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
: Hi, guys. Congratulations on the approval. A quick question on some specific language in the label. Tolga, what value do you see the urgent heart
failure visit reduction language presenting to payers, especially as you contemplate frontline coverage for AMVUTTRA in this indication? Thanks.
Question: Jessica Fye - JPMorgan Chase & Co - Analyst
: Hey, guys. Good evening. Thanks for taking my questions. So in the past, you've talked about 5,000 to 10,000 cardiomyopathy patients initiating
treatment in the US each year at an increasing rate. I'm curious, if we look out, say, five years from now, where do you think that 5,000 to 10,000
per year initiating treatment is going to go?
Question: Tazeen Ahmad - Bank of America - Analyst
: Hi, good evening. Thanks for taking my question. This is probably for Tolga. Can you give us a sense, you've talked a lot about your focus on frontline
new patients. But as the launch is going to progress, how should we be thinking about switch patients or add-on patients? I guess in the label,
there doesn't seem to be reference about how to use it in combination with stabilizers. And does that impact at all your view of the opportunity
beyond frontline? Thanks.
Question: Paul Matteis - Stifel Financial Corp - Analyst
: Hey, thanks so much and congratulations. I wanted to ask a little bit more about your thought process behind pricing. When you've engaged with
Medicare plans, to what degree do you think that the Part B book of business is truly looked at in a vacuum?
And I guess, is your thinking that the price of AMVUTTRA shouldn't really be comped or I guess, won't be comped by insurers to the price of tafamidis
and acoramidis? And I guess that is sort of what I'm ultimately wondering here as to what gave you the confidence to price and maintain price at
a significant premium. Thanks so much.
Question: Konstantinos Biliouris - BMO Capital Markets - Analyst
: Hello, everyone. Congrats on the approval. Thanks for taking our questions. One question from us on recent real-world evidence data from tafamidis,
which saw that the discontinuation rate is actually very high, 30% to 40% between 12 and 24 months. We would be interested in hearing your
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MARCH 20, 2025 / 10:00PM, ALNY.OQ - Alnylam Pharmaceuticals Inc To Discuss FDA Approval of AMVUTTRA
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thoughts around what can you do to secure that those patients who are discontinuing tafamidis, they prefer to come to silencers versus another
stabilizer. And also, although Tolga mentioned some of those, any measures you are taking to avoid such high discontinuation break with AMVUTTRA?
Thank you and congrats again.
Question: Maury Raycroft - Jefferies Group LLC - Analyst
: Hi. I'll add my congrats and thanks for taking my question. I was just going to ask about the -- for the patient assistance program and initial free
dose if there's coverage issues. What are your expectations for this at the start of launch? And how could this evolve over time? Maybe just provide
more perspective on that.
Question: David Lebowitz - Citi - Analyst
: Thank you very much for taking my question and congratulations on the approval. On pricing, while you're maintaining the current list price and
you're going to be making use of rebates and whatnot to lower the net price over time.
I'm curious to start with what type of differential might there be between the cardiomyopathy and polyneuropathy at the get-go relative to these
rebates and aspects on net pricing? And will that actually differentiate over time with polyneuropathy maintaining a more premium approach and
then cardiomyopathy potentially having more of an impact?
Question: Gena Wang - Barclays - Analyst
: Thank you. Also congrats on the early approval and with a strong label. So regarding the net price, you mentioned that it will be decreased over
time. And how much net price impact to your 2025 revenue guidance, especially given like it's a $1.22 billion revenue in 2024 in polyneuropathy.
Question: Gena Wang - Barclays - Analyst
: Thank you.
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MARCH 20, 2025 / 10:00PM, ALNY.OQ - Alnylam Pharmaceuticals Inc To Discuss FDA Approval of AMVUTTRA
for ATTR-CM
Question: Gary Nachman - Raymond James Financial Inc - Analyst
: Thanks and my congrats as well on the approval. So understanding this is more of a second half impact on the launch, what type of metrics will
you provide to give us a sense of how the CM launch is going in terms of payer access, patients treated, physician prescribing or anything else that
you could talk to give us a sense of the launch? Thanks.
Question: Luca Issi - RBC Capital Markets - Analyst
: Great. Thanks so much for taking the question. Congrats on the approval. I know it's a long journey, so congrats. Maybe Tolga, a quick one for you.
It feels to me that this drug is going to do really well at top academic centers, especially given the favorable buy-and-bill economics for the hospital.
But maybe it will be a little bit harder in the community settings where maybe such economics are less of a factor and maybe where patients and
physicians may prefer the convenience of an oral pill, especially if the patients live kind of far from an amyloidosis center. Would you agree with
that characterization? And if so, what's the strategy to capture market share across both urban and more rural areas there? Any color there much
appreciated. Thanks so much.
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MARCH 20, 2025 / 10:00PM, ALNY.OQ - Alnylam Pharmaceuticals Inc To Discuss FDA Approval of AMVUTTRA
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Question: Ellie Merle - UBS - Analyst
: Hey, guys. Thanks so much for taking the question. Just in terms of combination use, what the payers said about this in your initial conversation.
And then in terms of the at-home infusion option that you mentioned, what's the (inaudible) in polyneuropathy? And how do you expect that to
compare in cardiomyopathy?
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