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 Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: I wanted to follow up on a prior question about the changes to CARDIO-TTRansform. You talked about them being driven by patient demographics.
Are you changing any of the inclusion or exclusion criteria and/or the regions you'll focus on for further recruitment in addition to just increasing
the sample size and treatment duration?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Okay. So I guess...
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Yes. Because I think in the past, you've talked about your expectations for maybe a 50-50 split between patients who are on background tafamidis
and those who are not even though you're not limiting background tafamidis. So is that still your expectation that, that will be the mix?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Okay. And I guess last one on this topic. Were these changes to CARDIO-TTRansform prompted by a recommendation from the DSMB or were they
made without DSMB input? And has there been an interim analysis in this trial at this point?
Question: Gary Jay Nachman - BMO Capital Markets Equity Research - Analyst
: So first, just another follow-up on eplontersen and adding more patients to the cardiomyopathy study. So why didn't you wait until the PN data
before making a decision since the data are coming soon? And it sounds like we will have some cardio data in there for some of the patients and
it potentially could have been informative to your decision. That's one.
And then also for Beth. The $20 million that you got from AZ for eplontersen development costs, is that the kind of number we should expect on
a quarterly basis or is there some upfront loading in there? And then lastly, just what are the next steps for 449, the PCSK9 program? Do you have
an idea what types of dyslipidemic patients you'll target in the next phase based on the Phase IIb data that read out?
Question: Gary Jay Nachman - BMO Capital Markets Equity Research - Analyst
: Okay. Great. And when is that study going to start? I may have missed it. I jumped on late.
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: Just in your prepared remarks on 449, you did say AstraZeneca is going to make a formal decision on the Phase III in the second half, but the
commentary on the call today seems to be like that's well and truly going ahead. So Brett, can you just clarify what prepping for Phase III development
actually means? Is that just getting these Phase II trials done or have you seen a protocol? And then, Beth, can you talk to the milestone structure,
if anything, on that deal if a Phase III is started up in the second half? And my last one is just on the muscle LICA program. Are you planning to take
a single asset into IND-enabling studies there? I know you have multiple collaborations and programs there. Or would you take multiple programs
into the IND tox studies?
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