CRESCENT BIOPHARMA, INC. Q4 FY2022 earnings call

• Strengthened leadership team by hiring key executives like Chief Medical Officer, Vice President of Technical Operations, Chief Commercial Officer, and Chief Business Officer.
• Pivotal Phase III study of uproleselan in relapsed/refractory AML has a median follow-up >25 months, with interim analysis in February 2023 showing no safety concerns and recommending continuation to final analysis.
• Completed a $32.9 million financing in February 2023, which extends cash runway to the end of 2024 to advance clinical development and commercialization of uproleselan.

No specific product segment financial performance discussed as the transcript does not break down revenue contribution by product segments.

• The final survival event trigger for the Phase III study of uproleselan is expected to occur in the first half of 2024.
• The $32.9 million financing ensures the ability to continue the Phase III study of uproleselan in relapsed/refractory AML.

• Uncertainties related to forward-looking statements including clinical trial outcomes, regulatory interactions, strategic collaborations, and cash position as per SEC filings.

Q: This is Nick on for Boris. Just two quick ones for me. For the NCI sponsored trial in the frontline setting, when do you expect data for this to come out, is it reasonable to assume that it will be slightly delayed or pushed back similar to your Phase III? And then the second question is what's going on with the GMI-1687, are you planning to start a Phase I trial or are you looking for a collaboration partners with that?

A: So maybe let me take your second one first. So on 1687, as you guys recall, we have cleared IND back in June of last year and we're very excited about the potential of 1687. At this point, we're really doubling down on uproleselan to really get it across the finish line. So if there is an appropriate partnership then that's always a possibility. But at this point, we are really focusing on uproleselan. But if there's any changes, we will definitely communicate that. Regarding the NCI, that's an excellent question as well. As everybody knows on the call, this is an NCI sponsored trial in the frontline fit for chemotherapy patients. And that trial is an EFS as its primary endpoint in the Phase II portion of this adaptive Phase II/III. And the intent was that there was a pause in the enrollment of the Phase II with 267 patients enrolled as of December 2021 and the idea was that there was a one year pause until the EFS -- there was enough time for the EFS rates to mature, and we were supposed to get an update after that. Until now, Nick, we have not received any updates from NCI and we're at end March 2023. So it is reasonable to think that the EFS rates are probably not coming at the rate that originally was thought, which wouldn't be shocking because we're seeing something similar in the Phase III. But just to be clear, the NCI has not communicated officially to us any update on that trial. But that's kind of where -- to your question, is it reasonable? We think it is reasonable to think the same thing is happening.

Q: Maybe two questions from us. For maybe, Harout, you can add, you can give us some color how your team or the statistical team, predict the OSC event timing? And what have been changing or the driver for you to kind of keep pushing the data understanding you're probably seeing the blinded data in terms of MRD negative or the transplant rate or dropouts. So maybe just give us a little bit of color around the predictive model you have?

A: So as previously communicated, we have worked with our statistical external partners to have a robust projection tool that takes into consideration all the events, the different subgroups of what patients are in. As we know, if you're an MRD negative versus an MRD positive, it's a different outlook. If you're a transplant patients versus not, you'd see a different outlook. If you're a late stage disease versus an early relapse patient, it's a different outlook. So we took all these different patient characteristics that we had previously disclosed as well in our Phase III just to compare it with the Phase I/II and build this model. And from the beginning, we started communicating when the projection for the full event trigger would be. If you might recall, even when the full data was -- well, patients were enrolled back in November 2021. From then, we projected that this will be beyond year end 2022. If you recall at that time, although, many people were telling us, these are relapsed/refractory AML patients, typically they would live around six, seven months, so we should get data before that. But that wasn't what we were seeing from the beginning, from the get go. So we're very pleased with the way we've been projecting. And of course, as data matures, as events mature, as the event curve is something we look carefully at, we continue to update our projections and we communicated those projections to the market whenever there was a difference between what was previously communicated. So at the interim analysis, Roger, one of the things that has happened as well is our clinical operations team really did a good job in the data cleaning. As you can imagine, preparing for an interim analysis, we would have been ready to go should that threshold have been passed. So where we have a very robust database now. And based on that database robustness, we also updated our projections and now what we're saying is going to be within H1 2024. So we think the fact that patients continue to live longer is a good thing. We don't exactly know is it uproleselan, is it standard of care, is it a combination? Obviously, we're blinded, and we continue to be blinded to the data. But from what we see of this longer median follow-up from what we see in our pooled blinded data, we continue to remain very encouraged by the potential uproleselan has to relapsed/refractory AML patients and hopefully beyond that as well as the NCI data and other ISTs mature. Does that answer your question, Roger?