Crescent Biopharma, Inc.CBIONASDAQ
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Operator: Good morning, and thank you for joining the GlycoMimetics Investor and Analyst [indiscernible]
Please note this event is being recorded. Today's remarks will be followed by a question-and-answer session with the management team. [Operator Instructions]
I would now like to turn the conference over to Christian Dinneen-Long, Company Counsel at GlycoMimetics. Please go ahead.
Christian Dinneen-Long: Good morning. Today, we will review top line results from our Phase III study of our lead drug candidate, uproleselan.
A press release was issued this morning and is available on the company's website at glycomimetics.com.
This call is being recorded, and 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.
On the call today from GlycoMimetics are Harout Semerjian, Chief Executive Officer; and Dr. Edwin Rock, Chief Medical Officer. Brian Hahn, the company's Chief Financial Officer, will also be available for the question-and-answer session.
Today's call will include forward-looking statements based on our current expectations. Forward-looking statements may include, but are not limited to, statements about the company's product candidate, uproleselan, and analysis of data from the company-sponsored Phase III clinical trial; the progress and timing of clinical trials being conducted by our collaborators, including expectations regarding data readout from those trials; planned or potential data presentation; regulatory and development plans and activities; and the company's cash burn and budget plans. Such statements represent management's judgment and intention as of today and involve assumptions, risks and uncertainties.
GlycoMimetics undertakes no obligation to update or revise any forward-looking statement.
For information concerning the risk factors that could affect the company, please refer to GlycoMimetics' filings with the SEC, which are available from the SEC or through the GlycoMimetics website.
I'll now turn the call over to Harout.
Harout Semerjian: Thank you, Christian. Good morning, everyone. Earlier today, we announced top line results from our pivotal Phase III study of uproleselan or placebo in combination with MEC or FAI for the treatment of relapsed/refractory acute myeloid leukemia. Unfortunately, the study did not achieve a statistical significant improvement in overall survival in the intent-to-treat population.
Adverse events were consistent with known side effect profiles of the respective chemotherapies used in the study.
While this is not the outcome we were hoping for with data collected from 388 patients and an unprecedented follow-up of over 3 years, there will be important learnings from this study. We are diligently and rapidly evaluating the full data set with medical, statistical and regulatory experts and we plan to share further updates as appropriate.
We intend to present a comprehensive data analysis at an upcoming medical meeting and are developing a plan to discuss these data with the FDA given the significant unmet patient need.
I want to take a moment to offer my sincerest gratitude to the investigators, trial sites, and most importantly, the patients and families for their dedication to this large, well-controlled randomized trial.
I also want to thank the GlycoMimetics team for working tirelessly to develop uproleselan and our investors for their support.
We remain dedicated to exploring uproleselan's potential in AML. As a reminder, the National Cancer Institute is sponsoring an ongoing Phase II/III trial evaluating uproleselan in newly diagnosed older patients with AML who are fit for intensive chemotherapy. We look forward to sharing results from this trial when they become available.
I will now turn the call over to Ed.
Edwin Rock: Thanks, Harout. As a reminder, this study enrolled a total of 388 patients with relapsed and refractory AML across 70 sites in 9 countries and randomized them to uproleselan or placebo with both treatment groups receiving investigators' choice of either MEC or FAI chemotherapy.
All patients were randomized by November 2021, and the study had a low discontinuation rate of 3%.
In June 2023, the FDA cleared addition of an optional time-based primary analysis to this Phase III randomized study of uproleselan. And a time-based analysis was triggered with the March 31 data cutoff.
As Harout mentioned, our Phase III study did not meet its primary endpoint of achieving statistical significance in overall survival in the intent-to-treat population. Patients treated with uproleselan had a median overall survival of 13 months compared to 12.3 months in the placebo arm. The control group median survival of over 1 year is unprecedented in a randomized trial of therapy for relapsed and refractory AML. Also, adverse events in the uproleselan arm were consistent with known side effect profiles of the respective chemotherapies.
These features make this trial scientifically important and we will analyze these data thoroughly with medical, statistical and regulatory experts to understand these results and any lessons that could potentially benefit the AML population. We look forward to sharing results of our comprehensive analysis when available.
I'll now turn it back to Harout.
Harout Semerjian: Thanks, Ed. We remain committed to deepening our understanding of the study through rigorous analysis, and we look forward to sharing the full data set. We're also rapidly evaluating ways to reduce our cash burn as we complete the full analysis.
In the coming weeks, we expect to report back to investors regarding a revised budget. Additionally, we will share our updated plans for uproleselan as soon as reasonably possible.
I'd now like to open the lines to Q&A. Operator?
Operator: [Operator Instructions] Your first question comes from Ed White with H.C. Wainwright.
Edward White: So I just wanted to get your thoughts on why the study took so much longer to read out than you initially expected? And any thoughts on why, as you said, the median survival seen in the control group was unprecedented?
Harout Semerjian: Yes. Thanks, Ed. I mean, as a reminder for everyone, this trial had a primary endpoint of overall survival with a predetermined number to hit. The trial was taking longer because patients continued to live longer than what we anticipated. And given the 13 months of median overall survival in the uproleselan arm, but also the 12.5 months of median overall survival in the control arm, that is probably one of the highest control arms that we are aware of in a relapsed/refractory setting.
So that's what we know. Obviously, what's driving that? We -- this is where we are now running a full-on analysis across this data-rich trial to really understand additional insights. And once we have those, we plan to share them at a subsequent medical conference and also to the market as soon as we can.
Edward White: Okay. And just a question on the other studies that are ongoing. Is there going to be any impact on the NCI study? Is there an ability there to take an interim look before the anticipated data readout?
And then also is there any impact to Apollomics study in China?
Harout Semerjian: Yes. Probably the second question there about Apollomics, it's probably better off to ask to Apollomics, our partners. We wouldn't be in a situation to comment on their trials in China.
But regarding the NCI trial, as we have disclosed our last interactions in March, they still have not reached the EFS trigger at that time. And we are going to have additional conversations with the NCI team as part of our ongoing dialogue with them. And of course, in light of our data over here, we're going to have that conversation.
We -- it's a different -- it's an important thing to remember, it's a different line of therapy. It's a different backbone therapy. And it's a different endpoint. So those are important things to keep in mind is, yes, the broader area is both of them are under AML, but there are different lines, as I said, different combinations and different primary endpoints for the Phase II.
So we'll see once we see that data how that does today. But we don't think there's a tremendous impact or read-through from what we've seen over here, but we will know more once we actually see that data whenever that becomes available and they share as well.
Edward White: Okay. Harout, my last question is just on 1687, if you can just address that study and your thoughts on strategy going forward?
Harout Semerjian: Yes. 1687 is a very good asset. I mean, that is an asset that we have developed internally based on very key learnings from the previous generation. As you know, Ed, we have advanced it through IND. We've advanced it through Phase Ia. We do see that the drug candidate behaves as we wanted to.
Given that we're going to be looking at our financial situation and really revising a budget, it will be too early to say what else happens over there, but it's probably we're going to be looking at ways to conserve cash and really focus on deepening our understanding of what's going on with 301 and really work closely with regulatory partners, with clinical partners and with statistical partners as a full-fledged priority at this point.
Operator: Your next question comes from the line of Tara Bancroft with TD Cowen.
Tara Bancroft: Just one for me. So I'm wondering if you could discuss any sub-populations where it could be more effective? Like I know we won't know for sure until the full analysis, but if you had to postulate some potential outcomes, which types of patients do you think that this is most possible?
Harout Semerjian: Thank you, Tara, for the question. Yes, I mean, to your point, look, this is only a few days. We're literally 2, 3 days in, right, from when we got the data and we have an obligation to turn it around and communicate it.
So what we're communicating is really the top line. And at the same time, we're really paying tribute to -- with 388 patients and one of the longest follow-ups, we are going to have multiple different looks at it.
Obviously, when it comes to subgroups, the study is not actually powered for subgroups. However, the design does include multiple stratification factors such as age, disease status, backbone therapy. So it's really important for us to first conduct this comprehensive review of the full data set so that we can really understand what are the patients that we can help, are there additional signals in there because ultimately it's all under relapsed and refractory AML patient group, Tara. And as you know, those folks are in need of new therapies.
So it's our real duty to really look into it and really understand what's going on, on a sub-group level as well. And we are going to be conducting that as soon as possible.
Tara Bancroft: Okay. Great. And just one quick follow-up on that. So based on your engagements with the FDA, do you think the agency would be supportive of a potential filing in a more limited population?
Harout Semerjian: Yes. I wouldn't want to speculate or talk on behalf of the FDA at this point, it's just too early. But what I can say is we've had multiple, very good conversations with the agency as we were developing this trial. There are things that they've asked us to put in, which we did. And at the end of the day, this is relapsed and refractory AML. We do have fast track, breakthrough designation, orphan status for a reason because we're aligned with the FDA that those patients, relapsed and refractory AML patients, need new therapy options.
So we will do our part of deepening the understanding, having those conversations, reaching out both from a regulatory perspective, from a statistical perspective, but also from a clinical perspective and make sure that we are really advancing our understanding and engaging with partners such as the FDA to further look at the data. And based on what they see and if there is a path forward, then that would be obviously good news. But it would be too early to speculate at this point.
Operator: Your next question comes from the line of Naureen Quibria with Capital One Securities.
Naureen Quibria: Sorry to hear the news. I guess, I just have really one around MRD-positive/-negative patients. Do you know if MRD-positive patients proceeded on to transplant or was it just MRD-negative that went on to transplant? And are you able to comment on how many patients became MRD-negative in [indiscernible]?
Harout Semerjian: Thank you, Naureen, for the question. Yes. I mean, to your point, we have such data -- rich database with this patient, MRD-negativity, transplant rates, subsequent therapies, different backbone therapies, different stages of disease, Naureen. So all these are being looked at by the team as we speak. And as we learn more and more about it, we're going to be compiling these insights and having conversations with the regulatory agencies, such as the FDA.
So stay tuned. We are committed to deepening this understanding, given the rich database and given the high unmet medical need.
Operator: [indiscernible] back over to Harout for closing remarks.
Harout Semerjian: Thank you, operator. Thank you, operator, and thank you for everyone for joining our call today. So stay tuned. We are committed to updating the market as soon as we can. Thank you very much for your attention.
Operator: This concludes today's conference call. Thank you for your participation, and you may now disconnect.