The following is excerpted from the question-and-answer section of the transcript.
(Questions from industry analysts are provided in full, but answers are omitted - download the transcript to see the full question-and-answer session)
Question: Ritu Baral - TD Cowen - Analyst
: So let's start with PomOp, or Pombiliti-Opfolda, for Pompe. The launch is now one year out. What sort of trends are you seeing right now?
Question: Ritu Baral - TD Cowen - Analyst
: So on the last two points that you mentioned, Brad, I want to say this delicately. There has been an 800-pound gorilla in the space in Pompe for
years, Sanofi Genzyme. They have given many, many research clinicians, who also happen to be treating physicians, lots of dollars for research and
patient groups and support. They are well established, well loved.
Oftentimes, there's a sense of loyalty, I think is the best way of putting it. And when you go into that space, understanding that there are physicians
who are much more familiar with the Sanofi Genzyme drug, much more comfortable with their Sanofi Genzyme relationship, it's a classy way of
putting it. How are you seeing that evolve, going back to your comment on number of prescribers?
Question: Ritu Baral - TD Cowen - Analyst
: So given the indication in the US is for treatment-experienced patients, naive patients are part of the European label. In-clinic evaluation of patients
is very important to your Pompe opportunity. Can you walk us through what the average neuromuscular expert measures and assesses during
their Pompe patient visit? And how often in your market research do Pompe patients actually come back and get monitored?
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MARCH 04, 2025 / 2:50PM, FOLD.OQ - Amicus Therapeutics Inc at TD Cowen Healthcare Conference
Question: Ritu Baral - TD Cowen - Analyst
:
Question: Ritu Baral - TD Cowen - Analyst
: Is there a practical PRO scale that can be used?
Question: Ritu Baral - TD Cowen - Analyst
: I mean, there's ALT, ASTs, too, right?
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MARCH 04, 2025 / 2:50PM, FOLD.OQ - Amicus Therapeutics Inc at TD Cowen Healthcare Conference
Question: Ritu Baral - TD Cowen - Analyst
: About who's declining and therefore appropriate.
Question: Ritu Baral - TD Cowen - Analyst
: Got it. And what's the timeframe generally that these assessments are made over?
Question: Ritu Baral - TD Cowen - Analyst
: Whereas like decline happens over what period?
Question: Ritu Baral - TD Cowen - Analyst
: Has coverage been largely squared away in the US? We had heard something earlier from doctors about getting the Pombiliti part covered versus
the Opfolda part covered. Is anything left to optimize in time to fill?
Question: Ritu Baral - TD Cowen - Analyst
: I see. Okay.
Question: Ritu Baral - TD Cowen - Analyst
: So Genzyme has been sort of counter-detailing the fasting requirements around. We've had doctors who say this is no big deal, but we keep hearing
about it. On the investor side is a question. How are you addressing that, and how are patients handling that in the real world?
Question: Ritu Baral - TD Cowen - Analyst
: So on your 4Q earnings call, you noted that 40% of current Nexviazyme patients are approaching this two-year treatment mark that our surveys
suggest is really, really, really critical to their self-assessment for how they're doing on the therapy. Do you have any market insight on how those
patients are doing based on talking to clinicians? I mean, it's a psychology question. What's the willingness to tolerate decline in these patients
and in the clinicians? And that can be different.
Question: Ritu Baral - TD Cowen - Analyst
: Right. I hope those posters, which seem really important, are not at World Muscle because apparently investors are not welcome there to appreciate
that.
Question: Ritu Baral - TD Cowen - Analyst
: Excellent. Because I think those will be very important to the marketing effort going forward, given Genzyme was -- how should I put this? At their
commercial presence at World, the absence of their Nexviazyme materials, messaging, symposiums was noted, let's put it that way. Any additional
thoughts on that changing competitive dynamic, the counter-detailing dynamic? What have you noticed?
Question: Ritu Baral - TD Cowen - Analyst
: Can you walk us through the major regulatory submission and reimbursement agreements for PomOp outstanding in 2025?
Question: Ritu Baral - TD Cowen - Analyst
: Identified early, asymptomatic for now, and whether they are worth treating or not.
Question: Ritu Baral - TD Cowen - Analyst
: All right. Let's move to Galafold because obviously it is a big part of your revenue base. What are the biggest levers as you see it right now for
continued growth for Galafold 2025, 2026? Geography seems to be tapped out. You're pretty much expanded worldwide. So how are these growth
drivers evolving?
Question: Ritu Baral - TD Cowen - Analyst
: Got it. In the last five minutes, I do want to talk about your business development strategy and your internal pipeline. What sort of internal pipeline
development is underway? And what sort of timelines are we talking about?
Question: Ritu Baral - TD Cowen - Analyst
: As you think about internal and external focus, are you still focused on derisked late clinical development? And what therapeutic area? Are you
going to maintain this almost ultra-rare focus?
Question: Ritu Baral - TD Cowen - Analyst
: Sales force.
Question: Ritu Baral - TD Cowen - Analyst
: So we could be thinking less sort of like who's the same call point or which programs have the same call point, thinking more what's out there that
isn't being promoted in Europe and could slot into your very successful European sales operation.
Question: Ritu Baral - TD Cowen - Analyst
: Great. I think with that, we are at time. Brad, thank you very much. It was very insightful.
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MARCH 04, 2025 / 2:50PM, FOLD.OQ - Amicus Therapeutics Inc at TD Cowen Healthcare Conference
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