Amicus Therapeutics Inc at UBS Global Healthcare Virtual Conference Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : And maybe just to kick it off, John, just for some background, those who are newer to the story. Can you give us a quick overview of Amicus' rare disease portfolio and, I guess, key goals for the company in the coming years?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Thanks, John. Maybe just starting with Galafold. What do you see as the key drivers of growth over the next 2 to 3 years? And maybe how does this differ from, say, your initial expectations when you first launched the product years back and sort of your confidence in getting to your long-term sales target of $500 million and then $1 billion versus the $260 million sold in 2020?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Got it. That's really helpful. And just digging a little bit deeper into some of the geographic expansion. You mentioned about 90% of the amenable patients in Germany are treated with Galafold. I guess as you think about further penetration outside of Germany and EU, in some of the countries like U.S., Brazil, what are some of the hurdles to achieving this 90% share of the amenable patients for Galafold?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Got it. That's helpful. And I guess just thinking longer term in the Fabry market in terms of competition, both with gene therapy products in development. But then also how your own gene therapy product for Fabry could transform kind of the treatment paradigm and where you see that going? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MAY 24, 2021 / 2:00PM, FOLD.OQ - Amicus Therapeutics Inc at UBS Global Healthcare Virtual Conference

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Right. And where are you in sort of the IND-enabling work? And when should we expect a potential IND filing and first patient dosed?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Got it. That's helpful. And then just turning to Pompe. I mean what -- after sort of the Phase III data, the miss by sort of a hair in the primary end point, can you talk a bit about your confidence in a U.S. and ex U.S. approval in light of the study? And sort of why you think this was meaningful data in the context of the treatment paradigm?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Got it. Very helpful. And just turning to sort of the submission timeline and steps, maybe just with the BLA as well as the MAA in Europe. Just how should we think about the time lines both in terms of the clinical package as well as the manufacturing package? And I guess, when we should REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MAY 24, 2021 / 2:00PM, FOLD.OQ - Amicus Therapeutics Inc at UBS Global Healthcare Virtual Conference expect a potential decision by and theoretical launch? And just if you had to identify any key risks to the time lines or the approvability, I guess, what would those key risks or considerations be?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : And then I guess just key risk. Like if anything goes wrong with the filing, what do you think it would be?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Got it. That's helpful. And just in terms of competition, I mean, there is a lot of talk obviously about Sanofi's neoGAA and could potentially be launching ahead of AT-GAA. How are you thinking about patients that, say, might have switched from Lumizyme to neo and their willingness to, say, switch from neo to AT-GAA? And how are you planning for potential commercial strategies around perhaps neo switches to your product?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : And I guess just from sort of a commercial cadence and thinking about uptake. I mean how should we think about things like how often Pompe patients see their physician? Sort of, would this be something that physicians call their patients proactively to discuss switching once approved? Or is it something that could take maybe a slower cadence? And is it just going to be the patients that are declining on ERT that you think would be switched? Or would it be patients that even are stable or still improving on ERT that would be considering a switch?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Got it. Makes sense. And maybe just pivoting to some of your gene therapy programs. How should we think about the time lines and potential path to market in both of the Batten franchises in CLN6 and CLN3? And where you are in some of the manufacturing work post completing the tech transfer and before you can, say, dose patients in the registration studies?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Got it. Yes. Very exciting work. (Operator Instructions) REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MAY 24, 2021 / 2:00PM, FOLD.OQ - Amicus Therapeutics Inc at UBS Global Healthcare Virtual Conference But I guess while we maybe wait for any questions from the audience, I did want to ask a little bit more on this emerging gene therapy portfolio. You have a lot of programs in preclinical development. How are you thinking about manufacturing going forward? And when you could potentially have your own in-house manufacturing ready and live to dose in patients? And is this even necessary given your partnerships with some of the companies like Brammer, where you're working on manufacturing now, such as with the CLN programs?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Very exciting. Well, I think we're at 10:45 here, and I don't see any questions on the line. But John, thank you so much for your time. Great hearing your insights and vision for the company. And yes, thanks, everyone, on the line for joining, and we will speak to you all soon.