CBIO - NASDAQ

Good morning. And thank you for joining the GlycoMimetics Fourth Quarter and Full Year 2022 Earnings Call. At this time, all participants are in a listen only mode. Following the management’s remarks, we will hold a question-and-answer session at that time, lines will be opened for you. I would now like to turn the call over to Christian Dinneen-Long, Company Council at GlycoMimetics. Please go ahead.

Good morning. Today, we will review our business updates and financial results for the quarter and full year ended December 31, 2022. The press release we issued this morning is available on the company's Web site at glycomimetics.com. This call is being recorded. A dial-in phone replay will be available for 24 hours after the close of the call. The webcast replay will also be available for 30 days in the Investors section of the company's Web site. Joining me on the call today from GlycoMimetics are Harout Semerjian, Chief Executive Officer; Brian Hahn, Chief Financial Officer; and Dr. Edwin Rock, Chief Medical Officer. Today's call will include forward-looking statements based on our current expectations. Forward-looking statements may include, but are not limited to, statements about the company's product candidate uproleselan, or our other pipeline programs, along with statements of our expectations regarding our operations, the conduct of our data from clinical trials, planned or potential development activities, regulatory interactions or submissions pre-commercialization activities, potential strategic collaborations and our cash position. Such statements represent management's judgment and intention as of today and involve assumptions, risks and uncertainties. GlycoMimetics undertakes no obligation to update or revise any forward-looking statement. For information concerning the risk factors that could affect the company, please refer to our filings with the SEC, which are available from the SEC or through our Web site. I'll now turn the call over to Harout.

Thank you, Christian, and good morning, everyone. In 2022, we made great strides in our continued organizational evolution as we aspire to become a commercial stage company poised to deliver important medicines to patients with significant unmet needs. To deliver on this aspiration, we are focused on three key areas: first, strengthening our leadership team to support commercialization; second, sustaining strong clinical progress for our pivotal Phase III study of uproleselan; and third, raising capital to continue the trial through a final analysis as patients continue to live longer. I'm proud to say that we have made significant progress in all three areas. At this crucial point in our company's life cycle, strong clinical and commercial leadership is more essential than ever. We have hired highly capable and experienced leaders, including Dr. Edwin Rock as Chief Medical Officer; Deepak Kuvari as Vice President of Technical Operations; Bruce Johnson as Chief Commercial Officer; and most recently, Chinmaya Rath as our new Chief Business Officer. Ed provides critical oversight, strategic guidance and regulatory insight as we advance through the late stages of clinical development. While Chinmaya applies his expertise from over two decades as biotech and pharmaceutical companies to oversee our corporate strategy and early stage pipeline programs. In parallel, Bruce and his team remain hard at work educating the market on uproleselan's differentiated mechanism of action, driving awareness of the unmet medical needs in the relapsed/refractory AML and leading key commercial readiness activities to ensure we are well positioned to deliver uproleselan, if approved, to patients in need. Deepak and his team continue planning and preparing for commercial supply following recent completion of the API validation batches of uproleselan. Turning to our second key focus area, the execution of our pivotal Phase III study of uproleselan in relapsed/refractory AML. In this study, patients continue to live longer than expected, leading to a median follow up prior to primary analysis in our study that is currently greater than 25 months, which is remarkable. As a reminder, the final survival event trigger based on projections that reflect this longer follow up duration is currently expected to occur in the first half of 2024. Last fall, based on slower accumulation of survival events, we recognized an ethical need to address the possibility that this slowdown in survival events may relate to benefit from uproleselan study therapy. Following consultation with medical experts on the blinded pool data, we aligned with the FDA to establish an interim utility analysis in our Phase III study protocol at approximately 80% of the planned survival events. In preparation for this interim analysis, our team partnered with the clinical trial community to ensure thorough data cleaning ahead of the final database lock. The interim analysis, which employed a very high statistical threshold in order to preserve the statistical integrity of the originally planned final overall survival analysis was conducted in February of this year 2023. The independent data monitoring committee recommended we continue to the originally planned final analysis while noting there were no safety concerns. Later in the call, Ed will provide additional context on the high threshold used at interim analysis and how this preserves statistical integrity for the final analysis. As noted earlier, median follow-up for our study of uproleselan is currently greater than 25 months. Given this extended median follow-up duration, we have assessed other relapsed/refractory AML studies for the time from randomization to primary analysis. To our knowledge, as our study continues to mature, it is on track to have the longest follow-up of any study in relapsed/refractory AML at the point of primary analysis at potentially more than three years. We are encouraged by this continued demonstration of uproleselan's safety profile and remain optimistic about uproleselan's potential in light of the extended median follow-up duration in our study and the fact that patients continue to live longer than previously expected. Finally, we're pleased to announce in February that we completed a $32.9 million financing, which is expected to extend our cash runway to the end of 2024. We're encouraged by the support we received from a science driven investor base, recognizing the potential of our Phase III study and clinical programs. This funding ensures our organization is well positioned to continue advancing our pivotal Phase III study of uproleselan in relapsed/refractory AML. On today's call, I'm pleased to be joined by our CFO, Brian Hahn; and CMO, Dr. Ed Rock. Ed, I'll pass it over to you to share more detail on the interim analysis and other recent study developments.

Thanks, Harout, and thanks to everyone for joining our call today. As Harout mentioned, we remain encouraged by the recently completed interim utility analysis of our Phase III trial of uproleselan for relapsed and refractory AML. Interim analysis sought to assess whether the prolonged survival follow up that we have been observing could be due to uproleselan benefit in this population. With FDA concurrence, we designed interim analysis to use a conservative modified obrine flemming method to generate a high statistical threshold for stopping the study early. This high bar to an early stop had two intended outcomes. First, the positive result based on 80% of planned survival events would by itself represent compelling evidence of uproleselan benefit. Second, if the study was to continue then about 95% of the study's statistical significance, also known as ALFA, would be preserved for the subsequent final survival analysis of the primary endpoint. As mentioned, implementation of the interim analysis gave GlycoMimetics wto shots on goal with the uproleselan study. Keep in mind, those two shots were not designed equally as the interim analysis used roughly 120th or about 5% of available ALFA and the final analysis is planned to use the rest, about 95% of total available ALFA for the study. So these two shots on goal are aiming at different targets with about 5% of ALFA used to target for interim analysis was very small. Conversely, as the trial matures toward final analysis, the statistical boundary aligns much more closely to the original analysis designed as part of the original study protocol. The key takeaway here is that uproleselan remains a first in class drug candidate with potential broad utility, a clean safety profile and the possibility to demonstrate survival prolongation in patients with AML. Blinded pooled data show that the transplant rate in the study is above the 31% transplantation rate observed in the preceding Phase I/II trial. Additionally, we observed a patient dropout rate of about 3%. This low dropout rate gives us confidence that loss to follow up is not contributing to slow event accumulation in the trial. Rather, our biologic hypothesis is that uproleselan study treatment leads to deeper, more durable AML disease remissions that could help bridge more patients to and through stem cell transplantation, possibly resulting in improved overall survival. In summary, this study population continues to live longer than expected based on historical benchmarks seen in other relapsed and refractory AML studies. To our knowledge, this trial remains on track to have the longest follow up of any study in relapsed and refractory AML at its time of primary analysis, now at potentially more than three years. We look forward to providing our final overall survival analysis shortly after reaching the final survival events trigger currently projected to occur in the first half of 2024. In addition, we continue to monitor primary event accumulation closely. At GlycoMimetics, we remain focused on delivering new options to patients who continue waiting for a new and better standard of care therapy for this highly lethal disease. Now I'll hand over to Brian for an overview of financial results.

Thank you, Ed. As of December 31, 2022, GlycoMimetics had cash and cash equivalents of $47.9 million as compared to $90.3 million as of December 31, 2021. Expenses for the second half of 2022 decreased to $19.2 million as compared to [$30.4 million] over the first half of 2022. From the fourth quarter of 2022 until February 2023, we sold 11,776,784 shares of common stock under our existing at the market sales facility, raising a total of $32.9 million in net proceeds. These additional proceeds are expected to extend our cash runway to the end of [2020]. We intend to use our capital resources to advance the clinical development of and prepare regulatory filings for marketing approval of uproleselan and the plan for uproleselan's potential commercialization to continue the evolution of GlycoMimetics into a commercial stage company. Company's research and development expenses decreased to $5.9 million for the quarter ended December 31, 2022 as compared to $12.9 million for the same period in 2021. Decreased expenses were primarily due to the lower global Phase III clinical study and development costs as patient enrollment ended in November 2021. The company's general and administrative expenses increased to $4.7 million for the quarter ended December 31, 2022 as compared to $4.5 million for the fourth quarter of 2021, primarily due to higher pre-commercial expenses for uproleselan in 2022, offset by lower grant date fair market value for equity awards issued in 2022. I'd now like to turn the call back to Harout.

Thank you, Brian. This is a very exciting time for GlycoMimetics. We're encouraged that the recent interim analysis of our Phase III study found no safety concerns with uproleseran treatment, and the study was recommended to continue the final analysis. Patients in this study continue to live longer than expected and to our knowledge, it is on track to have the longest follow up of any study in relapsed/refractory AML at the point of primary analysis at potentially more than three years. With our recent $33 million financing, we are well positioned to continue the study to its final events trigger expected to occur in the first half of 2024. And we look forward to sharing updates with you on future calls as we work to become a commercial stage company capable of delivering important new medicines to patients who need them most. I'd now like to open the lines to Q&A. Operator?

[Operator Instructions] Our first question comes from Boris Peaker with Cowen.

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?

So maybe let me take your first one -- 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.

[Operator Instructions] We have a question from Roger Song with Jefferies.

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?

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?

Maybe just a follow up question. Understanding you are focusing on upro for the Phase II, Phase III R/R AML trial. And since you have some time about a year before you will have the top line data, what are the key backlog activity you have been -- you and the team have been preparing for the data and the potential registration commercial?

I mean, one of the things which -- now that the interim analysis, which was very important for us to do for ethical considerations is behind us and we have an updated look at when the full events would potentially happen. We continue to leverage this time to also engage with the medical community. As you know, uproleselan has a very unique mechanism of action, it's novel, it's complementary in terms of tackling an extrinsic chemo resistance pathway that can potentially complement other pathways in diseases that are as heterogeneous as AML. So we continue to really engage with the medical community through both Ed's team and Bruce's team to really make sure that uproleselan and E-selectin antagonism as a mechanism is really in the limelight of the medical expert community. So that's one of the things that we continue to do and this timing actually allows us a bit more ability to do some of that in a very smart, cost effective manner.

Thank you. There are no more questions in the queue. I'd like to turn the call back to Mr. Semerjian for closing remarks.

Thank you, operator. And thank you for everyone for joining our call today. We look forward to keeping you up-to-date on GlycoMimetics. Have a great day.