GlycoMimetics Q2 2023 Earnings Call Transcript

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August 2, 2023

There are 7 speakers on the call.

Operator

Good morning, and thank you for joining the GlycoMimetics Q2 2023 Earnings Call. At this time, all participants are in listen only mode. Following 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.

Operator

Please go ahead.

Speaker 1

Good morning. Today, we will review our business updates and financial results for the quarter ended June 30, 2023. The press release we issued this morning is available on the company's website at glycomimetics.com. This call is being recorded 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 Herutz Immersion, Chief Executive Officer Brian Hahn, Chief Financial Officer and Doctor.

Speaker 1

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 about the conduct of and our collaborators' clinical trials, plans or potential regulatory .commission, development plans and activities, pre commercialization preparations, the company's operations, cash position and runway, and are expectations regarding data from clinical trials. Such statements represent management's judgment and intention as of today and involve assumptions, risks and uncertainties. Glactoimetics undertakes no obligation to update or revise any forward looking statement.

Speaker 1

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 the GlycoMimetics website. I'll now turn the call over to Haru.

Speaker 2

Thank you, Christian, and good morning, everyone. This is a transformational time for our company as we continue to advance our clinical pipeline and to evolve ourselves into a commercial stage organization. Today, we would like to highlight 3 major advancements that position us well for a catalyst rich upcoming 12 months. First, the FDA cleared their protocol amendment for our Phase 3 uproleselan study that will enable us to report top line results by the end of Q2 2024. 2nd, We continue to broaden our clinical development strategy for uproleselan by moving forward with our pediatric development plan.

Speaker 2

And third, we plan to further expand our clinical pipeline and initiate a first in human Phase 1a study For GMI 1687 in the Q3 of this year. These three areas of progress each Present potential long term value drivers for our company. In June, the FDA cleared the addition of a protocol amendment to our Phase 3 study of rupressiran for relapsed and refractory acute myeloid leukemia. This amendment adds a time based analysis option That will enable us to announce top line results by the end of Q2 2024. Final analysis will evaluate effects of uproleselan on relapsed and refractory AML in a clinically mature database with more than 3 years of median follow-up.

Speaker 2

The analysis will also incorporate at least 2 years of post transplant data for a large majority of patients remaining on study who received stem cell transplantation. Doctor. Edrock, our Chief Medical Officer, will provide additional information on the significance of this timing Later in this call. The option for time based analysis aligns with regulatory precedent for an approved AML therapy and reflect our commitment to deliver uproleselan to relapsed and refractory AML patients in need of new therapy options as soon as possible. With top line results expected by the end of Q2 2024, we continue to pursue pre commercialization activities, while also advancing additional pipeline programs.

Speaker 2

We're also proud to expand our uproplaciran development strategy by exploring its potential in people with all ages who are living with AML. This past quarter, We achieved 3 key advances in the development of uproleseran for pediatric patients. The FDA agreed to our proposed initial Pediatric Study Plan or IPSP establishing a regulatory path forward to study uprolosiran as a therapeutic option for pediatric AML patients. The NCI notified us that they will initiate a Phase onetwo dose escalation study to investigate safety and early activity of uproplacelan plus salvage therapy for relapsed and refractory pediatric AML. And finally, in June, the 1st pediatric patient was treated in an investigator initiated Phase III study of uplacelan Cross a pretransplant regimen for AML treatment.

Speaker 2

This important milestone is the first step in evaluating girdolaselan The study is being led by Doctor. John Horan of the Boston Children's Hospital and Dana Farber Cancer Institute. We're grateful that the NCI and Boston Children's Hospital teams are assessing uproleselan for treatment of pediatric AML patients, and we look forward to learning more about its potential impact in this vulnerable patient population. In the Q3, We plan to initiate a Phase 1a study of GMI-sixteen eighty seven in healthy volunteers to evaluate the drug safety, Tolerability and pharmacokinetics. GMI-sixteen eighty seven is a 2nd generation E selectin antagonist with potential users in diverse inflammatory diseases.

Speaker 2

Our initial focus will be on sickle cell disease. GMI-sixteen eighty seven has been shown in preclinical models to be highly subcutaneously bioavailable and this Phase 1a study It's a vital first step in its clinical development. Turning to our finances, we have a cash runway to fund operations late into the 4th Quarter of 2024. So we're well positioned to continue executing our clinical development plan. Our pivotal Phase 3 trial in the relapsed refractory AML remains on track for a top line readout at the end of Q2 2024 and we will begin a Phase 1a study of GMI 1680 On today's call, I'm happy to be joined by our CFO, Brian Hahn and CMO, Doctor.

Speaker 2

Ed Rock. Ed, I'll now pass it on to you to share more details on our ongoing trial.

Speaker 3

Thanks, Arut, And thanks to all of you on the line for joining our call today. Our recent protocol amendment to the uproleselan Phase 3 trial in relapsed and refractory AML will allow a time based primary analysis of overall survival. Time based analysis will occur after a defined cutoff date if the 295 survival events Originally planned for event driven analysis are not observed by that date. As Harut mentioned, Top line results are expected by the end of Q2 2024. Median follow-up for this trial will be over 3 years at the time of top line analysis in Q2 2024.

Speaker 3

That's unprecedented for a therapeutic trial in relapsed and refractory AML. We completed enrollment of the study's 388 patients in November 2021. Correspondingly, Primary survival analysis of uproleselan benefit in relapsed and refractory AML will be based on a clinically mature data set. That's because a substantial majority of surviving patients on study received stem cell transplantation And almost all of these transplant recipients will have at minimum 2 years of post transplant follow-up at the time of analysis. As you know, allogeneic stem cell transplantation is the only known curative therapy for acute myeloid leukemia.

Speaker 3

2 years post transplant is an important milestone in that after 2 years, the graft versus leukemia effect Stem cell transplantation has had time to develop fully. As a result, incidence of AML relapse After 2 years post transplant is low and these patients may be considered cured of their AML. Risk persists in this population primarily for non AML mortality, including from infections and graft versus host disease. Still after 2 years post transplant disease relapse becomes infrequent and disease relapse is of course what uproleselan is intended to prevent. Accordingly, the company believes capture of survival events After Q2 2024 would provide only limited additional value for primary analysis.

Speaker 3

As a result, GlycoMimetics believes that Q2 2024 provides a clinically optimal time to confirm uproleselan benefit in relapsed and refractory AML. The RATIFY trial of mitostorin in newly diagnosed FLT3 mutant AML patients Less than 60 years of age provides a regulatory precedent for our path forward. In RATIFY, as in our trial, Interim analysis led to a data monitoring committee recommendation to continue the study. In both trials, death events slowed appreciably after interim analysis. Once it became evident and ratified that the planned primary endpoint events trigger might not be reached, the sponsor added a time based final analysis.

Speaker 3

In our protocol amendment, FDA also cleared addition of landmark event free and overall survival analyses as secondary endpoints. These unpowered metrics will provide patients and healthcare providers with clear, clinically important comparisons of uproleselan versus placebo. FDA in Q2 also agreed to our initial pediatric study plan or IPSP for uproleselan. As part of this IPSP, the National Cancer Institute will conduct a Phase onetwo trial of uproleselan plus chemotherapy for pediatric patients with relapsed and refractory AML. This study is Nearly open for enrollment of up to an expected 18 patients and each patient will receive 15 This trial will assess uproleselan safety, Pharmacokinetics and preliminary efficacy in this population.

Speaker 3

Adult and pediatric AML are expected to have similar E select in biology with chemo resistance driven by AML Last binding to bone marrow endothelial cells. So we're excited to evaluate uproleselan with the National Cancer Institute in this study. Finally, Doctor. Jon Horan of Boston Children's Hospital is leading a new single arm multicenter Phase III trial of uproleselan combined with pre stem cell transplant conditioning for patients with chemotherapy resistant AML. This investigator sponsored trial will describe safety, tolerability and recommended Phase 2 dose of uproleselan plus Busulfan, clifarabine and fludarabine chemotherapy.

Speaker 3

The first patient treated in this study recently received uproleselan And is our first ever pediatric patient treated. Recent progress in pediatric development underscores our commitment to for uproleselan's potential to help AML patients of all ages. We're excited to expand uproleselan's potential utility to pediatric patients and look forward to sharing more information with you as these studies advance. Now regarding our glycomimetics study drug platform, we're proud to share that we will initiate a Phase 1a Single ascending dose trial of GMI-sixteen eighty seven later this quarter. GMI-sixteen eighty seven is a potent subcutaneously bioavailable E selectin antagonist.

Speaker 3

This 2nd generation glycomimetic compound has potential applications in multiple inflammatory diseases. Our initial development focus will be on interruption of sickle cell disease vaso occlusive crises Since E selectin appears to play a major role in pathology of these acute painful episodes. Proof of mechanism preclinical studies show attenuation of VOCs by GMI-sixteen eighty seven In 2 separate mouse models of sickle cell disease, studies in non human primates confirm its high subcutaneous bioavailability. Our first in human single dose trial will assess GMI-sixteen eighty seven safety, tolerability, pharmacokinetics and pharmacodynamics in healthy volunteers. PK and PD data from this Phase 1a trial will support design of treatment Regimens for multiple inflammatory disease indications.

Speaker 3

Subcutaneous GMI-sixteen eighty seven May enable patient self administration at home. If successful, that would reduce need for intravenous therapy in sickle cell vaso occlusive crises And provide a patient controlled point of care treatment option available at pain crisis onset. Thus, GMI may uniquely offer a differentiated approach with potential to interrupt vaso occlusive crisis Now I'll turn it over to Brian for a review of financial results.

Speaker 4

Thank you, Ed. As of June 30, 2023, GlycoMimetics had cash and cash equivalents of $58,000,000 as compared to $47,900,000 as of December 31, 2022. The company's research and development expenses were $4,100,000 The quarter ended June 30, 2023 as compared to $8,000,000 for the same period in 2022. The decreased expenses primarily due to lower clinical trial and development costs related to our global Phase 3 clinical trial of uproleselan in individuals with relapsedrefractory AML, Which completed enrollment in November 2021. The company's general and administrative expenses decreased to $4,900,000 for the quarter ended June 30, 2023, As compared to $5,500,000 for the same period in 2022.

Speaker 4

The reduction was primarily due to lower outside consulting and professional expenses, partially offset by commercial readiness planning expenses for uproleselan. Given that we now have definitive timing for our data readout by the end of Q2 2024, We will be able to utilize budget contingencies to advance the 1st in human study of GMI 1687, while maintaining our anticipated cash burn of roughly $10,000,000 per quarter with cash runway into late Q4 2024. I'd now like to turn the call back to Varun.

Speaker 2

Thank you, Brian. So in summary, the Glaucomaetics team continues to execute on our plan. Together, we achieved multiple milestones in the last quarter that can drive long term value for our organization, including reaching an agreement with FDA to add the option of a time based analysis to our Phase 3 study of uproplacelan, which will enable us to announce top line results by the end of Q2 2024. Initiating research of uproleselan in pediatric patients with the announcement of the trials led by the NCI and Dana Farber Cancer Institute. And last, broadening our pipeline to advance GMI-sixteen eighty seven to a first in human study.

Speaker 2

With a cash runway to fund operations late into Q4 of 2024, GlycoMimetics is well positioned for a catalyst rich next 12 months. I look forward to sharing more updates on future calls. I'd now like to open the lines to Q and A. Operator?

Operator

Thank you. At this time, we will conduct the question and answer session. Our first question comes from the line of Boris Peter of TD Cohen, Boris, your line is now open.

Speaker 5

Thanks. This is Nick on for Boris. Thanks for taking our questions. Just a cue for me. For the Phase twothree NCI trial for Ro, will they also be running a time based analysis Similar to what you guys are running for your Phase 3 trial or are they still keeping with their original plan for their analysis?

Speaker 5

And then also Just like a separate question, but for the pediatric patients, for upro, what's the plan following this Phase onetwo trial? Will the NCI continue to run trials? Or will you guys then take it after the Phase onetwo? Thanks.

Speaker 2

Thank you, Nick. Thanks for the question. Maybe I'll start it off and then Moving over to Ed. Regarding the NCI trial in the Phase twothree, what we know is they're still On consistent with what we have communicated last quarter is that they have not reached their events. So I think we're going to have to wait for that and see where they go next.

Speaker 2

They haven't we're not aware of anything beyond that. So what we know is that A predetermined number of events, the EFS has not been reached. So that's been also communicated to us recently. And maybe, Ed, you want to add more color on that one and then on the pediatric plan?

Speaker 3

Sure. Importantly, that trial's Phase 2 analysis, which is pending, will be based on events free survival rather than overall survival. Both of the randomized trials completed enrollment in Late 2021, so both have been have taken a good long time to mature over 18 months. We don't anticipate that the NCI will change their analytic plan to change from an event based EFS analysis to a time based EFS analysis. And then for the second patient second question about NCI's plans beyond the pediatric Phase III, those have not yet been decided definitively and we'll

Operator

Our next question comes from the line of Roger Song of Jefferies. Roger, your line is now open.

Speaker 6

Great. Thanks for the update and taking our question. A couple from us. The first one is Regarding the uproleselone, the R and R AML period of study, Had the team taken another look at the data and how did that tracking your modeling? I understand now it's time based, but have you ever did additional kind of data look?

Speaker 6

I think on the call you mentioned most of the saliva, They are post transplant. So any additional color you can provide us to us? Thank you.

Speaker 2

Yes. Thank you, Roger. Yes, I mean, the trial continues patients continue to live longer in this trial. So obviously, as you know, we are blinded. We do take a look at the pooled blinded data And we're excited that we have now this option of the time based analysis should the 295 events not be reached By next year, we believe we're going to have a mature database given the 3 years median follow-up, given the 2 years plus Of post transplant follow-up as well for the vast majority of patients that we're going to have a mature database.

Speaker 2

So Things continue to be on track. Patients continue to live longer. Obviously, we don't know who's on what arm, but we are very encouraged By this updates and the fact that now we have the option of the time based analysis on top Gives us certainty, clarity and of course on a database that's going to be mature.

Speaker 6

Excellent. Thank you. And regarding the 1687, so what Do you expect to see the healthy volunteer data? And how would that support the next step? And do you have any Timing and then maybe the plan, strategic plan in terms of you will take this on your own or you will seek your partner to move forward?

Speaker 6

Thank you.

Speaker 2

Yes. Thanks. Excellent question, Roger. I mean, as you know, 1687 for folks on the phone, this is clear this other asset We have cleared IND back in June of last year and given where the markets were, the financial constraints we had, we really to double down on our Phase 3. We're very excited now to be able to be in a situation where we're able to start The Phase 1a of 1687, a compound we believe in, we have deep expertise as you know in this area in sickle cell And bringing out expertise on our 2nd generation E selectin antagonist that is potent and subcutaneously bioavailable.

Speaker 2

So the first step of that, Roger, as you know, is that single ascending dose, as Ed mentioned. And really, it's Healthy volunteer study, so I don't want to read more into it than what it is. It's really to give us the PKPD Safety signals, which are really needed, as a first step. And quite honestly, regardless of what indications we go, That single ascending dose trial in the Phase 1a is very consistent and is the same. So we're going to be doing this.

Speaker 2

Typically, these volunteer trials, they don't take that much time, but it's usually in the month. So sometime, I would say next year, we should have that information. It's just too early to tell now. Let's get started. As we're announcing today, we are planning to start.

Speaker 2

We haven't started yet. So let's get started, and we'll keep the market But I'm very excited about the ability to start 1687 and put yet another glycoimetic product In a first in human study in the marketplace.

Speaker 6

Got it. Yes. Maybe just after the Seeing those, what will be the next step and strategic plan in terms of your own versus a partner? The follow-up of that?

Speaker 2

Yes. I mean, it's too early to say, Roger. I mean, regardless, if we're going to be doing it ourselves or we're going to be partnering, We need this data anyway. And the fact that we're able to fund it, we're able to move this forward, it's a great, great step. As we've said Multiple times, we're always open to partnership conversations, but we don't really have to.

Speaker 2

In a way, the partner has to bring beyond just The cash has to bring in that expertise, dedication to sickle cell. This is an area where we've seen Prophylactic measures, let's say, have humbling uptakes over the last few years. And on the other side of the spectrum, Gene therapies, which are very exciting, I wonder how many patients would actually benefit from that. So we do believe that The vast majority of this patient population unfortunately will continue to have vasculoskeletal crisis and providing an option that is on demand At the start of that viva vaso occlusive trisis is going to be a tremendous help for this patient population. So while we hope that there is multiple different approaches, We do believe that this approach will have a market for it.

Speaker 2

But of course, before getting ahead of ourselves, first, let's start the Single ascending dose trials, let's get that going. Let's continue the conversations as well with people who are very Motivated in doing trials in sickle cell disease and that medical community is very active, that patient community is very active. So we look forward to partnering with them and potentially other institutions if that works for us and for them. But meanwhile, we have Thank you, Roger. Operator?

Speaker 2

We might have dropped our operator.

Operator

I am back. Thank you all. I apologize there is a slight disconnection. I believe it is time to wrap up. So now We can turn it back over to Haru.

Operator

We are done with questions for now. Haru, it is one for you.

Speaker 2

Thank you very much. And thank you to everyone for joining our call today. We look forward to keeping you updated on GlycoMimetics And seeing some of you at the H. C. Wainwright Healthcare Conference in September.

Operator

Thank you for your participation in today's conference. This does conclude the program. You may now disconnect.

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Key Takeaways

  • FDA-cleared protocol amendment for the Phase 3 uproleselan trial adds a time-based overall survival analysis, enabling top-line results by end of Q2 2024 on a database with over 3 years median follow-up.
  • Expanded pediatric development for uproleselan with an FDA-agreed initial Pediatric Study Plan, an NCI-led Phase 1/2 trial, and the first pediatric patient treated in an investigator-initiated Phase 3 pre-transplant study.
  • Planned initiation in Q3 2023 of a Phase 1a single-ascending-dose study of GMI-1687, a subcutaneously bioavailable E-selectin antagonist, to assess safety, tolerability and PK/PD in healthy volunteers ahead of sickle cell disease trials.
  • Strong financial position with $58 million cash at June 30, 2023, a quarterly cash burn of ~$10 million and runway into late Q4 2024 to support ongoing clinical and pre-commercial activities.
A.I. generated. May contain errors.
Earnings Conference Call
GlycoMimetics Q2 2023
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