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: Gregory Allen Harrison - BofA Securities, Research Division - Analyst
: So how does this update impact your view of the therapeutic potential of increasing progranulin above normal levels, both in this indication and
FTD, and also in other indications that you're looking at? Do you think that this -- that there's good read across to those other indications based on
this data set?
Question: Gregory Allen Harrison - BofA Securities, Research Division - Analyst
: Got it. That's helpful. One follow-up maybe. Could you comment on the difference in the level of severity for the C9orf72 patients versus GRN and
what that means for how we would interpret the CDR data? How noticeable is this change in this population compared to your previous updates?
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: I had another clarification question between the PR and what I'm seeing in the presentation. Just in the press release, it says at 49 weeks the CSF
levels of progranulin is 1.7 nanograms per ml, which would imply a decrease. But in your presentation, I mean, that same data point on Slide 15,
looks like it's coming in at around about 9 or something like that. So I'm assuming that the presentation is correct and maybe this version of the
press release that I'm looking at has an error in it. Can you just clarify that? And then I have a few follow-ups.
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: Yes. It's just the week 49 data point in the press release that I'm seeing that 1.7 nanograms per ml. The standard error of the mean of 0.9. And on
Slide 15, I think the correlating data point looks like it's coming in, I'm just reading off the graph, looks like it's coming in around, I don't know, 8.5
or 9 nanogram per ml. I'm assuming the presentation is correct and that the press release version that I've seen here is...
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: Okay. Cool. And then I did have a question, again just relating to the CDR sum of the boxes data. I'm curious, again, on like the use of the repeated
measurements model here, just given the variability specifically at that 3-month -- the patients that we have data points for 3 months. Like if the
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data cutoff for data that I'm seeing here is June 15, 2021, like why wouldn't you just use the 9-month data that you probably should have for that
patient and use that instead of a repeated measures model?
Question: Myles Robert Minter - William Blair & Company L.L.C., Research Division - Analyst
: No, no, no, it's still good. It was just Slide 18, there's definitely 1 patient that you're showing 3 months' worth of data for in the CDR, NACC FTLD.
Like if the data cutoff was June 15 last year, you should probably have internally at least the 9-month real data for that patient. And I'm just wondering
why in today's data cut, we're not seeing that and that's leading us to questions about the validity of the statistical model here when you probably
have the real data internally.
So maybe I can reshape that question. When will we see that data?
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