Q3 2023 GlycoMimetics Inc Earnings Call
Participants
Christian Dinneen-Long; General Counsel; GlycoMimetics, Inc.
Harout Semerijian; CEO; GlycoMimetics, Inc.
Edwin Rock; CFO; GlycoMimetics, Inc.
Brian Hahn; Chief Medical Officer; GlycoMimetics, Inc.
Roger Song; Analyst; Jeffries
Boris Peaker; Analyst; TD Cowen
Presentation
Operator
Good morning and thank you for joining the GlycoMimetics Q3 2023 earnings call. At this time, all participants are in a 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 Christian Dinneen-Long company counsel at GlycoMimetics. Please go ahead.
Christian Dinneen-Long
Good morning. Today, we will review our business updates and financial results for the quarter ended September 30 2023. The press release this morning is available on the company's website at glycomimetics.com. This call is being recorded. The dial-in phone replay will be available for 24 hours after the closing of the call. The webcast replay will also be available for 30-days in the Investor Section of the company's 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 candidates uproleselan, and GMI -1687, along with statements about the progress and timing of clinical trials being conducted by us or our collaborators planned approach, central regulatory agency interactions are submissions.
Development plans and activities, prelaunch preparations, the company's cash position and runway and our expectations regarding data readouts from clinical trials. 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 statements. 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 Harout.
Harout Semerijian
Thank you Christian, and good Morning everyone. In the third quarter, we continued to make strong progress advancing our clinical pipeline as we evolve into a commercial stage organization. We remain committed to executing on our vision to deliver about uproleselan to AML patients in need of new treatment options.
While we leverage our unique molecular biology approach to develop innovative medicines in additional diseases such as sickle cell.
Today, I would like to highlight three drivers that position us well to advance these programs and then coming here. First, we continue to expect top line results from our pivotal Phase 3 study of uproleselan relapsed and refractory AML by the the end of Q2 2024 and remain encouraged about the unprecedented median follow-up time.Should trial results be positive we expect US filing by end of 2024.
Second, as part of our ongoing commitment to evaluate the utility of uproleselan across AML, researchers from MD Anderson will present updated clinical data from their investigator initiated trial in treated secondary AML at the American Society of Hematology meeting this December.+
Lastly, for GMI-1687 and our sickle cell disease program. The Phase 1a study remains on track for safety data by the end of Q1 2024. With addition of the time-based analysis option to our pivotal Phase 3 study of uproleselan in the relapsed refractory AML. We expect to report top-line results by the end of Q2 2024.
We look forward to unveiling these data as this important milestone for patients, investigators and the company.
While recently FDA approved drugs have shown encouraging results in limited AML subpopulations. We're optimistic that your uproleselan has the potential to improve patient outcomes irrespective of mutation profile, cytogenetic risk or treatment backlog.
Building on the promise uproleselan in relapsed refractory AML , we are excited to explore its broader potential across different age groups and disease settings through studies run by partners and independent investigators.
For example, at the upcoming ASH Annual Meeting in San Diego, research from the MD Anderson Cancer Center will present updated clinical data from their Phase 1b 2 study, evaluating uproleselan with Cladribine and low-dose cytarabine in treated secondary AML patients.
We are grateful to be collaborating with leading organizations, including MD Anderson, the National Cancer Institute and the Dana-Farber Cancer Institute.
The commitment of our partners underscores uproleselan potential to improve and prolonged lives for a broad spectrum of AML patients.
We look forward to providing updates as these investigator-initiated clinical trials continued. Finally, we expect to announce safety data from our Phase 1a study of GMI -1687 by end of Q1 2024. We expect these data will advance us towards our aspiration to develop GMI- 1687 has a patient controlled point of care therapy to interrupt early sickle cell pain crisis before need to seek emergency care. We will evaluate next steps shortly thereafter.
As our clinical pipeline has progressed, we have strategically expanded our commercial and medical affairs capabilities, and we are now executing critical prelaunch activities, head of top line uproleselan readout expected in Q2 2024. Our educational disease awareness activities, target, academic and community hematologists that care for AML patients.
Recently, we welcomed Dr. Gaetano Bonifacio as our Vice President of Global Medical Affairs and Debora Peralta as our Vice President of Commercial Operations. Gaetano and Debora bring decades of hematology launch experience and we are pleased to have the opportunity to leverage their expertise during this transformative point in our company's evolution.
Now turning to our finances. We have a cash runway to fund operations into late Q4 2024, allowing us to continue executing our clinical development plan. On today's call, I'm happy to be joined by our CFO, Brian Hahn, and our CMO, Dr. Edwin Rock.
I'll now pass it over to Ed to share more details on our ongoing trials.
Edwin Rock
Thanks a Harout, and thanks to all of you on the line for joining our call today. As Harout mentioned, we expect to report top-line results of our Phase 3 trial by the end of Q2 2024. At that time, we'll have a clinically mature data set for time-based primary analysis of overall survival.
If the 295 survival events that trigger primary the analysis are not already reached by that time.
Median follow-up for our Phase 3 trial a few progressed well in relapsed and refractory AML stands now at 33 months and will be greater than three years at time of primary analysis that's unprecedented for therapeutic trial in relapsed and refractory AML.
Also, the substantial majority of surviving study patients received hematopoietic cell transplantation ended primary analysis a large majority of them will be at least two years out from their transplant. After two years post transplant disease relapse becomes infrequent. So it's at least two years of post transplant follow-up for almost all transplant recipients.
We're confident that our time-based primary analysis will provide adequate trial duration to demonstrate uproleselan benefit if present. Our biologic hypothesis is that edge point of view progressively and will lead to deeper more durable AML disease responses that help more people to and through potentially curative hematopoietic cell transplantation.
This effect may occur irrespective of specific gene mutations, cytogenetic risk or treatment backbone. Correspondingly, uproleselan and clinical activity is seen in both newly diagnosed and relapsed and refractory AML.
Importantly, uproleselan appears to generate noteably unremarkable safety profile, adding no additional toxicity when combined with other therapies.
So we are optimistic that uproleselan may one day be safely, combined with diverse other therapies that are broadly useful for most oral AML patients.
Partner and independent investigator studies are further exploring potential benefit of uproleselan across a AML subtypes. Most importantly, a randomized NCI Phase 23 trial conducted by the Alliance for Clinical Trials in Oncology is evaluating you for less land in newly diagnosed older patients with AML who are fit for intensive chemotherapy.
This trial completed Phase 2 randomization of 267 patients in December 2021. Phase 2 analysis of event-free survival has been pending since then, and now almost to years since enrollment completion.
And to see, I recently confirmed to us is that the event trigger for analysis hasn't yet been reached.
For reference. The Phase 2 portion of this trial was designed to demonstrate prolongation of median event-free survival from 7 to 11 months. We look forward to learning Phase 2 results when available. Significantly, NCI expanded our collaboration and now also supports a children's oncology group Phase 1 study conducted by their pediatric early phase clinical trial network.
This dose escalation trial will assess safety, pharmacokinetics and preliminary clinical activity uproleselan plus chemotherapy in pediatric patients with relapsed or refractory AML.
We're glad to announce that the first patient enrolled this study in October, another investigator initiated trial, led by Dr. John Horan from the Dana-Farber Cancer Institute and Boston Children's Hospital also dosed its first patient earlier this year.
Dr. Horan's trial is evaluating new progressively and with the pre-transplant regimen for pediatric and adult AML patients up to 39 years old.
Today, we announced that at ASH in December, researchers from MD Anderson Cancer Center will present updated trial data on uproleselan in treated secondary AML.
This rare very high risk study population is defined by prior chemotherapy and an [anti- treatment] of and antecedent hematologic disorder. Phorgnosis is abysmal with expected median survival of less than five months. In the trial at MD Anderson investigators sought to generate a safe approach to marrow blast reduction, disease control and potential for transplant.
By combining uproleselan with low intensity chemotherapy of cladribine and cytarabine. Bad prognostic features in the study population include adverse cytogenetic risk and prior hypomethylating agent used in all valuable patients and prior hematopoietic cell transformation in 25% of them. Among 18 evaluable patients of data cut off, there were minimal therapy related toxicities 72% showed a reduction in bone marrow blasts and one patient had a potentially curative transplants.
These results in is notoriously difficult to treat disease underscore broad potential utility uproleselan across the AML spectrum. In addition, researchers from Washington University will present at ASH safety and signal generating data from their trial of uproleselan to reduce GI toxicities of melphalan chemotherapy given before transplant for multiple myeloma.
Beyond uproleselan we also made progress in our Phase 1a single ascending dose trial of GMI -1687 for second generation E-selectin antagonist. We will evaluate GMI -1687 as a potential outpatient self-administered subcutaneous therapy to interrupt sickle cell basil occlusive crises.
E-selectin plays a key mechanistic role in early progression of such acutely painful pathologic events. And if successful, GMI -1687 offers potential for a point of care patient controlled treatment option at time of pain onset.
In addition to patient benefit from pain control, such a point of care therapy has potential to reduce emergency room visits and hospitalizations of recipients.
As mentioned, we expect to have safety data in hand from this healthy volunteer study by the end of Q1 2024.
Now I'll turn it over to Brian for a review of financial results.
Brian Hahn
Thank you Ed. As of September 30, 2023, GlycoMemitics cash- and- cash equivalents of $49.4 million as compared to $47.9 million as of December 31, 2022. This increase was due to the company's ability to raise additional cash earlier this year.
The company's research and development expenses were $5.3 million for the quarter ended September 30, 2023, as compared to $4.9 million for the same period in 2022.
Decreased expenses were primarily due to the clinical development costs related to the Phase 1a trial of GMI -1687 in healthy adult volunteers, which was initiated in August 2023, partilally offset by decrease in stock based compensation costs due to lower headcount.
The company's general administrative expenses increased to $4.5 million for the quarter ended September 30, 2023, as compared to $3.8 million for the same period in 2022. Increased expenses were primarily due to higher personnel related expenses and higher professional fees as a company advances uproleselan and preparing for potential regulatory filing and commercialization.
I'd now like to turn the call back to Harout.
Harout Semerijian
Thank you, Brian. In summary, we are at a crucial stage in our company's evolution. In the coming months. We will remain laser focused on delivering the data, our Phase 3 study of uproleselan in the relapsed and refractory AML and continuing strategic prelaunch activities to accelerate our transition to a commercial side stage company after top-line results by end of Q2 2024.
We have the right team with the right experience in place, and I'm confident that we will be able to build upon our collective track record of successful commercial launches should data permit. Also, we continue to explore the uproleselan across the ammo spectrum.
Thanks to studies conducted by institutions such as NCI. Dana Farber Cancer Institute and MD Anderson Cancer Center. Finally, we continued to progress our Phase 1 study of GMI -1687 towards initial data readout in Q1 2024.
In closing, I want to thank our employees whose hard work and dedication drive uproleselan forward as well as the patients and investigators that make these trial if possible.
I'd now like to open the lines to Q&A.
Question and Answer Session
Operator
(Operator Instructions)
Roger Song, Jefferies.
Roger Song
Great. Thank you, thanks for that for that update. A couple of questions on that. One thing is, can you share with us at how likely by the end of Q2 2024 you on the primary analysis to well-being event time-based just and awards how they get then accumulation since last update. Thank you.
Harout Semerijian
Good morning, Roger. Excellent question. So as as we have mentioned over the last year, we have seen at two stages a slowdown in the number of events, which really led us to the collaboration with the FDA and the introduction of the time-based event trigger.
So currently, we are monitoring this very carefully, Roger. And what's happening is a lot of the patients as we are now almost two years out from full enrollment the follow up more a lot of times goes into quarterly rather than monthly. So we're evaluating that our next couple of months, and we should know by end of year and beyond.
But currently, what we are very pleased about is either through the events based trigger the time-based could trigger. We have a definitive cutoff by end of Q2 2024.
Roger Song
Excellent. I look forward to the next update. And also you guided you will submit that NDA by the end of this year, up by the end of 2024 if the primary analysis is positive. Just curious, any outstanding CMC and preclinical work you need to finish before you can file the NDA or anything else to step into that NDA filing?
Harout Semerijian
Yeah, Roger, we're in a very good shape. As we have been really in this trial, as you know, has taken longer than what any of us have anticipated driven by the fact that patients continue to live longer. Meanwhile we have done a very good job in terms of advancing CMC conversations, making sure that we have drug, making sure that we are preparing for an NDA filing with whatever we can do before we actually see the data as we continue to be blinded to that.
So we think we have a foundation for very rapid enough move into an NDA filing after the data should the data be positive. So that's why we have now in a position to say that we're going to be doing that before end of the year.
Roger Song
Excellent. Yes, you do have some additional time to prepare our the work. Okay. Maybe just last one from us, if I can is in terms of GBI-1687, the healthy volunteer data by the end of 1Q next year, so how should we think about that data? What you really looking for from that data to move forward? And I see that dose response bio-marker, that would be helpful. Thank you.
Harout Semerijian
Yes. Maybe I'll tackle some of the strategic perspective and if you want to add a few things about what we're going to be seeing.
So strategically, Roger, as you know, 1687 is our second generation E-selectin antagonist that is subcutaneously bioavailable, and that's why we're very excited to target sickle cell disease as our next area. As you know, we have a very deep tradition in that area with our first generation molecule.
So once we see and of course, the Phase 1a is really about safety. We want to make sure that we are doing all the building blocks to get to the next level and depending data and depending on where we are as well with not just the 301 our company-sponsored trial, but also the NCI sponsored Phase 2 in the frontline setting. Which continues to also take longer, depending on what what happens with these, we will be in a situation to say what do we want to do next and when after that. But if you wanted to maybe tackle the what's the Phase 1a
Brian Hahn
Sure, Roger. As you know, key information from many first-in-human trial will include safety, data and pharmacokinetics, in particular, will the pharmacokinetics support achievement of a potential therapeutic level of the study drug.
Based on prior GlycoMimetics experience, we're confident that GMI -1687 will perform and will demonstrate that and share it when available.
Roger Song
That's great. Thanks for taking the question. Thats it from my end.
Harout Semerijian
Thank you, Roger.
Operator
Boris Peaker, TD Cowen.
Boris Peaker
Great. Thanks taking my question. First, can you remind us maybe I missed that, how many patients in uproleselan study went on to transplant and also what the dropout rate wise?
Harout Semerijian
Yes, good morning Boris in which, in which study?
Boris Peaker
In your study specifically. But I guess if you notice for the NCI study as well, I guess that's a helpful, both of them.
Harout Semerijian
Yes. What we have announced Boris, as you know, is that in our Phase 2 trial in relapsed refractory, we have seen at 31% response rates. And as as I mentioned before, in our Phase 3, we are seeing a number north of that number. We have not disclosed the exact number. What we are saying, it's north of 31%.
Boris Peaker
And the dropout rate?
Brian Hahn
The dropout rate is 3%.A very low number.
Boris Peaker
My last question, if I may, NCI study, do you have a sense of why that's taking so long?
Harout Semerijian
Yeah we have our opinions, we have to wait for data to see what's happening. And as you know, the last patient enrolled in that trial in the Phase 2 population 267 patient was enrolled in December 2021. And that is in EFS primary endpoint for this up for the Phase 2.
So we are we are very intrigued and hopeful that that's happening because, events are not reaching. And if that's the case, then that's good news to patients, obviously. So we'll see once they reach the events, we are we collaborate with NCI, we continue to have dialogues with them and they have told us that when they get to the events, they will let us know. And until now they have not done.
Boris Peaker
Great. Thanks for taking my questions.
Harout Semerijian
Thank you, Boris.
Operator
I'm showing no further questions at this time. I would now like to turn the call back to Harout for closing remarks.
Harout Semerijian
Thank you, operator, and thank you to everyone for joining our call today.
We'll look forward to keeping you up-to-date on GlycoMimetics and seeing some of you at the Jefferies London Healthcare Conference or at the ASH in December. Thank you very much.
