Q3 2023 GlycoMimetics Inc Earnings Call
Okay.
Good morning, and thank you for joining the glycol Memetics 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 at that time lines will be opened for you if anyone should require operator.
Assistance. Please press Star then zero on your Touchtone telephone I would now like to turn the call over to Christian Dinneen Long company Counsel and glycol Memetics. Please.
Please go ahead.
Good morning today, we will review our business updates and financial results for the quarter ended September 32023. The press release issued this morning is available on the company's website at <unk> Dot Com. 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.
For 30 days in the investors section of the company's website.
Joining me on the call today from glaucoma vertex, our hurts emerging Chief Executive Officer, Brian Hahn, Chief Financial Officer, and Dr. Edwin Iraq, 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 companys product candidates <unk> GMI 16, 87, along with statements about the progress and timing of clinical trials being conducted by us or our collaborators planned or potential regulatory.
<unk> interactions or 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 Glycomed ex 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 our filings with the SEC, which are available from the SEC or through the glaucoma <unk> web.
Site.
I'll now turn the call over to Gary.
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 delivery plus around two AML patients in need of new treatment options, while we leverage our unique graco biology approach to develop innovative medicines and ended in additional diseases, such as sickle cell.
I would like to highlight three drivers that position us well to advance these programs and then coming gear.
We continue to expect top line results from our pivotal phase III study of <unk> in relapsed and refractory AML by the end of Q2 2024 and remain encouraged about the unprecedented median follow up time.
Should trial results be positive we accept we expect U S filing by end of 2024.
Second as part of our ongoing commitment to evaluate the utility of your plus around 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 <unk>, <unk>, seven and our sickle cell disease program. The phase one <unk> study remains on track for safety data by the end of Q1 2024.
With the addition of the time based analysis option to our pivotal phase III study of <unk> in the relapsed and refractory AML. We expect to report topline results by the end of Q2 2024, we look forward to unveiling. These data at this important milestone for patients investigators and the company.
While recently FDA approved drugs have shown encouraging results and limited AML subpopulations. We are optimistic that <unk> has the potential to improve patient outcomes irrespective of mutation profile cytogenetic risk or treatment backbone.
Building on the promise of <unk> in relapsed and refractory AML, we're 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 researchers.
From the MD Anderson cancer Center will present updated clinical data from their phase <unk> study evaluating <unk> plus run 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 <unk> 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 continue.
Finally, we expect to announce safety data from our phase one study of <unk> 687 by end of Q1 2024.
We expect these data will advance us towards our aspiration to develop <unk> 687, as 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 ahead of topline your philosophy around readout expected in Q2 2024.
Our educational disease awareness activities targeted academic and community Hematologists that care for AML patients.
Recently, we welcomed Dr. <unk> <unk> as our vice President of Global Medical Affairs, and Debra for Alta as our vice President of commercial operations.
Tomo and Deborah brings 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. Ed Rock I'll now pass it over to Ed to share more details on our ongoing trials.
Thanks, a route and.
Thanks to all of you on the line for joining our call today.
As Rudy mentioned, we expect to report topline results of our phase III trial by the end of Q2 2024 and at that time, we will have a clinically mature dataset for time based primary analysis of overall survival. If the 295 survival events that trigger primary analysis.
<unk> are not already reached by that time.
Median follow up for our phase III trial of <unk> 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 a therapeutic trial in relapsed and refractory AML.
Sure.
Also a substantial majority of surviving study patients received hematopoietic cell transplantation and the 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.
At least two years post transplant follow up for almost all transplant recipients where confidence in our time based primary analysis will provide adequate trial duration to demonstrate <unk> benefit if present.
Our biologic hypothesis is that adjunct to view for lesser Lynne 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 cider genetic risk where treatment backbone.
Correspondingly you pro Leslie and clinical activity is seen in both newly diagnosed and relapsed and refractory AML.
Importantly, <unk> appears to generate a notably one remarkable safety profile, adding no additional toxicity when combined with other therapies.
So we are optimistic that <unk> may one day be safely combined with diverse other therapies that are broadly useful for most or all AML patients.
Partner and independent investigator studies are further exploring potential benefit of <unk> across AML subtypes. Most importantly, a randomized NCI phase two three trial conducted by the alliance for clinical trials in oncology is evaluating <unk> in newly noodle.
We diagnosed older patients with AML, who are fit for intensive chemotherapy.
This trial completed phase II randomization of 267 patients in December 2021.
Phase II analysis of event free survival has been pending since then now almost two years since enrollment completion.
And the NCI recently confirmed to us that the event trigger for analysis Hasnt yet been reached.
For reference the phase II 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 II results when available.
Significantly NCI expanded our collaboration and now also supports a children's oncology group Phase one study conducted by their pediatric early phase clinical trial network.
This dose escalation trial will assess safety pharmacokinetics and preliminary clinical activity of <unk> plus chemotherapy in pediatric patients with relapsed or refractory AML, we're glad to announce that the first patient enrolled to this study in October.
Another investigator initiated trial led by Dr. John Horne from the Dana Farber Cancer Institute in Boston Children's Hospital also dosed its first patient earlier this year Dr.
Dr. Horns trial is evaluating <unk> with a 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 <unk> when treated secondary AML.
This rare very high risk study population is defined by prior chemotherapy treatment of an anti cedent hematologic disorder.
Nurses is abysmal with expected median survival of less than five months.
In the trial at MD Anderson investigators sought to generate a safe approach did marrow blast reduction disease control and potential for transplant by combining <unk> with low intensity chemotherapy of Cladribine and cytarabine.
Bad Prognostic features in the study population include adverse cytogenetic risk and prior hypo methylated agent use in all evaluable patients and prior hematopoietic cell transplantation and 25% of them.
Among 18 Evaluable patients as a data cutoff there were minimal therapy related toxicities, 72% showed a reduction in bone marrow blasts and one patient had a potentially curative transplant.
These results in is notoriously difficult to treat disease underscore broad potential utility of <unk> across the AML spectrum.
In addition, researchers from Washington University will presented at Ash safety and signal generating data from their trial of <unk> to reduce Gi toxicities of <unk> chemotherapy, given before transplant for multiple myeloma.
Beyond <unk>, we also made progress in our phase one single ascending dose trial of GMI 1687.
Generation E Selectin antagonist.
We will evaluate <unk> 16, 87, as a potential outpatient self administered subcutaneous therapy to interrupt sickle cell vasal occlusive crises.
E. Selectin plays a key mechanistic role in early progression of such acutely painful pathologic events and if successful GMI $60 87 offers potential for a point of care patient controlled treatment option at time of pain onset in.
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.
Sure.
Now I'll turn it over to Brian for a review of financial results.
As of September 32023, Psychomimetic had 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 32023, as compared to $4 9 million for the same period in 2020 to Dean.
The increased expenses were primarily due to the clinical development costs related to the phase <unk> trial of <unk> $6 87 in healthy adult volunteers, which was initiated in August 2023, partially offset by decreased personnel related and stock based compensation costs due to lower head count.
The company's general administrative expenses increased to $4 $5 million for the quarter ended September 32023, as compared to $3 $8 million for the same period in 2022. The increase expenses were primarily due to higher personnel related expenses and higher professional fees as the company advances <unk> and preparing for potential regulatory filings and commercialization.
I'd now like to turn the call back to <unk>.
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 III study of your plus around in relapsed and refractory AML and continuing strategic prelaunch activities to accelerate our transition.
To a commercial stage company after our topline results by end of Q2 of 2024.
We have the right team with the right experience in place and I am 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 your for us around potential across the AML spectrum. Thanks to studies conducted by institutions, such as the NCI Dana Farber Cancer Institute and MD Anderson Cancer Center.
Finally, we continue to progress our phase one study of <unk> seven towards initial data readout in Q1 2024.
In closing I want to thank our employees, whose hard work and dedication drive year for us for our forward as well as the patients and investigators that make these trials possible.
I'd now like to open the lines to Q&A operator.
Certainly as a reminder to ask a question. Please press star one on your telephone and wait for your name to be announced to withdraw. Your question. Please press star one again, please standby, while we compile the Q&A roster.
One moment for your first question.
Our first question will be coming from Roger song of Jefferies. Roger Your line is open.
Great. Thank you. Thanks for the update a couple of question on that one thing is.
Can you share with us how likely by the end of 'twenty two Q2 thousand 20 for you.
Primary analysis will be based versus the time based.
The awards.
<unk> been accumulation since last update thank you.
Yes, good morning, Roger excellent question, so as as we have mentioned over the last year, we have seen at two stages, if a slowdown in the number of events.
Which really led us to the collaborations with the FDA and the introduction of the time based.
Event trigger. So currently we are monitoring this very carefully Roger and whats happening is a lot of the patients as we are now almost two years out from full enrollment.
The follow up.
A lot of the times goes into quarterly rather than monthly. So we're evaluating that over the next couple of months and we should know better by end of year and beyond but.
But currently what we are very pleased about is <unk>.
Either through the event based trigger or the time based credit with trigger we have a definitive cutoff by.
By end of Q2 2024.
Excellent.
The next update.
Also you guided you will submit that.
NDA.
By the end of this year by the end of 2024, if the primary analysis is positive.
Curious any outstanding CMC preclinical work you still need to.
Finish before you can file the NDA or anything else.
The steps into that NDA filing.
Yes, Roger we're in very good shape.
In.
As we have been really 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 the foundation for a very rapid.
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.
Excellent, yes, you have to have.
Some additional time to prepare.
Exactly the work Okay, maybe just last one from us if I can in terms of $16 87, the healthy volunteer data by the end of <unk> next year. So how should we think about that data.
You really looking for from that data to move forward and I could that dose response biomarker that would be helpful. Thank you.
Yes, maybe I'll tackle some of the strategic perspective.
You want to add a few things about what we're going to be seeing so strategically Roger as you know 60 87 is our second generation <unk>.
<unk> antagonist that is subcutaneously bio available 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 <unk>.
<unk>.
So once we see of course the phase one.
It's really about safety, we wanted to make sure that we are doing all the building blocks to get to the next level.
And depending on the data and depending on where we are as well with not just the <unk>.
301, our company sponsored trial, but also the NCI sponsored phase II in the frontline setting, which which continues to also take longer depending on what happens with these we will be in a situation too.
Say, what do we what do we want to do next and when after that.
Ed if you want to maybe tackle.
What's the phase one sure Roger as you know key information from any first in human trial will include safety data and pharmacokinetics in particular will the pharmacokinetics to support.
Achievement of a potential therapeutic level of the study drug.
Based on prior Glycomed ex experience, we are confident the GMI $60 87 will perform and we will demonstrate that and share it wasn't available.
Awesome, that's great. Thanks for taking the question.
On the line.
Thank you Roger.
Again, if you would like to ask a question. Please press star one on your telephone.
And wait for your name to be announced.
Our next question will be coming from Boris <unk> of TD Cowen Your line is open.
Great. Thanks for taking my question first can you remind us.
Maybe I missed that how many patients in the study went on to transplant and also what the drop out rate wise.
Good morning, Boris and which in which study.
In your study specifically, but I guess, if you know what for the NCI study as well I guess it'd be helpful for both of them.
Yes, what we have announced versus as you know in our phase two trial in relapsed refractory, we had seen at 31% transplants rates and as as mentioned before in our phase III. 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%.
Got you and.
The drop out rate.
Ed.
Yes.
The dropout rate is 3%.
Patrick it's pretty low I guess my life.
So very low number yes.
My last question is on the NCI study do you have a sense of why that's taking so long.
Yes, I mean, we have our opinions obviously, but.
We have to wait for the data to see.
What's happening I mean, as you know the last patient enrolled in that trial and the phase III.
Population 267th patient was enrolled in December 2021, and that is in Etfs.
Primary endpoint for the <unk> for the phase III. 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 a good news to patients. Obviously, so we will see once they reach the events, where we collaborate with the NCI, we continued 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 so.
Great. Thanks for taking my questions. Thank.
Thank you Laura.
And Im showing no further questions at this time I would now like to turn the call back to her route for closing remarks.
Thank you operator, and thank you to everyone for joining our call today, we look forward to keeping you up to date on glaucoma ethics and seeing some of you at the Jefferies, London Healthcare conference or at the Ash in December.
Thank you very much.
Ladies and gentlemen. This concludes today's conference. Thank you for your participation you may now disconnect.
Okay.
[music].
Okay.
Okay.
Yes.
[music].
Okay.
Okay.
Okay.
[music].
Yes.
Yes.
[music].
Sure.
[music].
Yes.
Okay.
Okay.
Okay.
[music].
Sure.
[music].
Yes.
[music].
Okay.
Yes.
[music].
Yes.
Okay.
[music].
Yes.
Yes.
[music].
Okay.
[music].
Sure.
Okay.
[music].
Okay.
[music].
Okay.
[music].
Thanks.
[music].
Okay.
[music].
Okay.
[music].
Thanks.
Yes.
Okay.
[music].
Sure.
Sure.
Right.
Yes.
Yes.
[music].
Yes.
[music].
Alright.
Sure.
[music].
Yes.
Sure.
Okay.
Okay.
Sure.
Yes.
[music].
Sure.
Okay.
Okay.
Okay.
Okay.
Thank you.
Okay.
[music].
Yes.
Okay.
[music].
Okay.
Yes.
Okay.
[music].
Yes.
Okay.
Okay.
Okay.
Sure.
Okay.
Okay.
Yes.
Yes.
Sure.
Okay.
Okay.
[music].
Yes.
Yes.
Yes.
Okay.
Yes.
Okay.
Great.
Okay.
Okay.
Okay.
[music].
Okay.
[music].
Thanks.
Okay.
Okay.
Yes.
Okay.
[music].
Sure.
Sure.
Okay.
Okay.
Okay.
Okay.
Yes.
Okay.
Okay.
Yes.
Yes.
Thanks.
Okay.
Yes.
Thank you.
Sure.
Okay.
Yes.
Okay.
Thank you.
Okay.
Yes.
Yes.
Sure.
Yes.
Sure.
Thanks.
Yes.
Okay.
Thanks.
Yes.
Okay.
Okay.
Okay.
Okay.
Okay.
Thank you.
Thank you.
Thanks.
Thank you.
Thank you.
Okay.
Thank you.
Okay.
Okay.
Yes.
Okay.
Okay.
Thanks.
Yes.
Thank you.
Okay.
Yes.
Thank you.
Okay.
Okay.
Yes.
[music].
Thank you.
[music].
Thank you.
Thank you.
Yes.
Okay.
Okay.
Sure.
Okay.
Okay.
Okay.
Yes.
Okay.
Okay.
Okay.
[music].
Yes.
Thanks.
Okay.
Okay.
Yes.
Okay.
Okay.
[music].
Yes.
Sure.
Okay.
Thank you.
Okay.
Yes.
Thanks.
Okay.
Okay.
[music].
Okay.
Okay.
Okay.
Okay.
Okay.
Okay.
Okay.
Yes.
Okay.
Yes.
Okay.
Okay.
[music].
Okay.
Yes.
Okay.
Okay.
Okay.
Sure.
Yes.
Okay.
Okay.
Right.
Yes.
Thanks.
Okay.
Yes.
Sure.
Okay.
Okay.
Yes.
Okay.
Yes.
Okay.
Okay.
Okay.
Okay.
Okay.
Okay.
Yes.
Sure.
Okay.
Okay.
[music].
Yes.
Okay.
Okay.
Okay.
[music].
Okay.
Yes.
Thanks.
Okay.
Okay.
Okay.
Okay.
Okay.
Thank you.
Sure.
Thank you.
Great.
Okay.
Sure.
Okay.
Okay.
Great.
Okay.
Yes.
Okay.
Okay.
Yes.
Okay.
Great.
Okay.
Okay.
Okay.
Hi.
Okay.
Yes.
Thanks.
Thanks.
Okay.
Okay.
Okay.
Great.
Okay.
Okay.
Okay.
Yes.
Okay.
Okay.
Yes.
Okay.
Thank you.
Yes.
Yes.
Yes.
[music].
Yes.
[music].
Okay.
Okay.
Hum.
Yes.
Okay.
Yes.
[music].
Great.
[music].
Okay.
Okay.
Yes.
Okay.
Yes.
Yes.
Okay.
Yes.
Okay.
Okay.
Yes.
Good morning, and thank you for joining the glycol Memetics 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 at that time lines will be open for you if anyone should require.
Operator assistance. Please press Star then zero on your Touchtone telephone.
I'd now like to turn the call over to Christian Dinneen Long company counsel at Guaco Memetics.
Please go ahead.
Good morning today, we will review our business updates and financial results for the quarter ended September 32023. The press release issued this morning is available on the company's website at Glycomed X Dot Com. 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.
For 30 days in the investors section of the company's website.
Joining me on the call today from Glaucoma, <unk>, 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 companys product candidates <unk> <unk> hundred 87, along with statements about the progress and timing of clinical trials being conducted by us or our collaborators planned or potential regulatory.
<unk> interactions, our 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 Glycomed ex 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 our filings with the SEC, which are available from the SEC or through the glaucoma <unk> web.
Site.
I'll now turn the call over to her.
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 a year plus around two AML patients in need of new treatment options, while we leverage our unique glycobiarsol approach to develop innovative medicines and ended in additional diseases, such as sickle cell.
I would like to highlight three drivers that position us well to advance these programs and then coming gear.
We continue to expect top line results from our pivotal phase III study of <unk> in relapsed and refractory AML by the end of Q2 of 2024 and remain encouraged about the unprecedented median follow up time.
Should trial results be positive, we accept and we expect U S filing by end of 2024.
Second as part of our ongoing commitment to evaluate the utility of your plus around 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 <unk>, <unk>, seven and our sickle cell disease program. The phase one <unk> 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 III study of <unk> in the relapsed and refractory AML. We expect to report top line results by the end of Q2 2024, we look forward to unveiling. These data at this important milestone for patients investigators and the company.
While recently FDA approved drugs have shown encouraging results and limited AML subpopulations. We are optimistic that <unk> has the potential to improve patient outcomes irrespective of mutation profile cytogenetic risk or treatment backbone.
Building on the promise of <unk> in the relapsed and refractory AML, we're 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 researchers.
From the MD Anderson cancer Center will present updated clinical data from their phase <unk> study evaluating <unk>, plus Ron 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 <unk> 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 continue.
Finally, we expect to announce safety data from our phase one study of <unk> 687 by end of Q1 2024.
We expect these data will advance us towards our aspiration to develop <unk> 687, as 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 ahead of topline year plus around readout expected in Q2 2024.
Our educational disease awareness activities targeted academic and community Hematologist that care for AML patients recent.
Recently, we welcomed Dr. <unk> <unk> as our vice President of Global Medical Affairs, and Debra for Alta as our Vice President of commercial operations date.
<unk> tomo and Deborah 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.
On today's call I'm happy to be joined by our CFO, Brian Hahn and our CMO, Dr. Ed Rock I'll now pass it over to Ed to share more details on our ongoing trial.
Thanks, <unk> and thanks to all of you on the line for joining our call today.
As <unk> mentioned, we expect to report topline results of our phase III trial by the end of Q2 2024 at that time, we will have a clinically mature dataset for time based primary analysis of overall survival. If the 295 survival events that trigger primary analysis.
We're not already reached by that time.
Median follow up for our phase III trial of <unk> in relapsed and refractory AML stands now at 33 months and will be greater than three years at time of primary analysis.
It's unprecedented for a therapeutic trial in relapsed and refractory AML.
Also a 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.
Two years of post transplant follow up for almost all transplant recipients were confident that our time based primary analysis will provide adequate trial duration to demonstrate <unk> benefit if present.
Our biologic hypothesis is that adjunct a viewport Westland 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 cider genetic risk where treatment backbone.
Correspondingly <unk> and clinical activity is seen in both newly diagnosed and relapsed and refractory AML importantly.
Importantly, <unk> appears to generate a notably one remarkable safety profile, adding no additional toxicity when combined with other therapies. So we are optimistic that <unk> may one day be safely combined with diverse other therapies that are broadly useful for most or all AML patients.
Partner and independent investigator studies are further exploring potential benefit of <unk> across AML subtypes. Most importantly, a randomized NCI phase two three trial conducted by the alliance for clinical trials in oncology is evaluating <unk> in newly.
<unk> newly diagnosed older patients with AML, who are fit for intensive chemotherapy.
This trial completed phase II randomization of 267 patients in December 2021.
Phase III analysis of event free survival has been pending since then now almost two years since enrollment completion.
And the NCI recently confirmed to us that the event trigger for analysis Hasnt yet been reached.
For reference the phase II 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 II results when available.
Significantly NCI expanded our collaboration and now also supports a children's oncology group Phase one study conducted by their pediatric early phase clinical trial network.
This dose escalation trial will assess safety pharmacokinetics and preliminary clinical activity of <unk> plus chemotherapy in pediatric patients with relapsed or refractory AML, we're glad to announce that the first patient enrolled to this study in October.
Another investigator initiated trial led by Dr. John Horne from the Dana Farber Cancer Institute in Boston Children's Hospital also dosed its first patient earlier this year Dr.
Dr. Horns trial is evaluating <unk> with a 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 <unk> when in treated secondary AML.
This rare very high risk study population is defined by prior chemotherapy treatment of an anti cedent hematologic disorder.
Gnosis is abysmal with expected median survival of less than five months in the trial at MD Anderson investigators sought to generate a safe approach did marrow blast reduction disease control and potential for transplant by combining <unk> with low intensity chemotherapy.
Cladribine and cytarabine.
Bad Prognostic features in the study population include adverse cytogenetic risk and prior hypo methylated agent use in all evaluable patients and prior hematopoietic cell transplantation and 25% of them.
Among 18 Evaluable patients as a data cutoff there were minimal therapy related toxicities, 72% showed a reduction in bone marrow blasts and one patient had a potentially curative transplant.
These results in is notoriously difficult to treat disease underscore broad potential utility of <unk> across the AML spectrum.
In addition, researchers from Washington University will presented at Ash safety and signal generating data from their trial of <unk> to reduce Gi toxicities of <unk> chemotherapy, given before transplant for multiple myeloma.
Beyond <unk>, we also made progress in our phase one single ascending dose trial of GMI 16, 87, our second generation E Selectin antagonist.
We will evaluate GMI $60 87, as a potential outpatient self administered subcutaneous therapy to interrupt sickle cell vasal occlusive crises.
E. Selectin plays a key mechanistic role in early progression of such acutely painful pathologic events.
And if successful <unk> 16, 87 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.
Thank you Ed.
As of September 32023, cyclone vertex had 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 32023, as compared to $4 9 million for the same period in 2020 to the.
The increased expenses were primarily due to the clinical development costs related to the phase <unk> trial of <unk> $6 87 in healthy adult volunteers, which was initiated in August 2023, partially offset by decreased personnel related and stock based compensation costs due to lower head count.
The company's general administrative expenses increased to $4 5 million for the quarter ended September 32023, as compared to $3 8 million for the same period in 2022. The increase expenses were primarily due to higher personnel related expenses and higher professional fees as the company advances <unk> land and prepares for potential regulatory filings and commercialization.
I'd now like to turn the call back to <unk>.
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 III study of your plus around in the relapsed and refractory AML and continuing strategic prelaunch activities to accelerate our transition.
To a commercial stage company after our top line results by end of Q2 of 2024.
We have the right team with the right experience in place and I am confident that we will be able to build upon our collective track record of successful commercial launches should data permit.
So we continue to explore <unk> potential across the AML spectrum. Thanks to studies conducted by institutions, such as the NCI Dana Farber Cancer Institute and MD Anderson Cancer Center.
Finally, we continue to progress our phase one study of <unk>, 7% towards initial data readout in Q1 2024.
In closing I want to thank our employees, whose hard work and dedication drive <unk> forward as well as the patients and investigators that make these trials possible.
I'd now like to open the lines to Q&A operator.
Certainly as a reminder to ask a question. Please press star one on your telephone and wait for your name to be announced.
Withdraw your question. Please press star one again, please standby, while we compile the Q&A roster.
One moment for our first question.
Okay.
Our first question will be coming from Roger song of Jefferies. Roger Your line is open.
Great. Thank you. Thanks for that update a couple of question on that one thing is.
Can you share with us how likely by the end of 'twenty two Q2 thousand 20 for you.
Primary analysis will be based versus the time based.
The awards, how they <unk> been accumulation since last update thank you.
Yes, good morning, Roger excellent question, so as as we have mentioned over the last year, we have seen at two stages.
So down 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 whats happening is a lot of the patients as we are now almost two years out from full enrollment.
The follow up more of a.
A lot of the times goes into quarterly rather than monthly. So we're evaluating that over the next couple of months and we should know better by end of year and beyond.
But currently what we are very pleased about is either through the event based trigger or the time based credit with trigger we have a definitive cutoff by end of Q2 2024.
Excellent look forward too.
The next update.
And also you guided you will submit the NDA.
By the end of this year.
And of the 224, if the primary analysis is positive.
Curious any outstanding CMC preclinical work you still need to finish.
Finish before you can file the NDA or anything else.
The steps into that NDA filing.
Yes, Roger we're in very good shape.
<unk>.
As we have been really 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 the foundation for a very rapid.
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.
Excellent, yes, you have to have.
Additional time to prepare all that exactly work.
Maybe just last one from us if I can is in terms of 16 87, the healthy volunteer data by the end of <unk> next year. So how should we think about that data.
You really looking for from that data to move forward and I could that dose response, all biomarker that would be helpful. Thank you.
Yes, maybe ill tackle some of the strategic perspective.
Want to add a few things about what we're going to be seeing so strategically Roger as you know 60 87 is our second generation.
Select antagonist that is subcutaneously bio available.
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 of course, the phase one a M.
It's really about safety, we want to make sure that we are doing all the building blocks to get to the next level.
And depending on the data and depending on where we are as well with not just the.
301, our company sponsored trial, but also the NCI sponsored phase II in the frontline setting, which which continues to also take longer depending on what happens with these we will be in a situation too.
Say, what do we what do we will do next and when after that.
But if you want to maybe tackle.
What's the phase one sure Roger as you know key information from any first in human trial will include safety data in pharmacokinetics in particular will the pharmacokinetics to support.
Achievement of a potential therapeutic level of the study drug.
Based on prior Glycomed ex experience.
We're confident that GMI 16, 87 will perform and we will demonstrate that and share it wasn't available.
Awesome that's great.
Thanks for taking the question.
On the line.
Thank you Roger.
Again, if you would like to ask a question. Please press star one on your telephone.
For your name to be announced our next question will be coming from Boris <unk> of TD Cowen. Your line is open.
Great. Thanks for taking my question.
Can you remind us.
I missed that how many patients in the <unk> study went on to transplant and also what the dropout rate was.
Good morning, Boris and which in which study.
In your study specifically, but I guess, if you know what for the NCI study as well I guess it'd be helpful for both of them.
Yes, what we have announced <unk> as you know.
Our phase two trial in relapsed refractory, we had seen at 31% transplants rates and as as mentioned before in our phase III. 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%.
Got you and.
The drop out rate.
Ed.
Yes.
The dropout rate is 3%.
Got it pretty well I guess my last question.
A very low number yes.
My last question is on the NCI study do you have a sense of why that's taking so long.
Yes, I mean, we have our opinions, obviously, but we have to wait for the data to see.
What's happening I mean, as you know the last patient enrolled in that trial and the phase III.
Population 267th patient was enrolled in December 2021, and that is in Etfs.
Primary endpoint for the <unk> for the phase III. 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 a good news to patients obviously.
We will see once they reach the events, where we collaborate with the NCI, we continued 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 so.
Great. Thanks for taking my questions. Thank.
Thank you Laura.
And Im showing no further questions at this time I would now like to turn the call back to her route for closing remarks.
Thank you operator, and thank you to everyone for joining our call today, we look forward to keeping you up to date on glaucoma ethics and seeing some of you at the Jefferies, London Healthcare conference or at Ash in December thank.
Thank you very much.
Ladies and gentlemen. This concludes today's conference. Thank you for your participation you may now disconnect.