Amicus Therapeutics Inc

NASDAQ:FOLD

Earnings Call Transcript 2019-Q4

[Operator]

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

I would now like to turn the conference over to your host, Ms. 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 two, we may make forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 relating to our business, as well as our plans and prospects. 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 the 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 Crowley, Chairman & 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 for 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 towards 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 a strategic review of our business in the second half of last year, we continued 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 a medical 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 patents program.

Fourth, in addition, we are progressing our Pompe gene therapy towards IND and we'll disclose up to two additional IND candidates this year. A lot of work is underway with our manufacturing partners for the manufacture and scale-up of the Pompe gene therapy, 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 – 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 Campbell, President & 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 U.S. 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 the United States and the remaining $52.5 million or about 29% coming from within the U.S. 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 the 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 in our future products as well.

Turning now to slide eight, I'll recap the performance over 2019. As John mentioned, on the right-hand side of the slide, you could see that 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 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 nine. 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 towards funding our R&D and OpEx over that period.

We also had 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 U.S. 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 Crowley, Chairman & 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 from 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, enrolled the pediatric studies and advanced manufacturing to support a 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 a full approval under the fast track designation.

Two new updates this morning with respect to AT-GAA are: first, in response to the many requests 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 biologic 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 is 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 solution 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 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 transitioned 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 Crowley, Chairman & 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 U.S. 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 plan 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' 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.

[Operator]

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

[Ritu Baral, Cowen]

Good morning, everyone. Thanks for taking the question. I wanted to ask about, what is left to complete data-wise before you start the 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 – for your desired label?

[John Crowley, Chairman & Chief Executive Officer]

Yes. To the last – 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 underway. 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 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's needed to begin the rolling BLA, there's 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]

Great. Thanks.

[John Crowley, Chairman & Chief Executive Officer]

Very welcome.

[Operator]

Your next question is from Anupam Rama with JPMorgan.

[John Crowley, Chairman & Chief Executive Officer]

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

[Anupam Rama, JPMorgan]

Can you hear me now?

[John Crowley, Chairman & Chief Executive Officer]

Now we can, yes.

[Anupam Rama, JPMorgan]

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 is the next Pompe update going to be PROPEL? Thanks so much.

[John Crowley, Chairman & Chief Executive Officer]

Yeah. I'll let Jay speak to that.

[Jay Barth, Chief Medical Officer]

Yeah. 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. But –

[John Crowley, Chairman & Chief Executive Officer]

Yeah. But just to remind everybody all patients continued not – nobody discontinued. Everybody continues. Nobody has gone back to Lumizyme in that study, who was on it before. 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]

Hey, 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 aspects. Specifically, as investors are anticipating the neoGAA 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 opportunity that you currently have modeling for those respective scenarios? Thanks.

[John Crowley, Chairman & Chief Executive Officer]

Yeah. 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 PIM's designation in the U.K. So I think we'll – we're really, really confident in AT-GAA to be very distinct.

Again, another major distinction we're studying a range of patients in our PROPEL Phase 3, including both switch patients who make up about 70% of the population for 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 waitlist of a 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]

Hi, guys. Good morning. This is Aaron on for Debjit. Congratulations on all 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?

All right. Next question is…

[John Crowley, Chairman & Chief Executive Officer]

Hold on. Let me stop you. We've got one question there. We will answer that one. Jay, I'll let you take that one.

[Jay Barth, Chief Medical Officer]

Yeah. I mean without getting into the technical aspects of your question, which I don't know if everybody appreciate, but having looked at the antibodies that was something we present at the World for -- 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 of showing a very robust effect and efficacy for six-minute walk. And that's why based on 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]

Hey guys, thanks so much for taking the question. Just in terms of gene therapy for Fabry disease, can you talk a little bit about the distribution and 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 talk about that versus your own gene therapy program, as well as some competitor programs that are reporting data. Thanks.

[John Crowley, Chairman & Chief Executive Officer]

Great. Thanks Ellie. I'm going to ask Jeff Castelli and Hung to comment in a moment, but at a very high level, we think patients are increasingly very satisfied with Galafold. And those patients with a minimal 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 though to help patients who only have an enzyme replacement therapy who have the burden of an every other week IV infusion who have the limitations of the biodistribution 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 Fabry.

So maybe Hung if you want to describe the technical approach that we're taking in Fabry? That's another program that we're collaborating with Dr. Wilson and UPenn on combining Hung and our science team's expertise in protein engineering with the Wilson Lab's 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 a 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 a 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 develop a novel gene therapy for Fabry disease.

Jeff maybe anything else to add?

[Jeff Castelli, Chief Portfolio Officer & Head of Gene Therapy]

No. I think I could just add that, we have various studies ongoing right now pre-clinically in animal models, looking at different constructs somewhat stabilized transgene, some with targeted transgenes. And we expect much of that data early this year. And this is one of the programs 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 Crowley, Chairman & Chief Executive Officer]

Great. Thanks guys. I'll just add too 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 Tapper, Citi]

This is Keith on for Mohit. Thanks for taking our question, and congrats on the progress this quarter and the World. 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 second half of 2021? Thanks.

[John Crowley, Chairman & Chief Executive Officer]

Two things. One this -- our plan is to initiate a rolling BLA application. We would begin with the first module, the pre-clinical 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 it's for full approval for a very wide patient population both pediatric and adult. And we would expect also the fast track designation that that would be about a half a year review time from there.

[Operator]

Your next question is from Whitney Ijem with Guggenheim.

[Whitney Ijem, Guggenheim]

Hey 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 what are the rate limiting steps there before you start to engage regulators thinking about next steps?

[John Crowley, Chairman & 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 in CLN6. Lots of other activities that would be necessary for BLA filing including a lot of activities around the manufacturing. To remind everybody our partner there is Thermo Fisher Scientific and their Brammer division. We are now well underway nearly complete with all the tech transfer and the scale-up activities with the GMP manufacturer in hyperstacks of that gene therapy, so a lot of activities on the manufacturing and 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 the Janney.

[Yun Zhong, Janney]

Hi. Thanks for taking the question. So the PROPEL study is enrolling both switch patient and naive patient. So I wonder, are you able to disclose more switch patient or more naive patient 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 Crowley, Chairman & Chief Executive Officer]

Sure. Thanks Yun. To remind everybody that PROPEL Phase 3 study for AT-GAA overenrolled 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 I -- the other question?

[Jay Barth, Chief Medical Officer]

Yes. To your second question, the Phase 1/2 study had separate cohorts for 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 Phase 1/2 study, so we are as I said very confident going along in the PROPEL study towards 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 Crowley, Chairman & Chief Executive Officer]

Yeah. It's pretty straightforward actually. Again if you just look at the -- we had the 10 patients in cohort one in the Phase 1/2. Those are the switch patients ambulatory switch. And our cohort three 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'd 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]

Hi, thanks for taking my question. 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.

[Jay Barth, Chief Medical Officer]

Yes, great questions. 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 switch 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're 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 untreated patients.

So if you look at our more mature markets like Europe, as an example, the EU5 countries, there we're starting to see an equal contribution of switch and naive patients coming on 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's 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 sweet 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 in 2021 to begin to be a significant growth driver from the Latin American market.

So a nice foothold so far, continuing to open up access for patients in those markets. But again, we see 2021, 2022, as when those would 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]

Hi, guys. Just real quick, not sure if I missed this in your prepared remarks. But was there – of the $182 million fiscal year 2019 revenue, how much of that is U.S. versus ex U.S.? And for 2020.

[John Crowley, Chairman & 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 U.S. portion of that is approximately 30% and ex U.S. will be the remainder 70% of revenue.

[Bradley Campbell, President & Chief Operating Officer]

We haven't given specific guidance as to the breakdown U.S. versus ex U.S. That kind of 70-30 number is – it will probably hold relatively stable. We're continuing to grow outside the United States in terms of base of countries that contribute. Of course, the U.S. 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 Crowley, Chairman & 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.