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
: Great. Thanks, Brett, for that presentation. (Operator Instructions) Maybe starting out with donidalorsen in HAE. You had some strong data in 2021
and have talked about, including every 8-week dosing regimen in the pivotal study. Have you seen any PK/PD data or attack rate data that supports
that every 8-week dosing, whether in your preclinical characterization or in the clinic? And if -- and do you plan to share any of that data with
investors in 2022?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Got it. So when you made the decision to include bimonthly dosing in the Phase III, have you seen the kind of data accumulating with that dosing
frequency in your Phase II extension?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Got it. How long are you expecting it will take to enroll that Phase III trial?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Got it. Switching to fesomersen, why do you -- ESRD patients have an increased risk of thrombosis? How are they currently managed? And what's
the unmet need for this group of patients? And why do you believe fesomersen can do better than standard of care?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Got it. In addition to fesomersen, I think Bayer is also evaluating another Factor XI targeting asset in a separate Phase II study. Can you help us
understand the key differences between the 2 therapies?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Great. There's a question on the portal here about TTR, specifically TTR cardiomyopathy. So the question reads, what did Ionis learn from BridgeBio's
ATTR cardiomyopathy Phase III setback? What's the implication for Ionis? And can you highlight the difference in Ionis' trial design, including patient
selection, stabilized or use, et cetera?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Great. Maybe switching to AGT-LRx for uncontrolled hypertension, where are you with the 2 Phase II, I think one is on controlled hypertension,
another one in chronic heart failure. And could we see data from these trials in 2022?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Great. For AGT-LRx, is this actually something you think about as a good candidate for a new partnership? And if so, when do you think of it as the
best time to partner? Is that something that would happen after Phase II?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Makes sense. Another question on the portal here. Can you confirm if the deal with Aro Therapeutics is for muscle targeting. Is that -- was that what
you said in the presentation, I think the original announcement just said kind of extrahepatic tissue.
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Great. So with that, I think we are out of questions and about out of time. So thanks for the presentation, Brett and a thoughtful Q&A, and thanks,
everyone, for listening in.
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