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: Thomas Smith - Leerink Partners LLC - Analyst
: Congrats on the data and all the progress. Just first on Bitopertin. It sounds like really great progress with the agency and ongoing engagement
with FDA. But because you're one of the first companies going through this process, I was wondering if you could just comment on the
acceptance notification and how it compares versus other typical sort of acceptance notifications.
Is there any indication of review types like priority review? Or was there any explicit acknowledgment that the FDA would be proceeding
under a more accelerated review process?
And then a second question, if I could, just on 974 and the competitive landscape in myelofibrosis. Like you're clearly seeing consistent
activity on top of JAK inhibitors with 974, including momelotinib, could you just comment on the mCALR targeted agents and how you think
974 fits into the treatment paradigm with those agents?
Question: Sean Laaman - Morgan Stanley - Analyst
: Congratulations on multiple fronts. Just to clarify, the major hematological responses were achieved regardless of concomitant JAK inhibitors.
But is there forward plans for specific combination strategies is the first question. And a similar question, opportunity for combination studies
with ESA, luspatercept or other agents to further enhance efficacy or broaden the addressable population?
And just lastly, first things first, of course, but beyond anemia and MF, what sort of other market opportunities might you observe with this
mechanism?
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DECEMBER 07, 2025 / 12:30PM, IRON.OQ - Disc Medicine Inc at ASH Meeting
Question: Rick Miller - Cantor Fitzgerald - Analyst
: This is Rick on the line for Kristen. Congrats on the RALLY-MF data and progress across the pipeline. Just one question from us. On fatigue
score, is there anything you can say even anecdotally about the TD High patients and the fatigue that they're experiencing and sort of relate
that to the baseline that you see the patients starting at?
Question: Rick Miller - Cantor Fitzgerald - Analyst
: Yes. Sorry, go ahead.
Question: Evan Seigerman - Bank of Montreal - Analyst
: So given the level of responses we've seen across both JAK and -- patients with both JAK and non-JAK background treatments, can you
talk about what the FDA might need to see beyond this data in a Phase III to support a broad label across JAK usage and across kind of
underlying hepcidin at baseline?
And secondarily, for patients that are nonresponders or super responders, can you comment on how they've reacted to dose escalation to
the 75 milligram, like how many patients have gotten to that level? And are they getting extra benefit from this escalation?
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DECEMBER 07, 2025 / 12:30PM, IRON.OQ - Disc Medicine Inc at ASH Meeting
Question: Tara Bancroft - Cowen and Company LLC - Analyst
: So I'm curious if you could tell us if baselines, especially absolute hemoglobin levels, if they differed at all between enrolled versus efficacy
evaluable patients and also by JAK use, if there's anything notable there at all if they were consistent.
And then the clinical relevance of these hemoglobin increases you saw. I know that they experienced less fatigue, which is great, but it
would be great to learn what you're hearing on how that impacts patient lives and their enthusiasm to continue or adopt therapy.
Question: David Nierengarten - Wedbush Securities Inc - Analyst
: I just had one left. When you look at anemia and IBD patients, are there any plans to stratify for baseline hepcidin or EPO levels in those
patients, just thinking about the experience with CKD.
Question: Danielle Brill Bongero - Truist - Analyst
: Congrats on the update. First, on the Phase II, I was wondering if you had the breakdown of response rates by background JAK use and
baseline transfusion dependency status readily handy. If not, any qualitative comments you can share on whether there were deviations in
response rates by the transfusion cohort when you look at background JAK use? And then I guess, looking at the optional continuation
phase participation, only 68% of completers continued on. Any thoughts on reasons why others did not?
Question: Rami Katkhuda - LifeSci Capital LLC - Analyst
: Congrats on the data. I guess do you think the slight differences in efficacy in the rux arm versus other JAK inhibitors is due to the small
sample size? Or could it ultimately reflect a potential ceiling on anemia benefit from targeting this pathway?
And then maybe secondly, on Bitopertin, the CNPV obviously accelerated the potential launch here. Could you provide a quick update on
the status of your commercial readiness and sales force?
Question: Derek Archila - Wells Fargo Securities LLC - Analyst
: Congrats on the update. Just a couple of questions. So just a follow-up to a couple of questions ago, but just any differences on the disposition
of the patients on momelotinib and pacritinib relative to the Jakafi patients? Just kind of reconciling that slightly lower response rate, but it
does sound like it's probably just small end. And also, what was the average Jakafi dose in the patients on Jakafi?
Question: Martin Auster - Raymond James - Analyst
: Curious if you guys had any observations in MF and the non-JAK inhibitor cohorts. I was just curious if there's any color on whether those
patients were those who maybe didn't meet criteria for JAK inhibition or if they've maybe been patients who previously exposed and a
discontinued drug?
And then secondly, I know you'll be thinking a lot about the registration path in MF next year. But I was curious for now if you could comment
on whether you're thinking about sort of a unified Phase III with multiple cohorts or if you'd be looking to maybe separate out the
non and/or low transfusion-dependent population from the high TD population and whether or not that can kind of
meaningfully accelerate Phase III enrollment and maybe help get to market a little faster?
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