CBIO - NASDAQ

Good morning, and thank you for joining the GlycoMimetics Q1 2023 Earnings Call. At this time, all participants are in listen-only mode. Following the management’s remarks, we will hold a question-and-answer session. At that time, the lines will be opened for you. [Operator instructions]. I would now like to turn the call over to Christian Dinneen-Long, Company Counsel at GlycoMimetics. Please go ahead.

Good morning. Today, we will review our business updates and financial results for the quarter ended March 31, 2023. The press release we issued this morning is available on the company's website at glycomimetics.com. This call is being recorded, and 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 our website. 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 operations, the conduct of our data from clinical trials, planned or potential development activities, regulatory interactions or submissions, pre-commercialization activities, 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 GlycoMimetics’ filings with the SEC, which are available from the SEC or through our website. I'll now turn the call over to Harout.

Thank you, Christian, and good morning, everyone. We have continued to make excellent progress advancing uproleselan clinical development, and setting the stage for our transition to a commercial stage company. At the heart of this progress is our ongoing pivotal Phase III trial of uproleselan in patients with relapsed and refractory AML, which continues to be projected to reach its final survival event trigger within the first half of 2024. Patients in this study continue to live longer than expected, leading to a median follow-up prior to primary analysis that is currently greater than 27 months. This slower than expected accumulation of survival events presents an ongoing ethical obligation to evaluate potential benefits from uproleselan in the observed results. Accordingly, in February 2023, we carried out an interim utility analysis based on 80% of planned survival events that incorporated a highly conservative analysis threshold. This conservative threshold preserved statistical power of the original analysis plan. An independent data monitoring committee conducted an interim analysis of the unblinded data and recommended we continue to the originally planned final analysis. Importantly, the DMC noted no safety concerns. Thanks to our team's excellent performance in preparing the clinical trial database and enabling the interim utility analysis, we're well positioned to complete trial analysis rapidly after the final survival event trigger projected to occur within the first half of 2024. We're optimistic and excited about uproleselan’s potential to improve overall survival in relapsed refractory AML, and are fully focused on delivering the potential of this important first-in-class therapy for patients in need of new, more effective treatment options. We remain keenly aware of our ongoing ethical obligation to patients to evaluate potential benefit of uproleselan as soon as possible. We will continue to monitor primary event accumulation closely to ensure that this potentially important treatment option reaches patients who need it. As part of our strategy to explore potential uproleselan benefits in patients across the AML spectrum, we're proud to work alongside the National Cancer Institute, and the Alliance for Clinical Trials in Oncology. The alliance will conduct a pre-planned interim analysis of event-free survival in its ongoing Phase II/III clinical trial, evaluating uproleselan in newly diagnosed older adults with AML who are fit for chemotherapy. We expect to provide updates on the outcome of interim analysis when available. With our experienced team and cash runway to fund operations late into fourth quarter of 2024, we're well positioned to execute on our strategy and continue advancing our uproleselan development program, including our Pivotal Phase III trial in relapsed refractory AML. On today's call, I'm happy to be joined by our CFO, Brian Hahn, and CMO, Dr. Ed Rock. Ed, I'll pass it over to you to give more details on our ongoing trials.

Thanks, Harout, and thanks to all of you on the line for joining our call today. As Harout mentioned, median follow-up for our Phase III trial of uproleselan in relapsed and refractory AML is now at 27 months. That means this study will potentially have the longest median follow-up duration of any trial in relapsed and refractory AML at time of primary analysis, with median follow-up of more than three years. Our trials population continues to live longer than expected based on historical benchmarks seen in other AML studies. Importantly, the independent data monitoring committee's recent interim utility analysis showed no safety concerns, and preserved statistical power to support final analysis of the primary survival endpoint. We continue to monitor primary event accumulation closely to ensure that this potentially important treatment option reaches patients who need it as soon as possible. We remain encouraged by uproleselan’s notably unremarkable safety profile, and are optimistic about its potential to change the treatment paradigm for patients suffering from relapsed and refractory AML. In addition to our Phase III trial, there's a second randomized trial of uproleselan that also has a pending readout, which may have been affected by slower than expected primary event accumulation, this latter trial in the large AML - the large frontline AML setting. The NCI Alliance Phase II/III trial, A041701, randomized 267 fit patients aged 60 years and older with newly diagnosed AML to7+3 chemotherapy, either with or without uproleselan. This study enrolled from the first quarter of 2019 through December 2021. We are now at 16 months after enrollment completion. For reference, the expected EFS medians for this trial were seven months for the control group, and 11 months for the uproleselan Group. AML incidents increases with age, and at time of diagnosis, over half of AML patients are older than 60, yet prognosis remains poor for these patients, as they are more likely to experience treatment-related toxicities and early mortality. New novel therapies remain needed to address the significant unmet need in this patient group. In the NCI Alliance trial, an improvement in median EFS from seven to 11 months would generate an EFS hazard ratio of 0.64. If the Phase II portion of this trial reads out with a hazard ratio of 0.64 or better, these data will be transferred to GlycoMimetics for regulatory purposes. Conversely, if the Phase II hazard ratio is between 0.64 and 0.831, the trial will proceed to Phase III enrollment of 670 total patients. At a hazard ratio above 0.831, the trial would stop for futility. NCI Alliance trial A041701 is being run under a confidential data safety monitoring board. When the pre-specified events trigger for Phase II is reached, Alliance statisticians will perform EFS analysis and inform NCI, then GlycoMimetics on next steps for the trial. This trial sponsor, the NCI division of Cancer treatment and Diagnosis, confirmed last week that it has not yet been informed by the alliance that the EFS trigger has been reached. We remain excited about the NCI Alliance trial, as it appears that the apparent long duration of follow-up tracks similarly to our own ongoing Phase III study. If successful, this trial has potential to position uproleselan as a foundational adjunct to both intensive and reduced intensity chemotherapy regimens. Our biologic hypothesis is that uproleselan will generate deeper, more durable disease responses that deliver more people to and through potentially curative stem cell transplantation. Now, I'll turn it over to Brian for a review of financial results.

Thank you, Ed. As of March 31, 2023, GlycoMimetics had cash and cash equivalents of $65 million as compared to $47.9 million as of December 31, 2022. During the first quarter, the company raised $28.7 million from sales of shares of common stock under its existing ATM facility. These additional proceeds are expected to extend our cash runway late into the fourth quarter of 2024. We intend to use our capital resources to advance the clinical development of and prepare regulatory filings for marketing approval of uproleselan, and to plan for uproleselan’s potential commercialization to continue the evolution of GlycoMimetics into a commercial stage company. The company's research and development expenses were $5.4 million for the quarter ended March 31, 2023, as compared to $9.6 million for the same period in 2022. The decreased expenses were primarily due to lower clinical trial and development costs related to our global Phase III clinical trial of uproleselan in individuals with relapsed refractory AML, which completed enrollment in November 2021. The company's general and administrative expenses increased to $5.5 million for the quarter ended March 31, 2023, as compared to $5.1 million for the same period in 2022, primarily due to commercial readiness planning expenses for uproleselan and additional patent fees. I'd now like to turn the call back to Harout.

Thank you, Brian. We're at a point in our company's evolution where our continued transformation relies on strong execution, and we're committed to advancing our pivotal Phase III trial of uproleselan. To our knowledge, this trial is on track to have the longest follow-up of any study in relapsed refractory AML at the time of primary analysis. We look forward to our trial's final readout projected within the first half of 2024. Also, we're excited about our partnership with the NCI and the Alliance and their Phase II/III trial of uproleselan in elderly patients with frontline AML, as we work to understand the drug's unique potential to improve lives of AML patients. We're grateful to the investigators and patients who make these large, randomized trials possible. We're well funded to complete these trials and continue our transformation to a commercial stage company capable of delivering impactful medicines to patients in need. I look forward to sharing updates with you in the future calls. I'd now like to open the lines for Q&A. Operator.

[Operator Instructions] The first question comes from Roger Song of Jefferies. Your line is now open.

Great. thanks for the update and taking our questions. So, first one maybe since you are confirming the current projection for the primary endpoint for the R&R AML Phase III data is first half 2024, just curious, have you take a new look of the data since the interim analysis in February, and what give you the confidence if you took a look at the interim, what give you confidence this will still happen in first half and maybe more broadly, what have been the key drivers to have you make the projection? Thank you.

Yes, thank you, Roger, for the question. It’s Harout here. So, I mean, yes, as we've communicated multiple times before, the way we've been handling these projections, I mean, overall survival is our primary endpoint. We continue to build a very robust analytical model that continues to project our events trigger, our final events trigger, and we've been communicating that since November 2021, when we finalized our enrollment, and we continue to do that, Roger. So, we haven't really disclosed like every time we do another projection, but that's ongoing basis. And as it stands now, as we look at that projections, we believe that H1 2024 is the appropriate timeline where we think that projections will land. Obviously, it all depends on as we go forward, as months pass as well, the number of events that happen in what subgroups of patients will happen. All these will dictate future projections and we'll keep you updated, but as of now, H1 2024 is where we are.

Great. Okay. Maybe another question is the - knowing the current cash runway funds through the end of 2024, just curious how much the pipeline development is baking the current cash runway, and if anything updates on your pipeline outside of the uproleselan. Thank you.

Yes, maybe I’ll take the first part and then Brian can give a bit more color about how we've extended the cash runway, in addition to the to the fuel up that we've done earlier this year. It's really important to kind of look at it and say, okay we have a platform. We are a platform company, glyco biology, we really believe is now at the cusp of starting to have medicines based on this platform. And uproleselan is really our most advanced asset that we have. As you know, 1687 is an asset that we have ready for clinics that has cleared IND. We have 1359 that has been already in clinic, and we've done a Phase I. But to be honest, we really have been putting all our efforts on uproleselan. We are at that 11th hour. We really want to make sure that we pull uproleselan through and then use some of the cash runway into funding our other therapies. So, at this point, we don't have anything new if we're starting any new clinical programs with the 1687 as an example, but always stay in tuned to that, Roger. That's always on the back of our mind that the faster we can do get there, the better it is, but at this time, it's really uproleselan. Maybe, Brian, on the side of giving some color on our cash runway extension.

Roger, yes, as we had disclosed previously, we were running about $15 million a quarter in FERN. That's reduced down to about $10 million a quarter. It's mostly from the reduced cost from the clinical trial expenses and a lot of the manufacturing expenses. So, as Harout was saying, a lot of the focus now is around uproleselan, with some small amounts to other research areas.

Excellent. Thank you. That's it from us.

One moment for our next question. And our next question comes from Boris Peaker of TD Cowen. Your line is open.

Great. Thanks for taking my question. This is Nick on for Boris. Just two quick ones for me. First, you mentioned that you'll complete the final analysis pretty rapidly after the OS event trigger. So, I was wondering if we should expect data in the first half of 2024 as well, or it'll take a little bit longer to like consolidate and parse through everything. And then the second question that I have is, are there any additional IST trials running testing grow? I know that there were two that had some positive data that actually was early. So, I was wondering if you have any idea on potential new data for these IST trials or anything else in that matter? Thank you.

Sure, thanks Nick. Maybe I’ll tackle the first question, then Ed, you can give an update on ISTs. So, from our part - sorry, Nick, can you just repeat your first question? I just want to make sure I understood correctly, if you don’t mind.

Yes, definitely. Yes. So, the event, the final OS event trigger is expected in 1H 2024, as you guys have mentioned. So, I was wondering if we should expect data then too, or if it'll take a little bit longer to parse through the data and everything, and then that'll push the data release back a little bit.

Got it. Okay. So, maybe I'll give you a couple of things, the way we are thinking about it. So, as we went into the interim analysis, Nick, we had to get ready in case we passed that interim analysis and that high threshold, we wanted to make sure that we are ready to really expedite and do the database lock and move into final analysis very, very quickly. So, that step has already been done thanks to the efforts of the team even over the holiday break as well, December, January, to really get us ready. So, we don't really have to do that again. That really shortens the time from the time when the DMC will take a look at it, until we have the full analysis. Now, it all depends on when within H1 we will have that an analysis, Nick. So, if we're having it sometime in the middle of the H1, so let's say end of Q1, I'm not saying that's what's going to happen. It’s just as an example, then within weeks after, we can get that data. If it's going to happen later in Q2, then there might be a spillover outside of H1. So, just some goalpost for you to think about is - the good news is, we have really accelerated the data cleanup, and as you know, a lot of the patients are now on long-term follow-up. So, that will make the job of our clinical teams to go back and get some of the queries and the data hopefully much faster now that we have the good base. So, that's kind of how you would want to think about it from a data disclosure perspective. And maybe on the ISTs, Ed, any updates that we have?

Nick, thank you for asking. As you know, we reported at ASH, or the investigators reported at ASH, on two ISTs, one in treatment of frontline AML unfit for intensive chemotherapy at the University of California and Davis by Brian Jonas. There were eight evaluable patients, of whom six had complete responses, and four of those six had MRD negative responses. We thought that was encouraging data. That trial continues to enroll. We are not disclosing numbers at this time, and like you, we’re keenly interested in progress of the trial as it moves forward. The other trial was at MD Anderson, led by Dr. Kadia there, and that's in patients with treated secondary AML. They are receiving uproleselan in combination with low-dose Cytarabine and cladribine. Similar numbers, similar responses, and similar MRD negative responses as the Jonas trial. Similarly, we understand enrollment continues, and we are not disclosing those numbers at this time.

Great, thank you very much. That's really helpful.

Thank you. One moment for our next question. And our next question comes from Ed White from H.C. Wainwright. Your line is open.

Hi, this is Steve on for Ed. Thanks for taking our questions. So, for GMI 1687, are you getting any partnering interest, and do you think you could find a partner for the program this year?

Yes, thanks, Steve. 1687 remains a very solid second program for us after uproleselan. As you know, this is a sickle cell market and potentially other indications, given its unique mechanism of action as well. So, at this point, we're not disclosing any specific partnership. That discussion, Steve, continues to be ongoing. For us, what's important as well is not only partners who bring funding to the table, but also capabilities and competencies. And the fact that we've been successful in refueling as well from a crash runway perspective, really gets us focused on uproleselan. So, stay tuned to that. At this point, we're not disclosing any specific partnership discussions.

Okay, great. Thanks. And one more if I may. So, how should we think about SG&A and R&D expense going forward as you prepare for the uproleselan long launch?

Sure. Maybe Brian, you want to tackle that?

Yes. For the guidance this year and into 2024, I would still anticipate about $10 million burn per quarter. And then after we get the trial results, we'll be giving guidance on what we think the expenses will be for our launch.

Okay. Thank you very much.

Thank you. As there are no more questions in queue, I'd like to turn the microphone back to Mr. Semerjian.

Thank you, operator, and thank you for everyone for joining our call today. We look forward to keeping you updated on GlycoMimetics, and seeing some of you at the Jefferies Health Conference, at ASCO and EHA. It's going to be a very busy and good time. Thank you so much.