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: Joseph Schwartz - Leerink Partners - Analyst
: Great, thanks so much. Congrats on all the progress and hi to Dae Gon. Look forward to working together again. I was wondering if you could talk
some more about the quantum of data you expect to report from the 12 mg cohort of Pioneer mid-year.
What range of follow-up duration do you anticipate we'll see for the 16 patients who've been enrolled, and will we get any data on the markers of
homolysis in addition to fetal hemoglobin data for these patients?
Question: Joseph Schwartz - Leerink Partners - Analyst
: Great, thanks. That's super helpful. And then, I guess, does the observed dosing provide you with a specific data on the number of doses that
patients are receiving real time, and do you happen to have any of that data handy that you can share with us?
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MAY 01, 2025 / 12:00PM, FULC.OQ - Q1 2025 Fulcrum Therapeutics Inc Earnings Call
Question: Joseph Schwartz - Leerink Partners - Analyst
: Yeah, I'm just, yeah, I guess any, is there any more colour that you can provide on, the timing of the doses? Are they all within the prescribed time
frame and I guess how many doses have occurred to date?
Question: Joseph Schwartz - Leerink Partners - Analyst
: Yeah, that's excellent. We're looking forward to your updates this year. Thanks for the inputs.
Question: Matthew Biegler - Oppenheimer & Co. Inc. - Analyst
: Oh great. Hey everyone, I'll send my congrats as well. Thanks for the updated color here. The baseline HBF is a bit higher, I think at 7% than we
anticipated given like the severity of disease for these patients at entry. So number one, do you think you've gotten a representative sample of the
broader demographic you're going to be trying to treat here when we do get the data and number two.
Do you think poster should work equally well or perhaps even better in patients with higher baseline HPF?
Question: Matthew Biegler - Oppenheimer & Co. Inc. - Analyst
: Okay, awesome. That makes a lot of sense. If I could just maybe squeeze one quick one minute on the guide going from, I guess mid-year to early
3Q, is that just kind of the nuts and bolts of the execution of the clinical trial, or you actually want to get more follow up on, potential disease
modification and points, such like that. Thank you very much.
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MAY 01, 2025 / 12:00PM, FULC.OQ - Q1 2025 Fulcrum Therapeutics Inc Earnings Call
Question: Unidentified Company Representative 1 - - Analyst
: Great thank you. Thank you very much for taking the call and I apologize, I'm bouncing a little bit between calls today, but I just want to get a sense
for and I apologize if this was asked, but what is, what's a win for you guys from the 12 week data?
Obviously we saw somewhere around 10% there's going to be some differences in terms of the patients who are enrolled maybe the baseline, he
will be different, but what are you guys really focused on from that data set to know that this is, working? And in the ballpark you're looking for.
Question: Unidentified Company Representative 1 - - Analyst
: Great. Excellent. Thanks and thanks guys. It really makes a lot of sense. Look forward to the data
Question: Kristen Kluska - Cantor Fitzgerald - Analyst
: Hi, good morning, everybody. Very encouraging to see you ended up with 16 patients in this cohort, and that enrollment overall seems to be going
a lot faster than a lot of us expected. How much of this, in your opinion, could just be attributed to the loss of Oxbryta and different dynamics or
how much of it is attributed to getting more sites on board?
Question: Kristen Kluska - Cantor Fitzgerald - Analyst
: Okay thanks and I know you're you talked about that you don't currently have reason to believe that baseline levels will determine your responses
but has there been any work or research done to understand why certain patient populations may present as more severe and why these patients
don't respond to other therapies that are part of your inclusion exclusion criteria.
And I think ultimately where I'm trying to go with my question. If you're able to show response in a population that's already deemed to be quite
severe and tougher to treat with any intervention, how does that help us understand how the data can translate potentially to a more traditional
all-comer's population.
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MAY 01, 2025 / 12:00PM, FULC.OQ - Q1 2025 Fulcrum Therapeutics Inc Earnings Call
Question: Kristen Kluska - Cantor Fitzgerald - Analyst
: Thanks Alex and Iain.
Question: Anish - RBC Capital Markets - Analyst
: Good Morning Alex and team. It's Ani on for Greg. Thanks for the updates this quarter and for taking our questions. Just a couple from us first, given
the shifts at the FDA, how are you thinking about the impact to Pay's broader developments such as on endpoint selection, HBF as a surrogate
marker, which I know you've talked about before, and even the overall development timelines? What's your take on the current set up with
regulators? And then just quickly in your deck you note novel HBF inducers in your discovery pipeline. Could you share how you're thinking about
differentiating from other mechanisms in the landscape such as wis degraders, DNM-T1 inhibitors, etc. to bring that novelty? Thanks so much.
Question: Anish - RBC Capital Markets - Analyst
: Great, thanks so much
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