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: Do Kim - BMO Capital Markets Equity Research - Analyst
: A question on the ENaC inhaled ASO, the Phase I data. Just wanted to get your thoughts on how the PK data tracked with the reduction of ENaC
mRNA as the dose escalated. Do you think you saw a dose response? And whether you had any thoughts on why the highest dose at 3 times weekly
didn't show much of a reduction?
Question: Do Kim - BMO Capital Markets Equity Research - Analyst
: Great. That's very helpful. And just a quick question on the ION541 for most types of ALS. Do you think that that therapy will also be effective in
the familial ALS, like SOD1 and C9ORF? And would you consider a strategy or Biogen would consider a strategy for a combo with the mutant specific
therapies?
Question: Gil Joseph Blum - Needham & Company, LLC, Research Division - Analyst
: This is Gil on for Chad. A little bit of a more general question. So as to the Akcea platform and the new commercial capabilities, how transferable
are they to -- across indications? Could you envision this platform being used, for example, to promote drugs in neurological indications?
Question: Gil Joseph Blum - Needham & Company, LLC, Research Division - Analyst
: That's very helpful. And maybe a question for Richard on ENaC. Just to remind us, is there something special in the ENaC agent chemistry for this
ASO that makes it more absorbable and a lot -- just a little bit of color on that.
Question: Gil Joseph Blum - Needham & Company, LLC, Research Division - Analyst
: All right. Excellent. And just one last question from me. So you mentioned this post-marketing study with SPINRAZA in gene therapy studies. I'm
assuming this is taking forward of a label expansion. Is that the right way to look at it?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Another one on vupanorsen. It looks like the dose is being studied in Phase IIb are generally higher than what was studied in Phase IIa. So thinking
about the non -- very high triglyceride population, kind of the broader high CV risk group. I'm just curious, when you look at those higher doses,
how much more efficacy you're hoping to achieve relative to the Phase IIa on endpoints like LDL-C?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Okay. Makes sense. And just kind of sticking with this product, and recognizing the lack of support for benefit in drugs that increase HDL. What do
you make of the declines observed with this product in HDL-C?
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: Just maybe one on something we haven't talked about the TMPRSS6 assets for beta-thal. Just wondering when we're going to see Phase II data
next year, whether you've got clarity on that. And just in terms of the primary endpoint, can you maybe explain the clinical -- the clinical
meaningfulness survey a 1 gram per deciliter increase in hemoglobin in this patient population and not the transfusion-dependent population?
Just trying to understand how to interpret that moving forward if this does go into a pivotal trial of some sorts?
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: Okay. Cool, that's helpful. Just -- yes, go ahead.
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: Understood. Last quick one from me, and I may be wrong here, but is there any reason why for the chronic hepatitis B program that GSK is running,
why they would have taken a non-LICA conjugated product in a Phase II as opposed to a LICA product?
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