Amicus Therapeutics Inc at UBS BioPharma 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 : Where do you want to start?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Exciting. Well, maybe just on profitability for -- to remain profitable going forward, particularly thinking on the OpEx side, what's baked in from a Pompe perspective? And do you expect OpEx to remain completely flat, any growth, any decrease in OpEx, how should we think about this as a business going forward?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Absolutely. And Pompe and Pombiliti and Opfolda will definitely be a key part of that, right? If your expenses are flat, then that essentially will go down to profits.

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : So let's talk about the launch. Okay. Maybe before I go into the specifics from the quarter, just high level, how should we think about the size of the Pompe market opportunity as a whole and then we can talk about competition from there.

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : And maybe with respect to competition in the Pompe space, how do you think that you're differentiated from next event?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : And I guess from a marketing perspective, how do you ensure that physicians are aware of this difference?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Absolutely. And let's speak a little bit about the label. So at least in the U.S., you have a bit of a different label than Nexviazyme. Maybe starting with not improving on ERT and what that means in terms of the indication statement.

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Makes sense. And maybe starting with like in the U.S. at 3Q? I mean you mentioned, I think, like 2/3 prescription referral forms already. Tell us a little bit more about that and what you saw with the conversion from the clinical trial patients.

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Absolutely. And so at the very least with the clinical trial and expanded access patients I mean, rough math, that's like a run rate of $50 million annually going into next year?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : That's helpful. And maybe curious about some of these dynamics you're seeing outside of the conversion to paying but with new starts. What's the mix of switches versus naive, maybe just in Europe [and] if not in the U.S. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 09, 2023 / 6:00PM, FOLD.OQ - Amicus Therapeutics Inc at UBS BioPharma Conference

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : I mean we started with saying that almost the majority of patients -- vast majorities, like 75% or so are not improving on their current ERT. How do we think about the patient motivation, how involved the patients are in their care?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Do you think it's the patients that are on Nexviazyme that are going to be the first to switch because they're the ones that are motivated in seeking new treatments? Or is it the patients that are on Lumizyme and really are waiting for a new next-generation treatment?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : And I know you've mentioned sort of reimbursement in Germany and the U.K., but maybe we can talk about the rest of Europe and how to think about the timing there. (technical difficulty) I guess what are you hearing in terms of like requests (technical difficulty) in the U.S., also the insurance process.

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Anything surprising?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Yes Absolutely. Going back to just thinking about the overall size of the patient population. I mean, you mentioned there's a large portion of Pompe patients that are not diagnosed. What's a reasonable like way to think about how this could change over time?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Interesting. And yes, when can we expect data from these studies? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 09, 2023 / 6:00PM, FOLD.OQ - Amicus Therapeutics Inc at UBS BioPharma Conference

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Absolutely makes sense. And I'm not going to ask if you're going to give guidance for Pompe, but maybe just...

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Maybe just what metrics can we expect as we continue to get updates like anything in terms of patient numbers? Like how should we think about that?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : And there's a lot of debate among investors about the competition and how to think about market share. I know it's early, but maybe can you speak to some of the initial learnings, particularly from the U.K. and the first couple of months in Germany in terms of thinking about market share relative to Nexviazyme?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : How should we think about how centralized or decentralized the treatment of Pompe is both in the U.S. and in Europe?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : I mean it seems a lot of the physicians have already been involved in clinical trials. How do you think about awareness, particularly of your clinical data?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Thinking longer term, and I know this is all very hypothetical, how do we think about duration of treatment, particularly for the 2 next-generation ERTs in the market? If you start on one, whether it's yours or Sanofi, at some point, you progress, then you switch to the other, maybe improve for REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 09, 2023 / 6:00PM, FOLD.OQ - Amicus Therapeutics Inc at UBS BioPharma Conference a bit and then unfortunately, it eventually progress. Do you switch back, I guess, from a mechanistic perspective, like how do you see this tying out?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Absolutely. Pivoting to Galafold, so tell us about the recent growth drivers. You recently raised your guidance and both in the near term as well as longer term, what brings you to the $1 billion in the next 5 years.

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Absolutely. It seems like a lot of factors at play driving the growth maybe over the next 5 years to get to this $1 billion. What are the (technical difficulty) some of the (technical difficulty) maybe just starting from a geographic perspective, what's the mix in that $1 billion?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Well, it's certainly a growing revenue base. Tell us more about the IP.

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : And I guess, with Galafold being a small molecule, I mean I think you've had a intentional patent strategy. And I think there's certainly been a lot more IP that's been filed over the last few years. Tell us about that strategy with respect to Galafold and maybe what the puts and takes are in terms of the key pieces of IP?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Understood. Maybe turning to the pipeline. Tell us about some of your work in gene therapy.

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : That's very interesting. How do you prioritize that relative to gene therapy?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : And given turning the corner on profitability, thinking longer term, how should we think about the potential for business development when you think about further growth, do you see it coming from internal R&D or perhaps external? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 09, 2023 / 6:00PM, FOLD.OQ - Amicus Therapeutics Inc at UBS BioPharma Conference

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Absolutely. What are you most excited about in the pipeline?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Understood. Can you tell us a bit more about this at least the science behind the next-generation Galafold?

Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst : Absolutely. And maybe just to round it out, what do you think is most underappreciated about your story? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 09, 2023 / 6:00PM, FOLD.OQ - Amicus Therapeutics Inc at UBS BioPharma Conference