CBIO - NASDAQ

Good morning and thank you for joining the GlycoMimetics Call. At this time, all participants are in listen-only mode. Following management’s remarks, we will hold a question-and-answer session. [Operator Instructions] I would now like to turn the call over to Shari Annes of Investor Relations Group at GlycoMimetics. Please go ahead.

Good morning. Today, we will review our accomplishments and financial results for the period ended March 31, 2021. We will also update you on recent achievements. The press release we issued this morning is available on the company’s website at www.glycomimetics.com under the Investors tab. 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 on the Investor Relations section of the company’s website for 30 days. Joining me on the call today from GlycoMimetics are Rachel King, Chief Executive Officer and Brian Hahn, Chief Financial Officer. We will start today’s call with comments from Rachel. Brian will follow Rachel to provide an overview of the company’s financial position. And we will then open the call for Q&A. Our Chief Scientific Officer, Dr. John Magnani and our Chief Medical Officer, Dr. Eric Feldman will join us in the Q&A to address your questions. I would like to remind you that today’s call will include forward-looking statements based on current expectations. Forward-looking statements contained on this call include, but are not limited to, statements about the company’s product candidates, uproleselan, GMI-1359, and GMI-1687 and our other pipeline programs, along with our operations and 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, are on the GlycoMimetics website. I will now turn the call over to Rachel.

Thank you, Shari. This year is an important one for GlycoMimetics. Our primary operational focus is on advancing the two uproleselan registration programs in AML. Leading clinicians, academic centers, collaborative networks and regulatory agencies are working with us here in the U.S. and abroad as uproleselan continues to garner significant attention and interest. We believe the progress being made in both registration trials stands as testimony to the excitement around this program as do the breakthrough therapy designations granted by both the FDA and the Chinese Health Authority. GlycoMimetics’ pivotal Phase 3 trial in relapsed/refractory AML continued to enroll patients in the U.S., Australia and in Europe at a steady pace through the first quarter of this year. Our remaining number of sites continued to be activated in Europe and in the U.S. as we make a final push toward reaching our target enrollment numbers. Once again, we are confirming that we anticipate completion of enrollment of all 380 patients by the end of this year. In parallel, the NCI-sponsored Phase 2/3 registration trial that’s evaluating uproleselan in newly diagnosed older adults with AML also continues to accrue participants at a steady pace. Based on the information provided by the NCI to-date, we continue to anticipate that the trial will complete enrollment of the Phase 2 portion by year end, supporting a subsequent interim analysis of event-free survival, or EFS, in the trial’s initial 262 patients. Together, the GlycoMimetics and NCI-sponsored programs will constitute a large dataset of patients treated with uproleselan and intensive chemotherapy. If both are positive, we would anticipate filing for approval for treatment of patients in both settings. Our collaboration with Apollomics in China underscores the broad global community interest in uproleselan. And in January of this year, uproleselan was designated as a breakthrough therapy in China for the treatment of relapsed/refractory AML, complementing a prior designation by the FDA. In addition, Apollomics also announced in March that it had dosed the first patient in Greater China in the Phase 1 clinical trial for treatment of adults with relapsed/refractory AML. This is a bridging study that’s expected to allow Apollomics to expeditiously advance development to a Phase 3 trial in Greater China. Beyond working with the NCI and Apollomics, we are continuing to receive significant interest from clinicians at key centers of excellence who would like to pair uproleselan with a variety of chemotherapy regimens and other AML therapies. As the year progresses, we expect to announce initiation of multiple investigator-sponsored trials or ISTs, designed to extend the use of uproleselan across the AML spectrum and potentially beyond AML as well. For example, the preclinical research that combines uproleselan, venetoclax and the hypomethylating agent, or HMA, sheds light on the novel benefits uproleselan could provide in combination with these important therapies. Similar to venetoclax, we may have one of the few drugs that in chemotherapy combos actually improve survival outcomes. Importantly and uniquely, uproleselan may improve outcomes without incremental toxicity. As you know, uptake on venetoclax HMA in the frontline unfit AML setting has been strong and while 60% to 70% of patients achieve a response, the responses are incomplete in approximately half the patients, reducing durability and leading to relapse. Thus there remains a significant unmet need. This broad clinical program of company sponsored, NCI-sponsored and investigator-sponsored trials, supports our single-unified vision, namely to establish upro as a foundational therapy across the entire spectrum of AML, whereas most other AML therapies and development are focusing on narrow patient populations as defined by certain genetic markers, the uproleselan targets the bone marrow microenvironment by inhibiting pathways that protects cancer cells from the effects of chemotherapy. This is a novel approach that we expect could be broadly applicable across the spectrum and range of therapies used for relapsed/refractory or newly diagnosed patients. As you know, uproleselan is an E-selectin antagonist. The scientific rationale for targeting E-selectin and the role it plays within the bone marrow microenvironment as a driver for AML resistance is robust. In addition, our preclinical data supports uproleselan’s protective effects against certain toxicities of chemo such as mucositis. You will recall that in our year end call in March, we described the data and research that had been presented at multiple scientific and medical meetings last year. If you haven’t already done so, I urge you to visit the scientific publications section of our website to review this exciting work. As we continue to make progress in our pipeline, our plan is to submit abstracts on key research findings to medical meetings in the U.S. and abroad. This past month, at the meeting of the American Association for Cancer Research, or AACR, we were invited to present a poster on our ongoing Phase 1b study of GMI-1359, a dual antagonist of E-selectin and CXCR4. This Phase 1b clinical trial was designed as a proof-of-concept study to evaluate pharmacodynamic, or PD markers, such as CD34 mobilization, mobilization of circulating tumor cells into the periphery, down-regulation of soluble E-selectin and other biomarkers of biological activity, following both single ascending and multiple doses within the same patient. Our goal was to use these PD markers to confirm our on target biologic activity. We believe we have accomplished this objective as the clinical and preclinical data presented at AACR demonstrate that GMI-1359 is clearly hitting both targets. This provides us confidence the drug candidate could be broadly active in disrupting the tumor microenvironment with added evidence of immune activation. Based on our findings, we are evaluating indications for moving the program forward in the clinic. In the sickle cell setting, based on input from the FDA with respect to rivipansel as well as feedback from KOLs, we are focusing development on GMI-1687. We have initiated IND-enabling activities with treatment of acute VOC as the potential lead indication. We and others believe that GMI-1687 may be ideally suited for this indication as it would enable patients to potentially self-administer treatment early in their VOC crisis. As we have shown clearly from the Phase 3 RESET and open-label extension clinical analysis, early intervention is particularly important to improve clinical outcomes in this acute setting. The intended profile of GMI-1687 as a satisfacting on-demand and self-administered drug candidate also dovetails nicely with the continued shift in patient care to the outpatient setting, a trend which has accelerated during the pandemic. We will keep you posted as to progress with the 1687 program as we get closer to first-in-human dosing, which we anticipate will occur in 2022. Our research efforts continue to progress, particularly in the galectin field. Also at AACR, our research team presented new evidence on the effects of one of our Galectin-3 antagonist in a cancer model. This adds to a robust and broad foundation of preclinical data that demonstrates the high potency and selectivity of our galectin-3 antagonists. The potency and selectivity of our compounds distinguished us from competitive approaches, and we intend to roll out additional data as we move toward our goal of selecting a lead candidate for clinical development. Also during the first quarter, we promoted Dr. Eric Feldman, to Senior Vice President and Chief Medical Officer. Eric is internationally recognized for his work in the development of new therapies for the treatment of leukemias and related bone marrow disorders. As CMO, he plays a critical role in overseeing the broad clinical pipeline for uproleselan as well as for programs in our advancing pipeline. Importantly, our current cash position provides a run rate to key milestones and Brian will now comment on our financial results.

Thank you, Rachel. As of March 31, 2021, GlycoMimetics had cash and cash equivalents of $132.5 million as compared to $137 million as of December 31, 2020. The company’s research and development expenses decreased to $11.2 million for the quarter ended March 31, 2021, as compared to $12.7 million for the same period in 2020, primarily due to lower clinical asset development and manufacturing expenses related to uproleselan. The company’s general and administrative expenses decreased to $4.2 million for the quarter ended March 31, 2021, as compared to $4.4 million for the same period in 2020, primarily due to lower stock-based compensation expense. I would now like to turn the call back to Rachel.

Thank you, Brian. Before I open the lines to Q&A, I would like to leave you with 2 takeaways. First, the momentum and excitement around uproleselan continues to build as we drive to complete enrollment of 2 pivotal trials. These are important milestones ahead. Second, in the interim, we look forward to sharing both news of newly initiating trials with independent investigators as well as progress in our expanding pipeline, underscoring the value of our unique GlycoMimetics platform. Operator, would you please open the lines for the Q&A session?

[Operator Instructions] Your first question comes from the line of Stephen Willey with Stifel.

Yes. Good morning. Thanks for taking my questions.

Good morning.

Good morning. Rachel, I was just wondering if you can update us, I guess, on the number of sites that you intend to open across the U.S. and Europe for the relapsed/refractory study before year-end to kind of help you get to that goal. And can you just remind us how many sites are activated and open to-date?

Yes. So you – so the sites are generally listed on clinicaltrials.gov. And I think we have right now around 60 sites open.

Okay. And then just the number that you are hoping to activate before the end of this year?

So, we are near the complete number of sites, actually. We have got a very robust network currently in place. We are continuing to activate a small number of sites, primarily in Europe and a couple in the U.S. But we are essentially at the full complement of sites today, as of today.

Okay. And just with respect to 1359. I know we saw the data at AACR. And it sounds like you are evaluating indications in which to maybe move this asset forward. Can you maybe just talk a little bit about what those indications may be? Obviously, we saw this in the context of breast cancer, I would imagine that might be a priority. But can you just provide a little bit more color around what development options are at this point? And if you do move forward in breast cancer, is that also a hormone receptor positive patient population, would you expect to have this against background standard of care? Just any color there would be helpful.

Sure. So, we are not yet in a position to describe the specific next plans. What will be important, though, is that whatever we choose is going to be a targeted and focused indication with – again, targeted and focused clinical trial. The preclinical data that we have presented has been actually fairly broad in the sense of looking at settings where there is bone involvement in the solid tumor case or in some cases, in liquid tumors, bone marrow involvement. We also have some very encouraging preclinical data in osteosarcoma. So, that’s generally the scope of indications that we are looking at. But again, we are – we think it’s going to be critical both from a strategic and an operational standpoint to find a setting where we can move forward in a targeted and focused way, and that’s going to be the same as we look through the potential indications that we could take 1359 into.

Okay. Thanks for taking my questions.

Sure.

Your next question comes from the line of

Good morning guys and congrats on all the progress, particularly on the enrollment for upro. Just had a question about that program as well as it relates to Apollomics. I was just wondering when you think they might be starting that study after the bridging study?

We expect that to start later this year.

Perfect. Okay. And then just again, just curious if there is any way for them to leverage some of the Phase 3 data that you are going to be generating as well for approval?

Yes. So I can’t speak to their specific path forward for approval in China. I’ll leave that to Apollomics. But we do believe that the programs are complementary, and that we will be able to – that they would be able to leverage some of they would be able to leverage some the data in our study.

Thanks, Rachel. And then again, just a follow-up, I know you mentioned potential investigator sponsor studies for upro. I was just wondering how you plan to leverage those studies for later clinical development and then what indications maybe prioritizing?

Sure. So I want to comment on the ISTs generally. And as we announce them as the year goes forward, we can address each one specifically. We’ve been really gratified in the uproleselan program with the support and the interest that we’ve received from many, many KOLs in the U.S. and outside the U.S. And I think you see that, of course, first, in the NCI trial, where there was a lot of enthusiasm for taking the program forward into the alliance network. But we’ve continued to see a number of investigators who have come to the company with very interesting proposals for expansion of use of uproleselan. So we’re very gratified by that. And we’re working with a small number of investigators to try to go forward in the most – what we believe are the most promising areas. So as those roll out, then we will announce them. But I think the general concept is to build on this investigator enthusiasm to broaden the use of the drug in these clinical trials, which potentially could give us opportunities as the program advances to continue to broaden the use ultimately in the commercial setting. So I would say stay tuned as these trials initiate one by one, we will announce them. And then we can speak specifically to each of those approaches as they come forward.

Thanks, Rachel. And then the last one here for me, just on 1757, I thought it was exciting, but a little bit different because you guys mentioned some solid tumor indications, which is a little different from what you’re currently doing. So just wondering how you’re thinking about that opportunity?

Yes. So the galectins represented an entirely new opportunity for the company, and they are involved. We know from the basic preclinical science. We know that they are involved in fibrosis as well as in cancer. And we think we have a unique approach from a chemistry perspective to creation of a new set of compounds, which, as I indicated, are, we believe, more potent and more specific than other compounds that have been described by anyone else. So we’re continuing right now to explore the potential use of these compounds in a broad range of preclinical studies, and we’re also moving toward eventual selection of a clinical asset. So that program is early, but it’s quite exciting in terms of the potential applications. And so I think it’s too early to describe any specific indication for that. But we’re very encouraged by the biological activity, by the binding affinities, and as I said, the specificity that we’re seeing in these compounds. We think they are quite unique in the field.

Thank you. And actually just one last quick one here. So you do have a solid cash position. I think you guys have put it $132 million, but I was just curious with multiple programs now in preclinical and clinical development, I was just wondering what the cash run-rate might be?

Yes. So it’s very important – well, go ahead – let me make one comment, and then I’ll turn it to Brian. I think it’s really important to recognize that we’re – our operations – our operational focus remains on uproleselan, and that’s the – that’s really where most of the spend is going and with the critical objective of getting the enrollment completed in these trials. But go ahead, Brian, as to runway.

Let’s get on to that. Cash – current cash run rate gets us through the end of ‘22 right now.

Thanks, Brian. Again congrats on all the progress.

Thank you. Thanks

Your next question comes from the line of Ed White with H.C. Wainwright.

Hi. This is Steve Bersey on for Edward. Sorry. Did you say to the end of ‘23 for the cash runway?

No, through the end of ‘22 right now. So we’ve got about $75 million of total expenses in ‘21, about $50 million in ‘22, so comfortably getting through the end of ‘22 for current cash.

Alright. Thank you.

[Operator Instructions] Your next question comes from the line of Kelly Shi with Jefferies.

Hi, good morning. This is Jason Bouvier on for Kelly Shi and thanks for taking my questions. I just had two questions. One was from the development, a regulatory perspective, are there any material differences for breakthrough therapy designation between the FDA and the Chinese health agency? And then the other question was just if you had any specifics on the timing of a data readout following the interim analysis for the Phase 2 NCI study? Thanks.

Sure. As far as the BTDs in China is concerned, I can’t speak to the specifics of the Chinese designation, but I believe it generally is consistent with what the FDA grants in terms of an opportunity to work more closely with the agency to try to expedite development of the compound there. And as far as data readout at the NCI, this actually is a question that applies both to day to read out at an tinted read out for our own trial, which is that two things have to happen. The trials have to complete enrollment and then a certain number of events has to occur to enable the locking of the database. And so I think as both trials progress more towards completion of enrollment, we will be able to give more specific guidance as to when we might expect the number of events to occur. But as of today, the NCI has not given specific guidance publicly as to when they expect the number of events to occur. But I think as they – again, as they complete enrollment that they may be able to provide some more guidance on that.

Alright. Great. Thank you.

Sure.

As there are no more questions in the queue, I’d like to turn the call back over to Ms. King.

Well, thank you. Thank you very much for your questions and for your attention. And again, I want to emphasize our excitement around the uproleselan program as well as the expectation that we be able to announce ongoing progress with newly initiating trials as the year goes forward as well as some progress with our underlying pipeline. So thanks very much for your support of the company and your interest. We appreciate it.