Amicus Therapeutics, Inc. Q1 FY2025 earnings call

Management Statement and Operational Highlights

• Core Growth: Amicus has achieved eleven consecutive quarters of double-digit revenue growth in Pompe and Fabry, with continued growth expected.
• Strategic Collaboration: Entered a strategic collaboration with Dimerix for US commercialization rights to a late Phase III compound for FSGS, featuring a modest upfront and success-based milestones.
• Supply Chain: Strengthening and diversifying supply chain, including onshoring part of Pombiliti drug product manufacturing.
• Galafold Details: Revenue grew 6% at constant rates, with 14% increase in new patient starts globally and strong market share in treated Fabry patients.
• Pombiliti and Opfolda Progress: Adjusted revenue guidance, expansion into new countries (e.g., Italy, Czech Republic, etc.), and strong patient start momentum in April.
• Evidence Base: Pom-Op benefits from differentiated clinical data, mechanistic insights, and real-world evidence supporting efficacy and safety across broader populations.

Segment Performance

• Galafold: Revenue reached $104.2 million in Q1, up 6% at constant exchange rates. New patient starts globally increased 14% in the quarter, with the company holding over 69% of the global market share of treated Fabry patients with amenable mutations.
• Pombiliti and Opfolda: Revenue was $21 million in Q1, up 92% at constant exchange rates. The full year 2025 revenue growth guidance for these products was adjusted from 65%-85% at constant exchange rates to 50%-65%. April saw the largest number of net new commercial patients globally, driven by the US.

Guidance

• Total revenue growth guidance updated to 15%-22% from prior 17%-24%.
• Galafold revenue growth guidance reiterated at 10%-15%.
• Pombiliti and Opfolda revenue growth guidance revised to 50%-65% from 65%-85% at constant exchange rates.
• Anticipate GAAP profitability in the second half of 2025.
• Non-GAAP operating expense guidance set at $380M-$400M, including $30M upfront for the DMX-200 licensing.

Risks

• UK Rebates: Higher-than-anticipated rebates in the UK impacted Galafold and Pom-Op revenues, though patient growth is expected to offset this for Galafold.
• Tariffs: Uncertainty around tariffs, but proactive supply chain planning is expected to mitigate material impact on operations in 2025.

Question and Answer

Q: Particular noted assessments for both Galafold and Pombiliti-Opfolda that led to the rebate being a little bit higher than anticipated for both products?

A: Thanks Anupam. It’s really frustrating. That’s a negotiated rebate by industry association in The UK and they give guidance to industry of what to expect for the year. And so our guidance was between 12-15%. And we assume the maximum of that rate 15% to be conservative. In the end, they came back with a negotiated rate of 22%. So almost a 50% increase in what we had anticipated.

Q: In terms of the cadence of ex-U.S. launches and patient starts, what has changed on your expectations there? And how much of that contributed to you guys taking down the Pom-Op guidance?

A: Yes, thanks for the question. I think there’s really two big elements there. The first is exactly as you said, you saw in previous slides as we came into the year and then we reiterated here, we reached a number of reimbursement settlements at the end of last year in the early part of the quarter. And we had assumed that those patients would start on therapy in the first quarter. Unfortunately, of them started, only a handful started in the first quarter and many of them are starting to start in the second quarter. So really that’s a timing thing. So it’s kind of just pushes the curve to the right.

Q: On DMX-200, even if qPCR is a fully approvable primary endpoint, what is any benefit in eGFR is FDA looking for besides the primary? Two, what timeline should we expect for the next interim analysis which is gated by Parasol and FDA? Does nine months sound reasonable? And three, what have you seen to justify acceleration of U.S. switches for Pom-Op in 2HAP-25?

A: Great. So maybe I’ll start with Palm Up first and then Jeff, you can speak to those other points. So, look, I think as Sebastien shared with you on the call, we’re already seeing a significant acceleration in switches in Pombiliti and Opfolda in The United States and around the world just in the month April. So it was really it was actually our best month for new commercial starts, so not clinical trial conversions but new commercial starts since we’ve launched. So that’s really exciting. Why are we seeing that? Part of it for sure is just the dynamic we had already talked about which is more and more Nexviazyme patients entering into that two year period. And I’ll mention again that we saw in that period, majority of the switchers coming from Nexviazyme. So we’re winning that game. I think also part of it is what Jeff talked about which is the growing body of real world evidence. We presented for the first time, or one of our thought leaders did at a medical congress, case studies supporting the switch from Nexviazyme for the first time. So that’s, I think really important real world evidence that will continue to grow. And I do think too, we’ve talked about this before, we’ve always seen Q1 as a sort of nonlinear quarter in Fabry. And I think you may start to see that in Pompe as well. So this could be a reflection of that. And then if you add in those kind of onetime factors we talked about ex-U.S., I think that kind of explains the quarter. Jeff, do you want to talk about FSGS and both the proteinuria GFR feedback from the FDA which is fantastic from the Type C meeting and then also some of the early timelines that we have line of sight to.

Q: Hi, Josh. On DMX-200, even if qPCR is a fully approvable primary endpoint, what is any benefit in eGFR is FDA looking for besides the primary? Two, what timeline should we expect for the next interim analysis which is gated by Parasol and FDA? Does nine months sound reasonable? And three, what have you seen to justify acceleration of U.S. switches for Pom-Op in 2HAP-25?

A: Great. So maybe I’ll start with Palm Up first and then Jeff, you can speak to those other points. So, look, I think as Sebastien shared with you on the call, we’re already seeing a significant acceleration in switches in Pombiliti and Opfolda in The United States and around the world just in the month April. So it was really it was actually our best month for new commercial starts, so not clinical trial conversions but new commercial starts since we’ve launched. So that’s really exciting. Why are we seeing that? Part of it for sure is just the dynamic we had already talked about which is more and more Nexviazyme patients entering into that two year period. And I’ll mention again that we saw in that period, majority of the switchers coming from Nexviazyme. So we’re winning that game. I think also part of it is what Jeff talked about which is the growing body of real world evidence. We presented for the first time, or one of our thought leaders did at a medical congress, case studies supporting the switch from Nexviazyme for the first time. So that’s, I think really important real world evidence that will continue to grow. And I do think too, we’ve talked about this before, we’ve always seen Q1 as a sort of nonlinear quarter in Fabry. And I think you may start to see that in Pompe as well. So this could be a reflection of that. And then if you add in those kind of onetime factors we talked about ex-U.S., I think that kind of explains the quarter. Jeff, do you want to talk about FSGS and both the proteinuria GFR feedback from the FDA which is fantastic from the Type C meeting and then also some of the early timelines that we have line of sight to.