This conference call transcript was computer generated and almost certianly contains errors. This transcript is provided for information purposes only.EarningsCall, LLC makes no representation about the accuracy of the aforementioned transcript, and you are cautioned not to place undue reliance on the information provided by the transcript.
spk06: Good morning, and thank you for joining the GlycoMimetics fourth quarter and full year 2022 earnings call. At this time, all participants are listed on the mode. Following the management's remarks, we will hold a question and answer session. At that time, lines will be open for you. I would now like to turn the call over to Christian Deneen Long, Company Counsel at GlycoMimetics. Please go ahead.
spk04: 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 website 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 website. Joining me on the call today from GlycoMimetics are Harit Samarjan, 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, upralesolin, or our other pipeline programs, along with statements of our expectations regarding our operations, the conduct of or 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 website. I'll now turn the call over to Haroud.
spk02: 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 three study of Eupalus RAN. And third, raising capital to continue the trial through 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 Tiwari 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 Eupolastiran's differentiated mechanism of action, driving awareness of the unmet medical needs in relapsed refractory AML, and leading key commercial readiness activities to ensure we're well-positioned to deliver Eupolastiran, 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 buprolasterine. Turning to our second key focus area, the execution of our pivotal phase three study of buprolasterine 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 benefits from eupalasterone 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 three 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 rupalacerin 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 Eupalasterin's safety profile and remain optimistic about Eupalasterin'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 upyletharan in the 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.
spk03: Thanks, Harut, and thanks to everyone for joining our call today. As Harut mentioned, we remain encouraged by the recently completed interim utility analysis of our Phase III trial of uprolessaland 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 uprolessaland benefit in this population. With FDA concurrence, we designed interim analysis to use a conservative, modified O'Brien-Fleming method to generate a high statistical threshold for stopping the study early. This high bar to an early stop had two intended outcomes. First, a positive result based on 80% of planned survival events would by itself represent compelling evidence of uprolacilin benefit. Second, If the study was to continue, then about 95 percent of the study's statistical significance, also known as alpha, would be preserved for the subsequent final survival analysis of the primary endpoint. As mentioned, implementation of the interim analysis gave glycomimetics two shots on goal with the euprolesolan study. Keep in mind, those two shots were not designed equally as the interim analysis used roughly 1 20th or about 5% of available alpha, and the final analysis is planned to use the rest, about 95% of total available alpha for the study. So these two shots on goal are aiming at different targets. With about 5% of alpha used, the 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 uprolacilin 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 pool 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. Our biologic hypothesis is that uprolacilin 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.
spk01: Thank you, Ed. As of December 31st, 2022, Blackwell Medics had cash and cash equivalents of $47.9 million as compared to $90.3 million as of December 31st, 2021. Expenses for the second half of 2022 decreased to $19.2 million as compared to $34 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 UPLS land, and to plan for UPLS-LAN's potential commercialization to continue the evolution of biomedics into a commercial-stage company. The 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 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 UPLS land 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 Haroud.
spk02: Thank you, Brian. This is a very exciting time for Glycomatics. We're encouraged that the recent interim analysis of our Phase III study found no safety concerns with buproliferan treatment, and the study was recommended to continue to 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 to 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?
spk06: Thank you. To ask a question, please press star 11 on your telephone and wait for your name to be announced. To withdraw your question, please press star 11 again. Please stand by while we compile the Q&A roster. Our first question comes from Boris Peeker with Cowan. Your line is open.
spk05: Great. Thanks for taking our question. 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'll be slightly delayed or pushed back similar to your Phase 3? And then the second question is, what's going on with the GMI1687? Are you planning to start a Phase 1 trial, or are you looking for collaboration partners with them? Thanks.
spk02: Thank you, Nick, for the question. 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 your Prolaceran 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're really focusing on your progesterone. 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 2 with 267 patients enrolled as of December 2021. And the idea was that there was a one-year pause until the EFS, that 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 update from NCI. And we are at end March. 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, you know, 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.
spk03: Great. Thank you very much.
spk06: Thank you. One moment for our next question. We have a question from Roger Song with Jefferies. Your line is open.
spk00: Great. Thanks for the update and taking our question. Maybe two questions on this. For maybe, Haru, you can add, you can give us some kind of how your team or the statistical team predicted the OS event timing and what have been changing or the driver for you to kind of keep pushing the data, understanding you probably seeing the blinded data in terms of MRG negative or the transplant rate dropout. So maybe just give us a little bit of color around the predictive model you have.
spk02: Sure, Roger. 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 transplanted patient versus not, it's 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 three, just to compare it with the phase one, two, 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, patients were enrolled back in November 2021, from then we projected that this will be beyond the 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 we have a very robust database now. And based on that database robustness, we also updated our projections. And now what we're saying, it's 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 your proletarian? Is it standard of care? Is it 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 your proletarian 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?
spk00: No, that's helpful. Thanks, Harul. Maybe just a follow-up question, understanding you are focusing on UPRO for this phase two, phase three R&R AML trial. Since you have some time, about a year, before you will have the top line data, what are the key background activities you and the team have been preparing for the data and the potential registration and commercial? Thank you.
spk02: Yeah, excellent point, Roger. I mean, one of the things which... you know, 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, you know, happen, we continue to leverage this time to also engage with the medical community. As you know, your proletarian 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, you know, Eupressoran and Eselectin antagonism as a mechanism is really in the limelight of the medical expert community. You know, 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. Perfect. Thank you. Hello. Thank you, Roger.
spk06: Thank you. There are no more questions in the queue. I'd like to turn the call back to Mr. Simardian for closing remarks.
spk02: Thank you, operator, and thank you for everyone for joining our call today. We look forward to keeping you up to date on glycometics. Have a great day.
spk06: This concludes today's call. You may now disconnect. Everyone, have a great day.
Disclaimer