Q4 2023 GlycoMimetics Inc Earnings Call
Good morning, and thank you for joining the glaucoma <unk> Q4, and full year 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 at that time lines will be opened for you I would now like to turn the call over to Christian Dinneen Long Company Counsel look like on my metrics. Please go ahead.
Good morning today, we will review our business updates and financial results for the quarter and year ended December 31, 2023 press release, we issued this morning is available on the company's website at <unk> Dot Com. This call is being recorded and a dial in phone replay will be available for 24 hours after the close of the call.
Webcast replay will also be available for 30 days in the investors section of the company's website.
On the call today from glaucoma metrics are hurts emergent and Chief Executive Officer, Brian Hahn, Chief Financial Officer, Dr. Edwin Rock, Chief Medical Officer, and Bruce Johnson, Chief Commercial 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 companys product candidate for a lessor in GMI 1687, the progress and timing of clinical trials being conducted by us or our collaborators, including our expectations regarding data readout from this trial.
<unk>, our potential regulatory agency interactions, our submissions development plans and activities prelaunch preparations and the company's cash position and runway.
Such statements represent management's judgment and intention as of today and involve assumptions risks and uncertainties glaucoma medics 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 Guac Amendment X website.
I'll now turn the call over to Eric Thank.
Thank you Christian and good morning, everyone.
In 2023, we made great strides on the path towards becoming a commercial company.
Building on the advances we made in 2023, I believe 2024 will be a transformational year for <unk>.
Eric: In the second quarter of this year, we expect to report topline results from our phase III trial of our lead drug candidate <unk>.
This is a significant milestone that can fundamentally impact our company's trajectory, while potentially helping patients affected with relapsed and refractory acute myeloid leukemia to live longer.
I want to thank the entire Glycomed X team, our shareholders collaborators investigators trial sites and patients for their trust and commitment and resilience. During this multiyear phase III trials, which has now reached critical maturity. Thanks.
Thanks to everyone's combined efforts, we're now very close to him blinding. This pivotal trial and should the data support. It we are ready to execute next steps rapidly today I would like to highlight three key strategic areas driving the transformation of <unk>.
First based on our prior alignment with the FDA, we are triggering the time based analysis for our pivotal phase III trial of <unk> in relapsed and refractory AML and expect top line results. In Q2 2024, we remain encouraged by the median follow up time, which is now well over three years.
Remarkably along for the relapsed and refractory population.
Pending positive results, we expect to submit a new drug application in the U S. By the end of this year.
Second we are further advanced we have further advanced our commercial readiness and continue to execute critical prelaunch activities, including the expansion of our commercial and medical affairs capabilities and educational disease awareness activities and.
And third we completed the phase one first in human trial for GMI 60, 87, a second generation is selectin antagonist being evaluated as an outpatient self administered subcutaneous therapy to potentially alleviate sickle cell badger perceived events IMAX.
I am excited to share that our phase <unk> has met its primary and secondary endpoints with no dose limiting toxicities or other safety signals.
As we look ahead to 2024 and beyond we believe <unk> is well positioned to deliver innovative glycobiarsol based medicines to patients in need of new treatment options, beginning with <unk> <unk>.
Despite recent advancements in AML therapies, there remains a significant unmet patient need, especially in terms of bending the survival curve upwards for relapsed and refractory patients.
With pulp with positive pivotal data <unk> has the potential to prolonged survival for patients with relapse and refractory AML.
This initial setting has a $650 million to $850 million near term potential market opportunity in the U S alone, which could more than double when considering the frontline AML market.
Were important partnerships with MD Anderson, the National Cancer Institute, and the Dana Farber Cancer Institute underscored <unk> unique mechanism of action and potential for broad utility across the AML spectrum.
Now turning to our finances, our disciplined approach focusing on targeted investments provides a current cash runway through year end 2020 for this.
This positions the company to be financed through our upcoming clinical milestones and data readouts and potential NDA submissions.
On today's call I'm happy to be joined by our CFO, Brian Hahn CMO, Dr. Red Rock and our CTO of Bruce Johnson I'll now pass it over to Ed to share more details on our ongoing trials.
Ed: So route and thank you to all on the line for joining us today.
As a reminder, in June 2023, the FDA cleared edition of an optional time based primary analysis to our phase III randomized trial of <unk> in relapsed and refractory AML.
This trial enrolled 388 patients and has a primary endpoint of overall survival.
Survival events have continued to slow over time. So we will proceed with a time based analysis after a data cut off at the end of this month.
We look forward to reporting topline results in Q2.
As Farooq mentioned median follow up for patients remaining on study will be over three years at time of analysis remarkable and a trial of therapy for relapsed and refractory AML.
Ed: Also a majority of surviving study patients received hematopoietic cell transplantation and a data cutoff a large majority of these patients will be at least two years post transplant. That's a notable milestone because after two years post transplant disease.
Relapse becomes infrequent.
Thus these phase III clinical trial data or clinically mature and support performance of time based primary analysis next quarter.
Our trial is testing two hypotheses.
Adjunct to view for Leslie and leads to deeper more durable measurable residual disease negative responses to therapy.
Second these deeper responses and reduced gastrointestinal toxicity enable more patients to get to and through potentially curative hematopoietic cell transplantation.
Rather than targeting a specific gene mutation <unk> is designed to be agnostic to cytogenetics gene mutation profile and backbone therapy.
Consistent with its molecular structure that mimics a natural complex carbohydrate <unk> demonstrates an unremarkable toxicity profiles and profile and trials conducted to date. So we see potential for broad <unk> utility in combination with diverse other AML treatments across.
<unk> lines of therapy.
Correspondingly.
<unk> potential outside of relapsed and refractory AML is under study and multiple ongoing investigator initiated trials across AML subtypes and lines of therapy. The largest of these trials is an adaptive NCI sponsored phase two three trial conducted by the alliance for.
Clinical trials in oncology.
This NCI Alliance study is testing neutral esol in newly diagnosed older patients with AML, who are fit for intensive chemotherapy.
The phase II portion has a primary endpoint of event free survival or Etfs and completed enrollment of 267 patients in December 2021.
Just this month NCI confirmed that the phase III <unk> event trigger has not yet been reached.
This trial was designed to show median PFS prolongation from 7% to 11 months, hence was expected to reach a phase II event trigger in 2022.
We look forward to sharing trial results when available.
As part of our collaboration the NCI also supports an ongoing childrens oncology group Phase one study conducted by Cogs pediatric early phase clinical trial network.
This dose escalation trial, which is part of the initial pediatric study plan agreed on with the FDA and EMA.
Assesses safety pharmacokinetics, and preliminary clinical activity of <unk>, plus chemotherapy in pediatric patients with relapsed or refractory AML.
Ed: Enrollment in this study is ongoing after first patient in occurred last October.
Additional ongoing investigator initiated trials continue to evaluate your progress land combinations in AML.
These trials include <unk> combinations with a conditioning regimen in patients up to 39 years old undergoing transplantation as well as with Azacitidine and <unk> in elderly patients with frontline AML.
In addition of <unk> in combination with low dose.
Cytarabine in Cladribine in patients with treated in secondary AML was recently updated at the 2023 Ash meeting.
And this is notoriously difficult to treat population all of whom had adverse cytogenetics and were previously treated with a hypo methylated agent Cladribine cytarabine and <unk> led to marrow blast reductions in 72% of 18 Evaluable patients.
The authors concluded that this combination provides a safe approach to Merrill <unk> reduction and disease control in preparation for potential hematopoietic cell transplantation.
In summary, we believe <unk> has broad potential utility across AML by targeting a novel form of chemo resistance from AML cell binding in bone marrow.
<unk> favorable safety profile makes it a good candidate for combinations with other AML therapies.
Finally, both of the two large ongoing randomized trials have seen slow event accumulation and that fact highlights potential for <unk> to become a valuable addition to diverse existing AML therapies.
Beyond <unk>, we have recently.
<unk>, our phase one single ascending dose trial of subcutaneous GMI $60 to 87.
We will evaluate this second generation E selectin antagonist as an outpatient self administered subcutaneous therapy to potentially alleviate sickle cell vasal occlusive events events at time of pain onset.
In addition to benefit from pain control such a point of care therapy may also reduce patient emergency room visits and hospitalizations.
<unk> first in human data in healthy volunteers support safety and fixed dose administration of subcutaneous GMI 16 87.
Ed: Full study results will be presented at an upcoming medical meeting.
Also we're pleased to announce that we've initiated a collaboration with the sickle cell disease clinical trials network of the American Society of Hematology research collaborative.
Through this relationship <unk> will obtain feedback from experts and people living with sickle cell disease on our GMI 16, 87 clinical development plan.
<unk> research collaborative clusters partnerships to expedite therapeutics development generate high quality evidence for clinical decision, making and improve outcomes for people living with sickle cell disease, we look forward to our partnership with them now.
Now I'll turn it over to Bruce to discuss the potential commercial opportunity pending positive results of our phase III trial.
Thank you Ed.
From a commercial perspective, there are a number of key factors that could position you for Leslie for success if approved.
As Farooq mentioned earlier on the call. Despite recent advancements in leukemia treatment. There remains a significant unmet need in AML. Currently this disease has a lowest survival rates in hematologic malignancies with a five year survival rate of around 30%.
The outcomes are progressively worse for elderly AML patients, who have a 15% five year overall survival rate and for relapsed refractory AML patients who have a 10% five year overall survival rate. Additionally.
Additionally, the vast majority of AML patients have no actionable mutation and therefore are not candidates for commercially available biomarker driven therapies for relapsed and refractory AML patients in particular outcomes remain dismal and there is currently no standard of care regimen for patients who are <unk>.
Eligible for intensive therapy thus.
<unk> novel, New treatment options that are complementary to existing standard therapy with little to no additive toxicity and our agnostic to mutation profile studied genetic risk and treatment backbone are desperately needed today.
Today hematopoietic cell transplantation remains the only treatment option with curative potential.
It has become increasingly clear that <unk> negative status prior to hematopoietic cell transplantation is a strong predictor of lower relapse rates and longer term survival post transplant.
Therefore, we have designed you for lesson as an adjunct to standard therapy with the goal of achieving deeper more durable <unk> negative remissions without additive toxicity, delivering more AML patients to and through a potentially curative hematopoietic cell transplantation.
<unk> has an unremarkable toxicity profile with no known drug drug interactions no pre medications no dose limiting toxicity, no qt prolongation or differentiation syndrome. It has been developed to be administered as a simple 20 minute.
IV infusion. We believe these features will provide a strong market advantage and potentially allow us to rapidly establish you for lesson as an important component of standard therapy across a variety of AML subtypes and lines of therapy.
We have been building focused market access strategies that complement our medical affairs capabilities.
External partnerships and teams to be ready to launch Super lesson should our pivotal study readout positively we are fortunate to have a critical mass of team members with long term connectivity with the hemo community with multiple successful launch experiences including in AML.
According to estimates from the American cancer Society in 2024, there will be more than 20000, new cases of AML and more than 11000 people will die from AML disease, relapse and unresponsiveness to standard therapy remain a significant problem with relapse rates of 50% to 60% after.
Michelle treatment.
We estimate that the relapsed and refractory adjustable market is a growing population with more than 8000 patients per year in an addressable market opportunity of $650 million to $850 million in the U S alone.
With its unique and differentiated profile, we believe <unk> can become an important adjunct to standard AML therapy.
The opportunity to be developed for future expansion into other settings, including frontline given it the potential to access a large and growing share of the over $4 billion U S market opportunity across the AML treatment continuum.
Ed: Now I'll turn it over to Brian for a review of financial results.
Brian: Thank you Bruce as of December 31, 2023, Black Amendment X had cash and cash equivalents $41 8 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 early in the year.
Company's research and development expenses decreased to $5 $3 million for the quarter ended December 31, 2023, as compared to $5 9 million for the fourth quarter of 2022.
Research and development expenses for year ended December 31, 2023 decreased to $20 1 million as compared to $28 4 million in the prior year. These decreases were due to lower clinical development expenses related to our ongoing global phase III clinical trial of <unk> in individuals with relapsed refractory AML and decreased manufacturing cost due to <unk>.
<unk> of engineering validation batches for U S land, partially offset with the completion of the phase one clinical trial for GMI $6 87.
The company's general administrative expenses decreased to $4 $3 million for the quarter ended December 31, 2023, as compared to $4 7 million for the fourth quarter of 2022.
General administrative expenses for the year ended December 31, 2023 increased to $19 $2 million as compared to $19 $1 million in the prior year.
These increases were due to higher personnel related expenses offset by a decrease in external consulting expenses I'd now like to turn the call back to <unk>.
Thank you, Brian and closing it has been a long road and we now have reached a very exciting time for our company. We are focused on the upcoming topline results from our phase III study of <unk>, plus Ron and relapsed <unk> refractory AML, we continue to work on our commercial readiness and are prepared to transition into a commercial.
Speaker Change: Stage company pending positive results I'd now like to open the lines to Q&A operator.
Thank you ladies and gentlemen, if you have a question or comment at this time. Please press star one on your telephone. If your question has been answered or you wish to move yourself from the queue. Please press star one again, we will pause for a moment, while we compile the Q&A roster.
Yeah.
Yes.
Our first question comes from Tara Bancroft with TD Cowen Your line is open.
Hi, Good morning. So my first question is whether you believe the frontline results could be available in time to potentially be included together with the relapse refractory data in a filing or what's the plan for that and then next.
<unk> phase III that Youre running is a successful can you describe how you think your pro will be used initially and how that could expand over time like which patients have a low hanging fruit because it does it include those mutations are.
Speaker Change: Will those be added over time.
Thank you Sarah good morning, maybe I'll turn it over to Bruce to address the second question about the potential sequence.
And then I'll take the first question go ahead for sure and thank you for the question I think initially.
Assuming positive results from the relapsed refractory pivotal trial, we see you per lesson.
Quickly being established as an adjunct to standard of care with patients receiving intensive.
Chemotherapy, we know there are a number of large volume.
AML centers of excellence that treat patients with intensive therapy.
Those would include patients with mutations you remember this is a therapy that is essentially agnostic to mutational profile set of genetic profile and treatment backbone. So we envision broad utilization initially with patients who are fit for intensive therapy beyond that of course.
We have ongoing trials Isps.
<unk> the unfit population and we'll wait for further data to readout on those thank you Bruce and regarding to your first question tariff or the regulatory pathway with the NCI led.
Frontline trial. So as you just heard we have reiterated that we have had conversations with the NCI and they have confirmed that they have not reached the RFS trigger yet which is getting quite unusual given the last patient in in that trial was in December 2021, with an <unk> trigger.
Rather than overall survival trigger which is our case.
Speaker Change: We kind of have to wait for that data until that happens and of course as you know the phase two three adaptive trial in the frontline setting is a registrational trial, so depending on the timing and depending on the data we will have that ability to either connect them together or have them separately, we can't make that assessment now.
Given how much delay there has been whenever that time data comes we're going to be ready to either do an NDA or have that to be a separate in parallel path. Because then it really opens the market to at least double the size of the relapsed refractory I hope I addressed your question.
Yes, you did thank you so much.
Thank you one moment for our next question.
Our next question comes from Irene <unk> with capital One Securities. Your line is open.
And good morning, and congrats on a bathroom. Thanks for taking my question I'm just curious in terms of the study is now time Dave.
Do you have a sense of what the event number is and would you report that with the top line data and then.
Quickly from the data cutoff, which will be this month would you be able to clean it up and report the top line I guess, what I'm asking is will you release it towards the end of June you or could it be slightly earlier.
Speaker Change: Yes. Thank you for the question, yes, we get asked that quite a bit.
Can imagine, yes, I mean, what we've said is we've seen.
A significant reduction in the number of events over multiple stages, which led us over.
And 'twenty two throughout 'twenty three to go back to the FDA, a couple of times and aligned with them on on additional path forward, where we are now is we are seeing a further slowdown of events, but given that we are aligned as of last summer with the with the agency on a time.
Analysis given that this database is now mature from a clinical perspective, both in terms of a median follow up of more than three years and the fact that the vast majority of the patients who've had a transplantation have a follow up of more than two <unk>.
Ears as well so that really makes the database quiet clinically mature end of the year, we're confident with this.
Method of triggering the data cutoff as you know we've also mentioned the end of March 31, So basically in a few days.
And then we got to allow the teams the timing that it takes to do the database cleanup.
The database lock their analysis that goes with it and then reporting hopefully.
Positive press release in Q2, so we obviously see the number of events in the back for our trial.
We are blinded, but we do see the number of events and then we're very confident in this time based analysis methodologies. So stay tune in Q2, we hopefully will have a good press release Thats why.
The aspiration.
Got it and can you share just a little bit more about this research collaboration that you have with Ashok <unk>.
Do you have any idea about the expected size of the study went in on this.
Any other details.
Sure Yes.
Sure I'll turn it to add.
Im very excited about this collaboration as you know sickle cell patients are still in dire need for treatment, especially with our approach and maybe Ed you can further expand on why we're excited with our collaborations.
Ed: The Ash research collaborative partners with multiple sponsors to give feedback on their study drugs in sickle cell disease. The disease indication that we are going to develop which is a point of care.
Method to alleviate sickle cell vasal occlusive events is unprecedented and we will appreciate getting their expert feedback boats.
Their investigators internally within the Ash research collaborators.
<unk> as well as from their patient Forum on our clinical development plan. So that we ensure that we are.
Keeping the patient perspective in mind first and tapping into the expertise of leaders in this field.
This is very exciting for us to be honest I mean, the fact that.
This unmet medical need and we know this is a disease area, where enrollment is difficult enrollment takes time and if you don't do it right from the beginning with a good collaboration it can it can lead to unfortunate.
Ed: Outcomes from a clinical clinical trial perspective.
We've learned that the hard way with our previous.
Generation asset and as you know if we have to stay true to the original design of having patients be exposed to our previous generation within the first 26 hours. There was a statistically significant benefit as we reported in our post hoc and blood last year. So this time, we really want to make sure we're working 10.
In hand with.
People, who are very much involved in the sickle cell community based on patient level bid on the investigators' level. So that we're working together in tandem to co create what would endpoints looked like which centers with we go so that we really are able to deliver a clinical trial that not only looks good on paper, but we can.
Actually get it done so I am very excited about this collaboration and hopefully we will report on progress in months to come.
That's helpful. Okay, sorry, one more question and then I guess, just looking back to the phase III study.
Speaker Change: Just final one for me.
Speaker Change: Assuming everything positive you plan it.
And then everything.
Before year end do you have any confirmed amongst E&P studies that you still need to complete before that.
We're supposed to do so we've been really cleaning a lot of the what's needed being on the data side, but also on the claim Barb do you want to.
Tackled the clean pharmaceuticals regarding clinical pharmacology in agreement with the FDA exposure response analysis and exposure toxicity analysis as well as population PK analysis are included in our phase III trial in our program. This will.
Accommodating FDA expectations and.
Ensure that we have appropriate information for the product label to direct safe and effective use of the drug.
Thank you Ed.
Okay. Thank you.
Okay. Thank you.
Speaker Change: Again, ladies and gentlemen, if you have a question or a comment at this time. Please press star one on your telephone one moment for our next question.
Our next question comes from Ed White with H C. Wainwright Your line is open.
Good morning, Thanks for taking my questions.
Perhaps I could just start with 16 87. This collaboration are there any financial.
Stipulations in this does it save you money somehow and then just.
If you can review your strategy for development. This is collaboration mean youre going to go it alone will you be looking for or will you be looking for a partner to further development.
Yes. Thank you Ed good to hear your voice.
Yes of course, I mean any collaboration for it to be effective we got to make sure that we're compensating peoples time and effort. So there is a financial component for their efforts.
It's modest but it's there.
And regarding your second part of the question are we going to do it alone or not I mean, that's really an open conversation.
What we would know is regardless, if we're doing it alone or not we want to advance <unk> hundred 87, and we wanted to make sure that we're learning more.
Before we advance it and move forward into full on clinical development program, we want to make sure that we're very.
Killing with the patient voice.
We're very much in tune with developing endpoints that can be delivered by physicians in a clinical trial setting, but also it's relevant for the patients. So we're gathering a lot of insights and regardless of how we forward the execution of those insights is going to be helpful. Anyway. So for US This is <unk>.
No regrets move with a very credible.
Speaker Change: Group, that's really is aligned with us on the.
The sickle cell patient outcomes. So that's why we're very pleased with our collaboration with them and then those.
Highlights will be used in due course, and a clinical development setting.
Speaker Change: Okay. Thanks Route and then my other question.
Police and that's just.
Can you give us your thoughts on your strategy for Europe, which is something that really haven't discussed all that much in the past, but since youre getting closer to two.
Potential launch in the U S. So they would start.
It's relevant to start thinking about that and get your thoughts on that yes.
Yes, no absolutely.
Speaker Change: And then those plans are ongoing they are underway. Our plans are not just to work with the FDA by also from a European perspective, we haven't guided on the European side, but the work is ongoing as you know our clinical trial, our phase III is half U S half globally.
<unk> many sites in Europe. So we have patients over there we have medical experts who are are exposed to your plus rent and know how to use it and of course, we're going to be working with the European agencies as well in due time. So the first focus is on FDA, but then we should be turning our attention to Europe as well. So that's also underway.
Okay, great. Thanks for taking my questions.
Speaker Change: Thank you.
And I'm showing no further question at this time I would like to turn the call back over to <unk> for any closing remarks.
Thank you operator, and thank you to everyone for joining our call today, we look forward to keeping you updated on graco metrics and sharing top line results in Q2. Thank you. This concludes today's call you may now disconnect.
Sure.
Okay.
Yeah.
Speaker Change: Okay.
Okay.
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