompe disease. So not necessary for the full approval, but we think important for the label and important to get it to as many patients as quickly as we can.

In terms of what needed to begin the rolling BLA, there is nothing else of note that would be needed for the preclinical module. So we're in a really good position there. And then, of course, the CMC module is dependent on completing successfully all of those downstream activities for the PPQ runs. And again, just to remind and reiterate, we have now successfully completed all three bioreactor runs, all the upstream work which was a major, major undertaking for our team and the WuXi team and really pleased that that's been done successfully.

Ritu Baral -- Cowen -- Analyst

Great. Thanks.

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Very welcome.

Operator

Your next question is from Anupam Rama with J.P. Morgan.

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Hi, Anupam, if you're talking, we can't hear you.

Anupam Rama -- J.P. Morgan -- Analyst

Can you hear me now?

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Now we can. Yes.

Anupam Rama -- J.P. Morgan -- Analyst

So, I've gotten this question in the last week or so. But are there any plans to update the Phase 1/2 Pompe trial in 2020 or the next Pompe update going to be PROPEL? Thanks so much.

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Yes, I'll let Jay speak to that.

Jay A. Barth -- Chief Medical Officer

We don't have plans right now to update the Phase 1/2 study. At this point, we read out the two-year results, which look very good, of course, as everyone knows and now we're very focused on completing the Phase 3 trial and getting that out as soon as we can.

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Yes. But just to remind everybody, all patients continued. Nobody discontinued. Everybody continues. Nobody has gone back to Lumizyme in that study Lumizyme before [Phonetic]. So anecdotally we continue to hear positive reports from patients having a good experience on AT-GAA. So, again, to Jay's point, we've completed the two-year extension study and now focused on encapsulating that for the purposes of the BLA and then also collecting significant additional data over the next year for the complete BLA for full approval.

Operator

Your next question is from Dae Gon Ha with SVB Leerink.

Dae Gon Ha -- SVB Leerink -- Analyst

Hi, good morning, guys. I hope you can hear me. My one question and congrats on all the progress. Just wanted to follow-up on the AT-GAA aspect. Specifically, as investors are anticipating the Neo-GAA data from Sanofi in the second quarter, John or maybe even Jay, I wanted to get your take on what are some of the internal scenarios that you have going for the outcomes? And what are some of the respective market opportunities that you're currently have modeling for those respective scenarios? Thanks.

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Yes. Again, I can't comment on somebody else's program, that data will have to stand or fall on its own, right. What I do know is that our program has been in the clinic now for -- we dosed our first patient in April of 2016. There are now more than 150 people living with Pompe on AT-GAA. We expect even more than that with the pediatric studies this year. Again, one major distinction aside from the difference in the drugs themselves and frankly the difference to date in all of the clinical data. The difference, of course, recognized by FDA with the Breakthrough Therapy Designation for our product, the Penn's designation in the UK. So I think we're really, really confident in AT-GAA to be very distinct. Again, another major distinction. We are studying a range of patients in our PROPEL Phase 3, including both switch patients who make up about 70% of the population with the PROPEL study, as well as naive patients in that Phase 3 study.

So we feel really, really good about where we are. We had no trouble enrolling our study. We did that with significant wait lists at number of the sites, again, more than 50 clinical sites on five continents for the PROPEL study. So we think we have as robust and as successful a program as we can have at this stage.

Operator

Your next question is from Debjit Chattopadhyay with H.C. Wainwright.

Aaron Welch -- H.C. Wainwright -- Analyst

Hi, guys, good morning. This is Aaron on for Debjit. Congratulations on all of the progress. So we noticed on a world poster that the Emax for the six-minute walk test increased with ADA titer. So that was unexpected. Do you have any interpretation for this? And how does this compare with Lumizyme? My next question is, with the recent..

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Hold on. Let me stop you. You have one question there. I'll answer that one. Jay, I'll let you [Speech Overlap].Yes, I mean, without getting into the technical aspect of your question, which I don't know if everybody appreciate, but having looked at the antibodies, something we present to world, four [Phonetic] in our Phase 1/2 study, there was no relationship between the antibodies and efficacy, safety or PK, including the six-minute walk test. So our results stand from our Phase 1/2 study is showing a very robust effect and efficacy for six-minute walk and that's why based on the Phase 1/2 results we're very confident in the Phase 3 data when that reads out.

Operator

Your next question is from Ellie Merle with Cantor Fitzgerald.

Ellie Merle -- Cantor Fitzgerald -- Analyst

Hi, guys. Thanks so much for taking the question. Just in terms of gene therapy for Fabry disease, can you talk a little bit kind of about the distribution of sort of severity and genotypes and phenotypes in this disease? And in particular the proportion of patients you think are most suited for gene therapy versus Galafold? And sort of talk about that -- both of you have your own gene therapy program as well as some competitor programs that are reporting data? Thanks.

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Great. Thanks, Ellie. I'm going to ask Jeff Castelli and Hung to comment in a moment, but you know at a very high level, we think patients are increasingly very satisfied with Galafold and those patients with amenable variance, we think Galafold will for a very long time, maybe for their entire lives be the standard of care. We do think there is a real opportunity to help patients who only have an enzyme replacement therapy, who have the burden of an every other week IV infusion, who have limitations of the bio distribution of the ERTs. For those patients, which represent half or more of the Fabry population, we do think gene therapy offers potential. It is a significantly complex development path ahead in February.

So maybe, Hung, if you want to describe the technical approach that we're taking in February? That's another program that we're collaborating with Dr. Wilson and UPenn on combining Hung in our science team's expertise in protein engineering with the Wilson Labs expertise in gene therapy. So Hung, I'll let you comment and then, Jeff, anything to add on the development path and the opportunity in the market, go ahead.

Hung Do -- Chief Science Officer

Sure. Thanks, John. And so as you know, there is at least half of the population who are not appropriate, let me say, not amenable to Galafold. So we feel we have an obligation and commitment to develop treatment for those patients. And so as John mentioned, we actually are utilizing our experience and knowledge being over the past 20 years in regards to understanding the lysosomal storage diseases and the actual enzymes, the characteristics that make these particular enzymes effective, we actually are applying those principles to our gene therapy. And so we intend to make the gene therapy with much better targeting and better stability for this particular -- with this particular approach. And then we utilize that in combination with Dr. Wilson and Penn's expertise for gene delivery. And so we think the combination of these two could prove to be quite effective to developing novel gene therapy for Fabry disease. Jeff, maybe anything else to add?

Jeffrey P. Castelli -- Chief Portfolio Officer and Head of Gene Therapy

No, I think, I could just add that. We have various studies ongoing right now preclinically in animal models, looking at different constructs, some was stabilized transgene, some were targeted transgene. And we expect lots of that data earlier this year. And this is one of the program that we could have a potential IND candidate declared this year based on the outcome of that data. So we're pretty excited, and really, an AAV gene therapy would apply to all patients. But as John mentioned, we think it would really be most probably attractive to patients that are not amenable to Galafold and don't have an oral option available.

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Great. Thanks, Jeff. I'll just add to that, we have got a significant amount of experience in the development of medicines for Fabry disease, significant reach around the world to all major treatment centers for Fabry disease, and of course, we've got experience now in about 40 countries with getting a medicine for Fabry disease approved. So we think that over other players in the field, we think that could be another significant advantage for us in addition to the very unique scientific approach that we're taking with Dr. Wilson and UPenn.

Operator

Your next question is from Mohit Bansal with Citi.

Keith Richard -- Citi -- Analyst

Good morning, guys. This is Keith on for Mohit. Thanks for taking my questions and congrats on the progress this quarter in the world [Phonetic]. Just wanted to ask a quick question, you may have mentioned this earlier, but questions on how you're planning to file in 2020 for AT-GAA, when the data might not be available until the second half of 2021? Thanks.

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Two things, one, our plan is to initiate a rolling BLA application, we would begin with the first module, the preclinical module followed by the CMC module. And then in the first half of 2021, where we will have the PROPEL data, we would then use that data in the first half of 2021 to complete the BLA submission with the clinical section, again, that's for full approval for a very wide patient population both pediatric and adult. And we would expect also with a Fast Track Designation that that would be about 0.5 years review time from there.

Operator

Your next question is from Whitney Ijem with Guggenheim.

Whitney Ijem -- Guggenheim -- Analyst

Hi, guys, thanks for taking the question. So on CLN6, I apologize if I missed it, but should we expect the data update there in terms of additional follow-up? And also on the natural history data? And then kind of what are the rate limiting steps there before you sort of start to engage regulators, thinking about next steps?

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Sure. Thanks, Whitney. So we expect in the second half of this year to have long-term follow-up data on the 13 patients who've been treated with the gene therapy and CLN6. Lots of other activities that would be necessary for a BLA filing, including a lot of activities around the manufacturing to remind everybody our partner there is Thermo Fisher Scientific in their Brammer division. We are now well under way and nearly complete with all the tech transfer and the scale up activities for the [Phonetic] GMP manufacturer in hyperstacks of that gene therapy. So a lot of activities on the manufacturing in the CMC side that would be necessary for a BLA. And again, we expect probably in the second half of the year to be able to articulate what's required for a BLA submission.

Operator

Your next question is from Yun Zhong with Janney.

Yun Zhong -- Janney -- Analyst

Hi. Thanks for taking the question. So the PROPEL study is enrolling both switch patient and naive patients. So I wonder, are you able to disclose more switch patient or more naive patients have been enrolled in the Phase 1/2 study. Have you ever looked at cohort combined including both switch patient and naive patient instead of individual cohort?

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Sure. Thanks, Yun. To remind everybody that PROPEL Phase 3 study for AT-GAA over enrolled and it enrolled a total of 123 patients, about 70% of them are switch patients from ERT standard of care, about 30% were treatment-naive. And Jay [Speech Overlap].

Jay A. Barth -- Chief Medical Officer

Yes, to your second question, the Phase 1/2 study had separate cohorts for a naive patients and switch patients. They weren't in one cohort, but they were in the same study. And therefore, we're able to take that data and that was the basis for the Phase 3 study design that included the switch patients and the naive patients. And the results that we saw were very favorable for both switch and naive in the Phase 1/2 study. So we are, as I said, very confident going along in the PROPEL study toward seeing the results in both switch and naive. It will be in the primary analysis, combining both those populations in the Phase 3 study in PROPEL.

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Yes, it's pretty straightforward, actually, Yun, if you just look at the -- we had 10 patients in Cohort 1 in the Phase 1/2. Those are the switch patients, ambulatory switch. And our Cohort 3 were five ERT treatment-naive patients. We saw, I think, Jay, if I remember, a six-minute walk, I think it was nine out of 10 -- eight or nine out of 10, at one year who showed improvements on AT-GAA, again very different from what you would expect to be a decline based on multiple natural history studies that have been done in our Phase 3 where five out of five showed improvements at one year on -- significant improvements at one year. So if you were to combine that, adding the naive would make it only look stronger.

Operator

Your next question is from Salveen Richter with Goldman Sachs.

Andrea Tan -- Goldman Sachs -- Analyst

Hi, thanks for taking the questions. This is Andrea on for Salveen. Could you speak a little bit about the Galafold naive switch dynamics, where you stand with respect to increasing that proportion of naive patients? And if there have been any gating factors there? And then I'm wondering if you could comment on the launch dynamics for Galafold in the Latin American geographies and if you're still expecting meaningful revenue contribution there in 2021 plus? Thank you.

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Yes, great question. So first on the dynamics between switch and naive patients, the last update we gave which was for full year was 66% of patients were switched and 34% were naive patients. And that's on track really with our strategy as you may recall, which is when we launch, we target switch patients first, they are already in the system getting their infusion every other week and so that's the lion's share of patients within the healthcare system. And then as we move along, we start to target the naive patients or previously unfilled [Phonetic] patients.

So if you look at our more mature markets like Europe, as an example of the EU five countries there, we're starting to see an equal contribution of switch and naive patients coming on to Galafold whereas in countries like the United States and Japan we're still seeing a predominant uptake in switch patients first, although we are seeing strong uptake in naive patients as well.

So that -- the dynamic that we continue to expect to play out in three or four years from now, we would expect to start to see perhaps more naive patients coming on Galafold as we've maximally penetrated into the switch market.

So that's the sort of switch, naive dynamic and again, it's well within our strategy and continues to move well.

From a Latin American perspective, as we mentioned, we did get some key approvals last year in Argentina and Brazil, and we would expect to begin to. And we've already seen some named patient sales in those markets. We would expect to start to see commercial sales in those markets. We will caveat though, as a reminder, in Brazil, as we seek national reimbursement, we're still in that judicial review process, which takes about a year for patients to go all the way through. And so that's why we've said we've expected 2021 to begin to be a significant growth driver from the Latin American market. So nice foothold so far, continuing to open up access for patients in those markets, but again we see 2021, 2022 as when those really start to meaningfully contribute to the overall revenue.

Operator

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

Aaron Welch -- H.C. Wainwright -- Analyst

Hi guys, just real quick. Not sure if I missed this in your prepared remarks, but was there -- of the $182 million fiscal year '19 revenue, how much of that is US versus ex-US? And for 2020?

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Sure. We've reported -- we did cover that. We'll repeat it. No problem. Daphne, please?

Daphne Quimi -- Chief Financial Officer

Yes, sure. So the US portion of that is approximately 30% and ex-US would be the remainder 70% of revenue.

Bradley L. Campbell -- President and Chief Operating Officer

We haven't given specific guidance as to the breakdown of US versus ex-US, that's kind of 70-30 [Phonetic] number is -- it will probably hold relatively stable, we're continuing to grow outside of the United States in terms of base of countries that contribute [Phonetic]. Of course, the US continues to launch well. So that's kind of right in the zone. It might move around a little bit from there. But I would expect that to be pretty consistent this year.

Operator

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

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Great. Thank you, operator. Thank you everybody for listening. As you see, it was a great 2019 and we're off to a really strong start on all metrics in 2020. Thanks for listening. Have a good day.

Duration: 45 minutes

Call participants:

Andrew Faughnan -- Director of Investor Relations

John F. Crowley -- Chairman of the Board, Chief Executive Officer

Bradley L. Campbell -- President and Chief Operating Officer

Daphne Quimi -- Chief Financial Officer

Jay A. Barth -- Chief Medical Officer

Hung Do -- Chief Science Officer

Jeffrey P. Castelli -- Chief Portfolio Officer and Head of Gene Therapy

Ritu Baral -- Cowen -- Analyst

Anupam Rama -- J.P. Morgan -- Analyst

Dae Gon Ha -- SVB Leerink -- Analyst

Aaron Welch -- H.C. Wainwright -- Analyst

Ellie Merle -- Cantor Fitzgerald -- Analyst

Keith Richard -- Citi -- Analyst

Whitney Ijem -- Guggenheim -- Analyst

Yun Zhong -- Janney -- Analyst

Andrea Tan -- Goldman Sachs -- Analyst

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