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: Eliana Merle - UBS - Analyst
: Maybe just to start off, you've mentioned that we're approaching a large inflection point for the company. Maybe to start off, can you provide a
summary of some of the highlights from this past year, but then also going forward, what we should be focused on?
Question: Eliana Merle - UBS - Analyst
: Great. It's an exciting time.
Question: Eliana Merle - UBS - Analyst
: Makes sense. Maybe before we dive into some of the pipeline programs starting with TTR. So the cardiomyopathy launch next year is going to be
watched very, very closely and helpful Investor Day on the TTR commercial landscape.
But maybe I just want to dive into a little bit sort of your views of the frontline market and how you view uptake in, say, naive patients versus
switches. I think you made some interesting commentary around seeing sort of a lot of uptake in naive patients. Maybe just as we think of the
cadence of the launch next year, where do you see sort of this initial uptake between the naive and switches?
Question: Eliana Merle - UBS - Analyst
: Absolutely. It's a very compelling data. And another topic of conversation, and this is something that we've certainly heard from physicians. I find
that sometimes I get a different answer from physicians on if I say, well, what medicine would you want to be on versus what are you going to
prescribe where reimbursement is a huge factor? I know there's a lot of conversations around BD and understanding that. But yeah, what drives
your confidence and --
Question: Eliana Merle - UBS - Analyst
: Absolutely, it makes sense. And just from a coverage perspective, like logistically, how does this take place? You already have the coverage in
polyneuropathy, do you then automatically get coverage in cardio --
Question: Eliana Merle - UBS - Analyst
: Any color you can share from recent conversations with the payers?
Question: Eliana Merle - UBS - Analyst
: Understood. And another topic of conversation is how to think about the market long term, particularly when tafamidis goes generic. I mean,
theoretically, this could be a very good thing or a very bad thing depending on how the payers treat it. What's your perspective?
Question: Eliana Merle - UBS - Analyst
: Interesting. Could you tell us a little bit more about that? Maybe just the different potential strategies that you could take with Sc04.
Question: Eliana Merle - UBS - Analyst
:
Question: Eliana Merle - UBS - Analyst
: Understood. Okay. And another question that you perhaps might decline to answer. But we're all trying to think about how to model the cadence
of uptake in cardiomyopathy next year. How are you thinking about that?
Question: Eliana Merle - UBS - Analyst
: Great. Well, we'll definitely come back to some of the modeling and thinking about breakeven. But first, I just want to touch a little bit more on the
pipeline. So Sandeep, you mentioned a handful of programs. Maybe just in some of the earlier stage pipeline, Huntington's, Alzheimer's, what are
you most excited about? And talk us through some of the milestones we can expect there.
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NOVEMBER 12, 2024 / 4:45PM, ALNY.OQ - Alnylam Pharmaceuticals Inc at UBS Global Healthcare Conference
Question: Eliana Merle - UBS - Analyst
: Very exciting. In terms of the Huntington's program, you recently started a Phase 1 study. Can you tell us a bit more about what you're looking to
see from this study, what would be good data, and when can we maybe see this data?
Question: Eliana Merle - UBS - Analyst
: Great. Yeah, absolutely. And then when it comes to the Alzheimer's and CAA programs, I mean, we've seen target engagement as we've seen with
all of your programs, you're successfully able to reach the protein. But maybe how should we think about the timing for when we could see clinical
data and understand the effects on cognition?
Question: Eliana Merle - UBS - Analyst
: Got it. SP1 And it seems like you do have a growing pipeline. But from a BD perspective, can you tell us a little bit more about your philosophy from
here? Are there areas that you think that, for instance, we've seen a lot of development in the RNA editing space. Are there any sort of technologies
that you feel like would complement the Alnylam platform?
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NOVEMBER 12, 2024 / 4:45PM, ALNY.OQ - Alnylam Pharmaceuticals Inc at UBS Global Healthcare Conference
Question: Eliana Merle - UBS - Analyst
: Absolutely, that makes sense. And maybe just when it comes to thinking about profitability you're doubling your pipeline, major launch in
cardiomyopathy. How do we think about the outlook from here?
Question: Eliana Merle - UBS - Analyst
: Great. And as we think about the cardiomyopathy sort of sales force and the build-up there, just the US as well as ex-US, sort of where are you in
this build-up already?
Question: Eliana Merle - UBS - Analyst
: Okay. Great. And then ex-US, how are you thinking maybe about the commercial opportunity there for in cardiomyopathy relative to the US as
well as the investment.
Question: Eliana Merle - UBS - Analyst
: Okay. Great. And one thing we didn't touch on as it relates to TTR are the mixed phenotype patients. And I mean, we've heard a wide range of
estimates of what proportion? How does this play into some of the prescribing behavior?
Question: Eliana Merle - UBS - Analyst
: Great. Well, I think we'll end it there. But Jeff, Sandeep, thank you so much for the time and the insight.
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