Crescent Biopharma, Inc. Q2 FY2023 earnings call

• FDA cleared a protocol amendment for the Phase 3 uproleselan study enabling top line results by the end of Q2 2024. Final analysis will have over three years of median follow-up and incorporate post-transplant data. • Continued pediatric development of uproleselan with FDA agreeing to initial pediatric study plan, NCI initiating a Phase 1/2 dose escalation study, and first pediatric patient treated in an investigator-initiated trial. • Plan to initiate a Phase 1a study of GMI-1687 in Q3 2023 to evaluate safety, tolerability, and pharmacokinetics in healthy volunteers. • Cash runway to fund operations late into Q4 2024, allowing execution of clinical development plan.

• Pivotal Phase 3 trial of uproleselan on track for top line readout by end of Q2 2024. • Plan to begin Phase 1a study of GMI-1687 in the coming weeks. • Cash runway extends into late Q4 2024.

• Forward-looking statements involve assumptions, risks, and uncertainties. • Refer to SEC filings for risk factors affecting the company.

Q: For the Phase 2/3 upro NCI trial, will they also be running a time-based analysis similar to what you guys are running for your Phase 3 trial? Or are they still keeping with their original plan for their analysis? And then also, just like a separate question, but for the pediatric patients for upro what's the plan following this Phase 1/2 trial? Will the NCI continue to run trials? Or will you guys don't then take it after the Phase 1/2?

A: Thank you, Nick. Thanks for the question. Maybe I'll start it off and then hand -- move it over to Ed. Regarding the NCI trial and the Phase 2/3 what we know is they're still on -- consistent with what we've communicated last quarter, is that they have not reached their events. So I think we're going to have to wait for that and see where they go next. They haven't -- we're not aware of anything beyond that. So what we know is that a predetermined number of events, the event has not been reached. So that's been also communicated to us recently. Maybe Ed, you want to add more color on that one and then on the pediatric plan.

Q: Regarding the uproleselan, the R&R AML to the study, had the team take another look at the data. And how did that track in your modeling? Understand now it's time based but have you ever done additional kind of data look? I think on the core, you mentioned most of the survivor, their post transplant or any additional color, you can provide to us A: Yeah, thank you, Roger. Yeah, I mean, the trial continues. Patients continue to live longer in this trial. So obviously, as you know, we're blinded. We do take a look at the pooled blinded data. And we're excited that we have now this option of the time-based analysis should the 295 events not be reached by next year, we believe we're going to have a mature database, given the three years median follow-up, given the two years plus of post-transplant follow-up as well, for the vast majority of patients, that we're going to have a mature database. So things continue to be on track. Patients continue to live longer. Obviously, we don't know who's on what arm, but we're very encouraged by this updates and the fact that now we have the option of the time-based analysis on top, gives us certainty, clarity, and of course, on a database that's going to be mature.

Q: Regarding the 1687, so what do you expect to speed up healthy volunteer data and how would that support the next step? And do you have any timing and the maybe the plan -- strategic plan in terms of you will take this on your own or you are seeking a partner to move forward?

A: Yeah, thanks. Excellent question, Roger. I mean, as you know, 1687 for folks on the phone, this is clear this other asset that we have cleared IND back in June of last year. And given where the markets were, the financial constraints we had, we really had to double down on our Phase 3. We're very excited now to be able to be in a situation where we're able to start the Phase 1a of 1687, a compound we believe in, we have the deep expertise, as you know, in this area in sickle cell, and bringing that expertise on our second generation, E-selectin antagonist, that is potent and subcutaneously bioavailable. So the first step of that, Roger, as you know, is that single ascending dose, as Ed mentioned. And really, it's a healthy volunteer study. So I don't want to read more into it, than then what it is. It's really to give us the PK/PD safety signals, which are really needed at first step. And quite honestly, regardless of what indications we go, that single ascending dose trial and the Phase 1a is very consistent and the same. So we're going to be doing this, typically these volunteer trials they don't take that much time but it's usually in the month. So sometime, I would say next year we should have that information. It's just too early to tell now. Let's get started. As we're announcing today, we are planning to start. We haven't started yet. So let's get started and we'll keep the market updated about it. But I'm very excited about the ability to start 1687 and put yet another GlycoMimetics product in a first-in-human study in the marketplace.