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: Kristen Kluska - Cantor Fitzgerald, L.P. - Analyst
: Great. So, I'll start the focus of questions on AT-GAA for Pompe disease, especially considering you just announced the FDA acceptance of your
filings yesterday. I think it would be helpful to first contextualize the current market centered around current standard of care, Lumizyme. While
patients may start treatments at different times of their life at different levels of severity, could you please touch on the trends that have been
observed with this treatment?
Question: Kristen Kluska - Cantor Fitzgerald, L.P. - Analyst
: Okay, great. Thank you. And to your point that patients on Lumizyme have been followed up for so long, I did want to ask you a question about a
study that you referenced earlier this year with 68 patients collected across multiple countries who had been on ERT for at least three years. In this
study it was shown that the weakest efficacy was for lung capacity which led to ventilatory support requirements. So, I wanted to ask how this
study and other studies of course that are out there strengthen the importance of evaluating the forced vital capacity measurement, as well as,
again, reinforcing the unmet need in this condition.
Question: Kristen Kluska - Cantor Fitzgerald, L.P. - Analyst
: Yes, thank you. So, keeping in mind everything you just said about this opportunity and having a data set on hand for these patients, when do you
think, if approved, physicians might consider switching their patients to AT-GAA, again, because the literature is out there, and they've been
following up the patients for so long?
Question: Kristen Kluska - Cantor Fitzgerald, L.P. - Analyst
: Okay, thanks. And you've guided that your existing sales force for Galafold could be leveraged for AT-GAA if approved. So, how are you thinking
about the trajectory of reaching peak sales for this opportunity versus Galafold given you already have this team built in place and many of the
physicians you have relationships with see patients with both conditions?
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SEPTEMBER 30, 2021 / 12:40PM, FOLD.OQ - Amicus Therapeutics Inc at Cantor Fitzgerald Global Healthcare
Conference (Virtual)
Question: Kristen Kluska - Cantor Fitzgerald, L.P. - Analyst
: Great. So, over the past three years, you've set out revenue guidance for Galafold in Fabry disease in early January. And each of these three years,
your actual numbers have been slightly ahead of your guided range. As this is the first commercial product and the cornerstone of your business,
could you discuss the criteria that goes into putting these estimates each year, including the 2021 guidance of $300 million to $315 million? And
of some of the growth drivers, which do you think are going to be the most instrumental in reaching the peak opportunity of $1 billion-plus?
Question: Kristen Kluska - Cantor Fitzgerald, L.P. - Analyst
: Great, thank you. So, yesterday you announced plans to form a next-generation genetic medicine company, Caritas Therapeutics. John, looking
back at your 20-plus-year career in the rare disease space, companies like Amicus have been able to build therapies that show improvements over
existing standard of care. That said, there's always room for even more improvement on these therapies and many conditions out there still don't
have any effective options. So, why do you believe this is a critical time to focus more of your energy and attention to the gene therapy space?
Question: Kristen Kluska - Cantor Fitzgerald, L.P. - Analyst
: Okay, thanks. And as there was a lot of announcements that took place yesterday, I wanted to ask if you could touch upon maybe some of the main
questions or comments that you received. But then also open the floor to you to see if there's anything that you believe may have been misunderstood
or needs to be clarified.
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consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies.
SEPTEMBER 30, 2021 / 12:40PM, FOLD.OQ - Amicus Therapeutics Inc at Cantor Fitzgerald Global Healthcare
Conference (Virtual)
Question: Kristen Kluska - Cantor Fitzgerald, L.P. - Analyst
: Great. Well, thank you so much, John. I appreciate your time. Thanks for answering my questions. Hope everybody has a good day.
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