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: Sean Laaman - Morgan Stanley - Analyst
: Fantastic. And we'll dig into that in a minute. But I've got three macro questions that I'd like to ask you. And the first one is, with China's rise in
biotech innovation, how do you think about Disc Medicine's competitive position here, and will this influence your R&D and business development
strategy?
Question: Sean Laaman - Morgan Stanley - Analyst
: Wonderful. And how are you currently leveraging AI or thinking about AI's future disruption?
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SEPTEMBER 10, 2025 / 5:05PM, IRON.OQ - Disc Medicine Inc at Morgan Stanley Global Healthcare Conference
Question: Sean Laaman - Morgan Stanley - Analyst
: Okay, wonderful, thank you. And what's been most impactful on Disc from the regulatory side? Has it been interactions with the FDA? Has it been
MFN, it's probably a bit early, or tariffs?
Question: Sean Laaman - Morgan Stanley - Analyst
: Right. Thank you. Maybe to get more specifically on Disc. So starting with bitopertin, you expect to submit the NDA in EPP next month, I believe.
How are you thinking about the launch plans and the commercial strategy?
Question: Sean Laaman - Morgan Stanley - Analyst
: So would you be able to walk us through -- I think you said 14,000 patients. Just walk us through how you think you'll approach that, what the
actual commercial opportunity might prove out to be?
Question: Sean Laaman - Morgan Stanley - Analyst
: So it sounds out of the gate that you've got a pretty accessible patient population that might be on a therapy. That's the way that you're approaching
it, hope that the market will build out from that initial subset of patients.
Question: Sean Laaman - Morgan Stanley - Analyst
: Yeah. Thank you. I guess, based on the data observed today, can you provide more color on how you're thinking about the commercial dosing
strategy as well as expectations around price?
Question: Sean Laaman - Morgan Stanley - Analyst
: Got you. Thank you, John. What are the key learnings from the HELIOS long-term extension trial regarding sustained PP9 reductions and improvements
in quality of life measures?
Question: Sean Laaman - Morgan Stanley - Analyst
: Okay, and what are your expectations for the confirmatory APOLLO trial in terms of enrollment timeline and the trial needing to be well underway
for bitopertin to be approved?
Question: Sean Laaman - Morgan Stanley - Analyst
: Great. And I've got a couple of questions on the APOLLO co-primary endpoint. What are you hoping to show in terms of PP9 reduction? How
confident are you in the study's ability to demonstrate stat sig average monthly time in sunlight?
Question: Sean Laaman - Morgan Stanley - Analyst
: Wonderful. And maybe for investors that might be less familiar, just talk about the severity of the disease, so how it compromises life, and maybe
draw a comparison with bitopertin to what is currently on the market.
Question: Sean Laaman - Morgan Stanley - Analyst
: Wonderful. Thank you. I might jump over to 0974. So the Phase 1b data showed that greater than 50% of patients receiving concomitant JAK
inhibitor therapy achieved major hematological responses. How might this influence treatment paradigms for MF patients with anemia?
Question: Sean Laaman - Morgan Stanley - Analyst
: Got you. And can you discuss the decision to update the RALLY-MF trial protocol?
Question: Sean Laaman - Morgan Stanley - Analyst
: Wonderful. Thank you, John, and what are our expectations to the initial data readout in the Phase 2 RALLY-MF trial, which I think is in 4Q? And
what would you consider a clinically meaningful outcome?
Question: Sean Laaman - Morgan Stanley - Analyst
: So wonderful. And what biomarkers or baseline characteristics might help identify which MF anemia patients are most likely to benefit from 0974?
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SEPTEMBER 10, 2025 / 5:05PM, IRON.OQ - Disc Medicine Inc at Morgan Stanley Global Healthcare Conference
Question: Sean Laaman - Morgan Stanley - Analyst
: Sure. And just thinking about the competitive landscape, has there been any recent updates on the competitive front and how you view the
positioning of 0974 in the MF space?
Question: Sean Laaman - Morgan Stanley - Analyst
: Sure. Thank you, John. To move on to 0974 in non-dialysis-dependent CKD. With the MAD Phase 1b data expected in 4Q, what will you be specifically
looking for, for this to select the right Phase 2 dose?
Question: Sean Laaman - Morgan Stanley - Analyst
: Sure. And can you talk a little bit about the mechanism of 0974 and whether the differentiation from current standard of care in CKD?
Question: Sean Laaman - Morgan Stanley - Analyst
: Sure, sure. And I guess, sort of back to the population, just to clarify, could you discuss any potential biomarkers being evaluated to identify NDD
CKD patients most likely to respond to 0974?
Question: Sean Laaman - Morgan Stanley - Analyst
: Sure, sure. Wonderful. And what are your thoughts on potential combination approaches with 0974 still in CKD?
Question: Sean Laaman - Morgan Stanley - Analyst
: Wonderful. Thank you. And moving on to 3405. You more recently initiated Phase 2 in PV. The Phase 1 data showed meaningful reductions in
hematological parameters, including hemoglobin and hematocrit. But how might these translate to clinical benefits in PV patients?
Question: Sean Laaman - Morgan Stanley - Analyst
: Wonderful. Thank you. And what are you expecting or what should we expect from the Phase 2 data to PV next year?
Question: Sean Laaman - Morgan Stanley - Analyst
: Wonderful. Thank you. And are there other iron overload conditions that you might be able to throw 3405 at?
Question: Sean Laaman - Morgan Stanley - Analyst
: Sure. And coming up to time, but a couple of more questions. So the first one, just your balance sheet position, cash position, and sort of how that
ties up with sort of commercialization.
Question: Sean Laaman - Morgan Stanley - Analyst
: Okay. Wonderful. And final question is, is there something I should have asked that I didn't?
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SEPTEMBER 10, 2025 / 5:05PM, IRON.OQ - Disc Medicine Inc at Morgan Stanley Global Healthcare Conference
Question: Sean Laaman - Morgan Stanley - Analyst
: Wonderful. Thank you for coming, John. Appreciate hosting you.
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