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: Jessica Fye - JPMorgan Chase & Co. - Analyst
: Thank you. Thanks for the presentation. So we're in 2025, the company had laid out the P5 by 25 goals. Where do you stand on
delivering on those targets?
Question: Jessica Fye - JPMorgan Chase & Co. - Analyst
: Great. So I mean, turning to the '25 guidance that you put out there, it looks like it came in ahead of consensus. You're reflecting
contribution from cardiomyopathy, you've already established AMVUTTRA in polyneuropathy. Anything you can share on how you're
thinking about growth this year in polyneuropathy specifically.
Question: Jessica Fye - JPMorgan Chase & Co. - Analyst
: So maybe sticking with PN for just one more moment. You've had wea now in the market competing for what I guess about a year,
what are you seeing there? What defines the patients who opt to go on? (inaudible)
Question: Jessica Fye - JPMorgan Chase & Co. - Analyst
: And then sticking with the guidance and the contribution from cardiomyopathy. How would you characterize the degree of
conservatism that's embedded in the assumptions on the CM aspect of the guidance.
Question: Jessica Fye - JPMorgan Chase & Co. - Analyst
: One question, we get a lot from investors as it relates to this product AMVUTTRA broadening into a larger patient population. The
cardiomyopathy setting from polyneuropathy is, how do you think about the evolution of net price, any kind of color or context you
can share for folks, I keep hearing from investors that people are all over the place.
Question: Jessica Fye - JPMorgan Chase & Co. - Analyst
: Is there any update to your expectation for an ADCOM for AMVUTTRA and cardiomyopathy?
Question: Jessica Fye - JPMorgan Chase & Co. - Analyst
: Maybe switching to the pipeline and vutrisiran where I think you've talked about the back half of the year for the next Phase 2B
study. What would represent a win? Like what do you want to see on top of multiple background agents?
Question: Jessica Fye - JPMorgan Chase & Co. - Analyst
: I guess recognizing that the ultimate goal is to have the information to power the cardiovascular outcomes trial, just to kind of set
investor expectations a little bit. Should we expect kind of a similar number of millimeters of mercury delta when we have multiple
background in the mix or should we expect something different than what we've seen from the prior studies?
Pushkal Garg - Alnylam Pharmaceuticals Inc - Chief Medical Officer, Executive Vice President - Development and Medical Affairs
Yeah, look in general, we know that as you add on drugs, on top of multiple, right, as a monotherapy, we're seeing 15 millimeters
plus blood pressure reduction as you're looking at combinations that inherently gets to be a smaller effect.
But overall, remember we're looking at a short term period of time whereas this is going to be a chronic therapy. And so we expect
those benefits to magnify over time. But I think generally people believe anything over 5 millimeters of mercury leads to a pretty
substantial impact in terms of mortality in this disease.
Question: Jessica Fye - JPMorgan Chase & Co. - Analyst
: So maybe switching to mivelsiran, what data thus far for that asset is most exciting to you?
Question: Jessica Fye - JPMorgan Chase & Co. - Analyst
: And maybe we just with the last kind of minute here. I guess a little bit bigger question with the pipeline, but what kind of targets
and diseases do you see as most attractive as you kind of take this platform forward?
Question: Jessica Fye - JPMorgan Chase & Co. - Analyst
: Great. Thank you.
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