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
: Not to belabor this, but just following up on the prior questions on ENaC. If the tox you noticed is not related to the platform or exposure in the
lung and it's not related to the target, what's your current thinking about what it might be related to?
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MAY 05, 2021 / 3:30PM, IONS.OQ - Q1 2021 Ionis Pharmaceuticals Inc Earnings Call
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Got it. Makes sense. If I can work in 2 other questions. Just on the Alexander disease program, can you talk about some of the efficacy measures
you have for ION373 that give you the confidence to advance that into a pivotal study and the size of that opportunity and just how well you think
the product might be able to address the market?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Got it. And maybe just a last question. Going back to the comment about reaching the peak royalty tier for Spinraza around midyear, similar to last
year. Can you just unpack that a little, some of the assumptions underlying that in light of the competition you're seeing in the U.S. from Evrysdi?
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: Just a question on Biogen sort of pushing forward a higher dose for the C9ORF program. Just wondering whether that's got any read-through to
VALOR and whether there's any room to maybe push the dose afterwards kind of in a Spinraza-like scenario? And for the FUS programs and the
ataxin programs that are earlier stage, is it likely that we'll see a push in the dosing in those early clinical studies?
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MAY 05, 2021 / 3:30PM, IONS.OQ - Q1 2021 Ionis Pharmaceuticals Inc Earnings Call
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: Okay. Yes. I guess that was my question, just like why the C9ORF program specifically they're testing a higher doses? I know they're not involved
in the FUS program, but even the ataxin program, like why wouldn't you just go with a higher dose here? Is there a risk of overshooting the
knockdown of this protein?
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