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: Matthew Biegler - Oppenheimer & Co - Analyst
: Maybe just a high-level question, Alex, on the platform tech and how you're thinking of seat in the pipeline with new wholly-owned assets. How
do you think about the difference between keeping it wholly-owned versus partnering something out?
And for maybe next-gen assets, are there certain disease therapeutic areas that you want to focus in? I mean, should the focus be hematology
going forward? Or are you kind of open to exploring new avenues?
Question: Dae Gon Ha - Stifel, Nicolaus & Company - Analyst
: Congrats on the progress. I guess two questions on pociredir, specifically. Going back to your 10-Q filing, it seems like on the Risks section, you
have been having this information or the language around request for information to define a potential risk in any further studies that may be
conducted in healthy volunteers. You are announcing your intention to do another healthy volunteer study. So curious what exactly was the
feedback from the FDA post OXBRYTA withdrawal that led you to having this trial run sort of concurrent with the PIONEER study.
And then second, I guess, in terms of the African conduct, just curious what have been sort of the latest FDA feedback on whether or not that meets
the standards for the FDA review?
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NOVEMBER 13, 2024 / 1:00PM, FULC.OQ - Q3 2024 Fulcrum Therapeutics Inc Earnings Call
Question: Edward Tenthoff - Piper Sandler - Analyst
: So following up on Dae Gon's questions, I wanted to get a little sense in terms of -- can you provide any better guidance on how this data from
PIONEER may roll out next year. Will you report it all together? Would you split the 12 mg and 20 mg and kind of reported as it's available? And
how quickly do you envision being able to transition from that into a registrational study?
Question: Rick Miller - Cantor Fitzgerald - Analyst
: This is Rick Miller, on for Kristen. Just a quick follow-up on the healthy volunteer studies. You've talked about initiating. How are you thinking about
dose in these trials? And how this relates to kind of the previous Phase 1 trial dosing strategies you looked at? And in terms of main outcomes here,
will this be mostly looking at safety? Or are there any kind of PK/PD measures you're interested in getting a look at?
Question: Gregory Renza - RBC Capital Markets - Analyst
: Just a couple from us, Alex, that just as you reflect on the losmapimod experience, how are you using that journey to inform the value proposition,
the risk assessment of future programs, not just with pociredir, but also, of course, with the early discovery programs.
And then maybe secondly, if you could just comment a bit about how you expect to balance the early discovery programs internally and with
camp that you're looking at versus the thought of looking externally for assets.
And maybe I'll sneak a third one, if I may, for Alan. Maybe related to all of this, as you've talked a bit about resources and spend for 2025. How does
that allocation pan out? How should we be thinking about what's implied in some of the runway there?
Question: Joori Park - Leerink Partners - Analyst
: I'm Joori Park, dialing in for Joe. So this summer at EHA, you presented a map of sites you were onboarding. And I believe there were 11 in the US
and two ex US. I was curious what your latest thoughts were on these sites? And then separately, I believe that you plan to activate up to 20 sites.
That's what you said on our previous call, with more sites to activate, which you've mentioned on today's call. So I was curious if you've identified
the remaining seven sites and where you are on the onboarding process for them.
Question: Corinne Jenkins - Goldman Sachs - Analyst
: You mentioned the pociredir program is fully funded. I guess does that apply to a broader scope of patients and is currently allowed within the
trial? Or would that be specific to this higher risk group you're currently focused on?
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