GlycoMimetics Inc (GLYC) 2020 Q3 法說會逐字稿

Good morning, and thank you all for joining GlycoMimetics Corporate Update Conference Call. (Operator Instructions) I would now like to turn the call over to Ms. Shari Annes of the Investor Relations Group at GlycoMimetics. Please go ahead.

Good morning. Today, we will review our accomplishments and financial results for the third quarter of 2020. We'll also be updating you on several more recent achievements primarily related to emerging data from the Phase III RESET trial evaluating rivipansel in vaso-occlusive crisis in sickle cell disease.

The press release we issued this morning is available on the company's website at www.glycomimetics.com under the Investors tab. This call is being recorded. A dial-in phone replay will be available 24 hours after the close of the call. The webcast replay will also be available in the Investor Relations section of the company's website for 30 days.

Joining me on the call from GlycoMimetics are Rachel King, Chief Executive Officer; Brian Hahn, Senior VP and Chief Financial Officer; and Dr. Helen Thackray our Senior VP of Clinical Development and Chief Medical Officer. We'll start today's call with comments from Rachel, and after that, Helen will review the new data with rivipansel. Following our sickle cell update, Brian will provide an overview of the company's financial position and we'll then open the call for Q&A. Our Co-Founder and Chief Scientific Officer, Dr. John Magnani, will join us in the Q&A to address your questions on the emerging preclinical data.

I'd like to remind you that today's call will include forward-looking statements based on current expectations. Forward-looking statements contained on this call include but are not limited to statements about the company's product candidates, uproleselan, rivipansel, GMI-1687 and GMI-1359 and our other pipeline programs, as well as the potential impact of the ongoing global COVID-19 pandemic on our clinical development, operations, and cash burn. 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 GlycoMimetics' filings with the SEC, which are available from the SEC or on the GlycoMimetics website.

I'd now like to turn the call over to Rachel.

Thank you, Shari, and thank you all for joining our call this morning. Let me begin by saying that the third quarter's momentum continues as we speak today. In both the oncology and sickle cell programs, we're making progress on 2 key objectives: first, to complete enrollment of our Phase III trial of uproleselan in the second half of next year; and second, following on encouraging additional analysis of the RESET study data and return of our rights to work in sickle cell disease to identify whether there's an opportunity for GlycoMimetics to move ahead to develop a therapy to treat acute vaso-occlusive crisis, or VOC, in sickle cell disease.

I'll begin with the uproleselan program. Enrollment in the company-sponsored pivotal Phase III trial evaluating uproleselan in patients with relapsed/refractory acute myeloid leukemia, or AML, continues according to plan. After slowdown at most of our sites in the earlier stages of the pandemic, the pace of enrollment for the trial has returned to forecasted levels. As a result, we are maintaining our prior guidance. We expect to complete enrollment in the second half of 2021. In our collaboration with the National Cancer Institute, or NCI, enrollment in that Phase III pivotal trial has also recovered after an initial slowdown due to the pandemic. Their focus is treating a newly diagnosed AML patient who's fit for intensive chemotherapy and 60 years of age or older.

Enthusiasm for both of these trials continues to be strong, highlighting uproleselan's potential to provide benefits across several clinical end points, including improving chemotherapy outcomes such as depth or duration of remission and overall survival, and ameliorating some of the serious adverse events of intensive cytotoxic chemotherapy. As we've said before, we believe our data supports our vision of uproleselan as a foundational treatment for AML across the spectrum of patients and as a key element of a variety of therapeutic regimens.

To that end, at the Society of Hematologic Oncology, in September 2020, our collaborators at MD Anderson Cancer Center presented statistically significant preclinical data pointed to the potential for a combination of uproleselan with venetoclax and HMA in AML to prolong survival. This was demonstrated in a xenograft model derived from the patient resistant to venetoclax and HMA treatment alone. The potential importance of this data set was underscored earlier this week when we announced that it was also accepted for oral presentation at the upcoming December annual meeting of the American Society of Hematology, or ASH. Clinical investigators have recognized the significance of this data given the relatively short duration of response to venetoclax combination therapy in this patient population. We're currently exploring options to take this combination forward in the clinic.

In sickle cell disease, emerging data from the Phase III RESET trial continue to highlight the importance of early intervention with fast-acting E-selectin antagonists that disrupt the underlying inflammatory mechanisms driving acute vaso-occlusive crisis. Our additional analysis of data from the RESET trial are providing valuable new perspective to us in our E-selectin programs primarily in support of the clinical benefit that can potentially be achieved by administering our target E-selectin product candidates early in acute VOC. We've presented findings at multiple sickle-cell-focused meetings and conferences, and at the upcoming ASH meeting, we plan to recap key secondary end point subgroup and subset data. We believe these data provide a clear biologic and clinical foundation for treatment with an E-selectin antagonist in acute VOC.

The FDA recently granted rivipansel a rare pediatric disease designation for the treatment of sickle cell disease in patients under 18 years of age, which recognizes the significant needs of pediatric patients. Should development of rivipansel in the pediatric setting advance, this designation could provide us with a priority review voucher. As we continue to roll out data from our completed post hoc analysis of the RESET trial, analyze new data from the open-label extension study, and continue to engage with the FDA, it remains premature to provide any guidance as to whether we will take the rivipansel program going forward.

The therapeutic options available for patients with sickle cell disease have increased substantially with recent approval. Even with these approved drugs however, the acute pain crises of sickle cell disease remain an area of high unmet need. And to our knowledge, there are no other late-stage therapies and development focused on acute pain crisis for adult and pediatric patients. As we continue to refine our analysis and approve our understanding of fast-acting E-selectin antagonism, our options, if any, will become more clear. We're actively exploring all opportunities, including options with GMI-1687.

With regard to GMI-1687, it's a more potent and more specific E-selectin antagonist than rivipansel and, importantly, bioavailable following subcutaneous administration. As such, we believe this product candidate may be ideally suited for treatment of acute VOC in the outpatient setting, potentially even providing patients with the opportunity to treat themselves at home in the early stages of VOC. We've received multiple invitations to give oral presentations at key sickle cell and hematology conferences on GMI-1687, the highlights of which I'll share after Helen's comments.

Finally, I'd like to leave you with my thoughts in the remainder of the year as well as 2021. The end of 2020 will be data-rich with multiple oral presentations and posters at ASH, covering GlycoMimetics' 4 compounds: uproleselan, rivipansel, GMI-1687 and GMI-1359. In all, we'll have 3 oral presentations and 2 poster presentations, which is a testament to the strength, novelty and relevance of our data. Within the coming year, we anticipate the completion of enrollment in our Phase III uproleselan trial in relapsed/refractory AML. This will clearly be an important milestone for this potentially foundational therapy. If realized, our success in the relapsed/refractory patient population could herald the arrival of a new treatment option for patients with AML.

In addition, as enrollment continues in our Phase Ib trial with GMI-1359 in patients with advanced breast cancer, we hope to share our findings in the first half of 2021. We will present preclinical data at ASH in December to highlight the importance of the biological activity demonstrated by targeting CXCR4 and E-selectin with that molecule. Importantly, as we've always operated on a lean and focused budget, we have cash to get through these milestones.

I'd now like to ask Helen to comment on the data we presented at the September meeting of the Foundation for Sickle Cell Disease Research, or FSCDR; the Annual Scientific Conference on Sickle Cell and Thalassaemia, or ASCAT, meeting in October; and the data accepted for oral presentation at ASH. Helen?

Thank you, Rachel. Our first analysis of new supportive data from the rivipansel Phase III RESET trial was reported at the virtual FSCDR meeting in September. There, investigators presented new efficacy and biomarker data. In particular, the data pointed to statistically significant improvement for patients treated early in acute vaso-occlusive crisis in the primary efficacy end point of time to readiness for discharge compared to placebo. For the group treated within 26.4 hours of onset of pain, which represents the first quartile, this primary end point analysis demonstrated a p-value of 0.03, a hazard ratio of 0.58 and a median improvement at 56.3 hours compared to placebo. In other words, if treatment with rivipansel was initiated early, patients receiving rivipansel were ready for discharge a median of over 2 days earlier than those patients randomized to placebo.

The context for this is the Phase III RESET trial, which evaluated 345 patients who are experiencing acute VOC and required hospitalization for treatment. We enrolled patients 6 years and above with a mean age of 22 years. Hence, we're often asked, I can explain that the time to readiness for discharge, or TTRFD, end point was the end point specifically agreed to with the FDA. It reflects achievement of multiple clinical criteria assessing health care utilization and the patient's medical improvement prior to leaving the hospital, including no longer needing IV opioids, IV hydration or other related treatments. These are generally the criteria that physicians and patients assess to determine when the patient is ready to manage his or her care with oral medications and therefore to be discharged from the hospital to home.

Equally important, we observed that patients treated with rivipansel showed a rapid, deep, sustained and statistically significant reduction in soluble E-selectin, a biomarker of vascular inflammation that is known to increase in acute VOC. We believe this shows that rivipansel has its intended on-target, biological effect with the resulting dampening of inflammation in the vasculature. The effect observed on soluble E-selectin in this trial provides valuable insight into the mechanism for the improvement in the clinical criteria for discharge from the hospital observed in those patients treated early in their acute VOC. Data from the RESET trial additionally demonstrate a favorable safety profile for rivipansel. The safety profile for rivipansel observed in this trial, as evaluated in a large population with pediatric, adolescent and adult patients, strengthens the potential risk-benefit proposition of rivipansel.

At the European ASCAT meeting in October co-hosted by the European Hematology Association and the British Society of Haematology, we were given the opportunity to share expanded findings from our analyses. The GlycoMimetics poster specifically highlighted new subset/subgroup efficacy data from the Phase III RESET trial and, additionally, data from key secondary outcomes in patients treated with rivipansel early in acute VOC. These new findings confirm the critical role of E-selectin in acute vaso-occlusion and the opportunities to resolve that occlusion and pain with effective intervention.

Specifically, the data for the pediatric patients, who represented 41% of all patients treated in the RESET trial, are striking. Data from children 6 to 17 years old in the study who were treated within 30 hours of VOC onset show a significant reduction in median time to readiness for discharge by 29.3 hours with a p-value of 0.02, a significant reduction in median time to discharge by 23.2 hours with a p-value of 0.02, a significant reduction in median time to discontinuation of IV opioids by 15.4 hours with a p-value of 0.045, and more children ready for discharge by 24, 48 and 72 hours compared to placebo.

Expanded data for the total population from the Phase III trial showed that those treated within 26.4 hours of the onset of acute pain also achieved statistically significant improvements in the same 2 key secondary end points, as just listed, for the pediatric group, namely time to discharge and time to discontinuation of IV opioids. Specifically, of the Phase III study's 320 evaluable patients, 80 individuals were treated within 26.4 hours of pain onset, earliest quarter (sic) [quartile] of duration of VOC until treatment. For those 80 patients, a comparison of those receiving rivipansel to those receiving placebo showed reduced median time to discharge by 41.5 hours from 112.8 to 71.3 hours with a p-value of 0.02 and reduced median time to discontinuation of IV opioids by 50.5 hours from 104 to 53.5 hours with a p-value of 0.03.

In sum, the new data demonstrate that treatment with rivipansel early in the course of acute VOC confirmed clinically meaningful improvements for 2 key secondary end points not previously reported, shortening IV opioid use and decreasing the hospital stay, for both the pediatric subgroup and for the total all-ages group in the trial. Furthermore, the favorable safety profile of rivipansel observed in the Phase III RESET trial, as now evaluated in a population that includes pediatric, adolescent and adult patients, is highly encouraging to us. We are very pleased that ASH has also chosen these data for an oral presentation at this year's annual meeting. The abstract posted this week also recaps data for the pediatrics subgroup and for all ages on the key secondary end points. As part of our oral presentation at ASH, we also intend to report on a new data analysis from the open-label extension trial. This study evaluated rivipansel in patients hospitalized for treatment of VOC subsequent to their initial administration of study drug in the Phase III RESET trial.

In summary, the additional analyses are compelling for us and for the medical community as to the critical importance of early aggressive intervention targeting E-selectin for the treatment of acute VOC. I look forward to your questions later in the call, and at this point, I'll ask Rachel to continue. Rachel?

Thank you, Helen. In addition to the exciting new rivipansel data indicating that E-selectin antagonism can reverse the course of acute painful episodes if used early, the data we've obtained with GMI-1687 has been well received and recognized with oral presentations at recent conferences and in December at ASH. This drug candidate is an E-selectin antagonist. It's more potent and more specific than rivipansel. We believe self-administration could move available treatment for acute VOC not only into the outpatient setting but potentially also into the home setting.

Our September 24 oral presentation of preliminary preclinical data at FSCDR meeting supported development of GMI-1687 as a possible follow-on to rivipansel, concluding that this product candidate has the potential for subcutaneous self-administration that's amenable for use in the outpatient setting. At ASCAT, in late October, another oral presentation disclosed new data from 2 different preclinical models and showed the drug candidate's efficacy as a subcutaneously administered treatment for VOC that prevents sickled red blood cell adherence to inflamed vasculature, inhibits vessel occlusion and restores normal blood flow. And at the upcoming ASH meeting, an abstract has been accepted for oral presentation that will once again highlight the product candidate's potential for intravenous and subcutaneous administration to treat VOC by inhibiting occlusions and restoring blood flow. A mouse model of VOC sickle cell disease is featured.

We believe we could be at a turning point in the treatment of acute VOC. We now have statistically significant data showing the benefit of early intervention. This finding has renewed interest in rivipansel but also highlighted the relevance of GMI-1687 as a possible superior follow-on product candidate that could change the face of VOC treatment by allowing patients to treat early in crisis at home. The idea of treating early has numerous precedents, including most recently the evolving treatment of hereditary angioedema, another acute inflammatory condition. You can see treatment with GMI-1687 potentially altering the treatment paradigm in a similar fashion with patients being able potentially to treat themselves at home at the earliest sign of the acute event to disrupt the underlying inflammatory cascade.

I'd now like to turn to Brian to both review the quarter's financial results and to provide his perspective on our financial position. Brian?

Thank you, Rachel. As of September 30, 2020, GlycoMimetics had cash and cash equivalents of $142.9 million as compared to $158.2 million as of December 31, 2019. During the quarter, the company received a $1 million clinical development milestone from Apollomics pursuant to the company's collaboration and license agreement for the development and commercialization of uproleselan and GMI-1687 in Mainland China, Hong Kong, Macau and Taiwan.

The company's research and development expenses were $10.7 million for each of the quarters ended September 30, 2020, and 2019. The company's research and development expenses decreased to $33.2 million for the 9 months ended September 30, 2020, as compared to $35.6 million for the same period in 2019. Manufacturing and formulation expenses decreased in the 3 and 9 months ended September 30, 2020, as compared to the same periods in 2019 as a result of lower raw material cost purchased in 2020. These decreases were offset by higher clinical expenses due to the increased enrollment in the ongoing global Phase III clinical trial of uproleselan in individuals with relapsed/refractory AML and the Phase II/III clinical trial being conducted by the National Cancer Institute in 2020 as compared to 2019. Contract research services, consulting and other costs were lower in the 3 and 9 months ended September 30, 2020, as research activities were affected at outside universities and travel by research and development personnel was largely eliminated due to the COVID-19 pandemic.

Now turning to G&A expenses. The company's general and administrative expenses increased to $4.1 million for the third quarter ended September 30, 2020, as compared to $3.4 million for third quarter of 2019. General and administrative expenses for the 9 months ended September 30, 2020, increased to $12.7 million as compared to $10.5 million in the same period in 2019. Personnel-related expenses increased due to additional general and administrative head count and annual salary adjustments awarded in the first quarter of 2020. Patent, legal fees, consulting and other professional expenses, including directors' and officers' insurance premiums, increased as compared to 2019. Other general and administrative expenses decreased for the 3 and 9 months ended September 30, 2020, as compared to the same periods in 2019 due to lower travel, meals and conference registration expenses as a result of travel restrictions imposed during the COVID-19 pandemic.

I'll now turn the call back over to Rachel.

Thank you, Brian. But before opening up the call to your questions, I'd like to reiterate our confidence in our clinical pipeline and, of course, in our specialized glycomimetic chemistry platform. Our chemistry insights have fueled several innovations that we believe have the potential to improve the standard of care in AML, and it may do the same in sickle cell disease and other diseases as well.

Now operator, please open the call for questions.

Your first question comes from Ed White with H.C. Wainwright.

So maybe I'll just start on rivipansel and 1689 (sic) [1687]. I'm curious to know. I know you haven't made a decision and you said that it is -- the timing isn't there yet to make the decision. But what information do you need to make a decision before rivipansel to advance?

And then also on 1687, how are you thinking about that development? Will you be preparing an I&D and looking to move into clinical development? Or do you have to decide first on what to do with rivipansel before you make a decision on the path forward for 1687?

Yes. Thanks, Ed. Thanks for your question. So let me put this into context. As you recall, a year ago, we weren't doing anything in sickle cell disease, and after the disappointing outcome of the RESET study, we weren't anticipating to do anything in sickle cell disease. What's changed for us, which we think is exciting and opened some opportunities for us potentially, is that we have this new analysis from the RESET study that shows this potential for early treatment benefit. And in addition, as part of Pfizer turning back the program to us, we now have the right to go forward in sickle cell disease generally, which we did not have as long as the program was licensed to Pfizer. So that gives us an opportunity to look at the pipeline that we have and to compare the opportunities and to decide what makes sense to go forward. And we are very encouraged by both what we've seen with the recent study and what we see with the preclinical data with 1687. So we're reviewing those in -- each in the context of the other. And there are a couple of things that we're looking at as we decide what makes sense for us to go forward.

With respect to rivipansel, we are planning to complete the data analysis, as Helen described, and we expect that at ASH, we will make a data presentation that will be substantially complete as to what we've learned from the RESET study. We've also -- we will look at that in the context of getting FDA input and looking at what we think it would require to take 1687 forward. And as we evaluate and compare the opportunities with those 2 programs, we'll be making a decision about what makes sense. But again, I think in the -- to put this into context, we do feel that the combination of the RESET data and the 1687 data are both encouraging with respect to potential opportunities in sickle cell disease, and we're now evaluating those and we'll determine what path forward makes the most sense.

Okay. Great. Thank you. And just on upro, you have your license agreement in Greater China. Will you be giving any update dates on the development there and the potential for time lines and everything in Greater China?

And then also, if you complete enrollment in the second half of '21 in your Phase III trial, when could we expect to see data?

So with respect to upro in China, we will give updates as that program progresses. And I think the next thing you might expect to hear would be an announcement when the study begins in China. So we will give updates as that progresses.

As far as the timing of data, that depends on 2 things, the data from the uproleselan trial. One is, of course, completion of enrollment. And the second is establishment or achievement of a certain number of events. And so we need to see how both of those play out. As we said, we do expect enrollment to complete in the second half of 2021. And then at that point, we may have a sense of how we're looking in terms of number of events. Unfortunately, in this trial by events, what I mean by that is the number of patients who have died in the aggregate population that then would enable us to compare the treatment versus the placebo arm. So we need to follow up for both of those and then we'll be able to look to data. But we do anticipate that the cash that the company currently has on hand would get us through that data readout.

Great. Thanks. And then my last question is just on 1359. Great to hear that the study has restarted. I think you had been expecting to enroll 6 to 12 patients. Can you give us an update as to how many have enrolled so far? And what -- you said that we should expect to see data in the first half of 2021. What data will we see at that time?

Sure, sure. So we haven't given specific updates on numbers of patients. But as you indicated, we do expect it to be a small number of patients. And just let me remind you what we are looking for in that study. That's the first trial of that drug candidate in patients, and it gives us an opportunity to look at some PK/PD biomarkers for the first time. And specifically, we're looking at biomarkers related to mobilization of certain cell types, for example mobilization of normal hematopoietic stem cells and mobilization of cancer cells that could be resident in the bone. So we're looking for a first indication of those PK/PD biomarkers, also gathering some additional safety data.

And in the aggregate, what we anticipate is that data would then put us in a position to move into what you might consider to be a more traditional expanded trial in a patient population, where we begin to look at more traditional efficacy-related end points. We've not yet defined what that patient population would be in this group. Obviously, we're looking at breast cancer patients. You've also seen some preclinical data from this program in osteosarcoma as well as other interesting indication. So we think there are a number of ways we could go with it, but we do expect that the initial data would be biomarker-related data.

Your next question is from Stephen Willey with Stifel.

This is Ellen on for Steve. Congrats on the data you've been able to present recently. So I know you're pretty limited in what you can provide in terms of rivipansel and next steps, but can you provide any details on the timing of maybe when you're having these discussions with FDA or when you expect to have these discussions with regards to next steps?

Yes. So we haven't provided specific guidance and we wouldn't on any specific conversations that we might have with the agency. But what we do -- what we will commit to is that once we have evaluated both any input we have from the agency as well as data that we have in hand that at that time when we made a decision about what we think are the definitive next steps, then we will provide guidance on that.

Okay. Great. And I think you mentioned this, but with regard to the upcoming data presentations at ASH for rivipansel, are those presentations representative of kind of the last batch of post hoc analysis we'll be seeing from RESET? Or do you expect to be sharing kind of more data, I mean, throughout early '21?

We do believe that the data presentation at ASH will be a substantially complete presentation on the data from the rivipansel program to date. And as Helen indicated, we anticipate that will include data from the RESET study as well as data analyzed from the open-label extension trial. So we expect that the data at ASH will be a substantially complete data presentation. We can't say that there might be some additional data that could be presented in publications going forward, but I think the ASH data would be a substantially complete presentation of the data.

Okay. Great. And then maybe one on uproleselan. With regard to that AML Phase III trial, I know you mentioned that enrollment has resumed its normal pace. That being said, as COVID is starting to spike in the US again, do you expect to take any COVID mitigation measures going forward to make sure that enrollment kind of stays on track? Or do you expect that to just not be an issue for whatever reason?

Well, I can't obviously predict what the impact of COVID may be, and that's certainly a concern, I think, for all of us in many respects. With respect to the clinical trial itself though, what we have seen is that because of the way the study is designed and the way the patients are treated, we think that it is well-positioned to advance through potential COVID impact. And I can turn to Helen for more specific comments on that, but I would generally observe that as compared to trials in other settings, we think a trial in the acute AML setting may be better positioned, as you said, to weather COVID-related delays. But it's -- it certainly can't predict what those might look like as the pandemic progresses. But Helen, could you comment further on the situation with the clinical trial?

Yes, yes. Thank you. So we do know that patients with acutely diagnosed refractory AML or relapsed require treatment that is in the hospital setting for the intensive chemotherapy that they receive. That is an acute need and one that proceeds regardless of the situation with the pandemic. So patients do need to be hospitalized for treatment, and that means that they are coming in for treatment and participating in the trial.

In terms of mitigation, there is one other element of the trial that I would I mention. That is we have seen getting some advantage to continuing accrual over the course of the pandemic, and that is the global nature of the trial. So we are not limited to any one country or region in terms of the sites participating in the trial. We have sites in North America, in Europe and in Australia. And when we have seen one region or area with increased severity of the pandemic, we've had other areas with more routine operations in terms of their hospital procedures and patient care. So that has allowed the trial to have a steady pace of enrollment as we would have expected globally, so in the aggregate, while there may be more local effects here and there as local areas are responding to the pandemic in their region.

Thank you, Helen.

Okay. Got it.

And so this...

Yes.

It's [unlike an active state]. I think that's the key point -- one of the key points.

Your next question is from Zegbeh Jallah with ROTH Capital.

Again, congrats on the abstract at ASH, particularly oral presentations. I was really excited. I think I'll just have 2 quick ones here. The first one is on rivipansel. I'm excited that this will be an oral at ASH. So a lot of clinicians will get to see the updated post hoc data, but I was just curious, as you've been engaging with KOLs, what some of that feedback has been like. I know we're planning to have our own KOL call coming up, but we're just wondering what your feedback has been.

Yes. So I think that's a question I'd also turn to Helen at this point or -- yes. Well, let me just turn that to Helen.

Thank you, Rachel, and thank you for the question. That's -- it's been very interesting. We are hearing and I think -- and in discussions with the investigators who've been involved in the trial. Confidence that the inhibition of E-selectin is having a clinical effect, as seen in this front group of patients from the trial who were treated early. The biomarker data is extremely supportive of that and there is, I think, excitement about the potential for a therapeutic candidate that could be used to treat this and they -- you know that there remains the complete unmet need for therapies that would interrupt the acute vaso-occlusive process. And so there has been strong interest engagement and excitement from the investigators around the evolving data set as we learn more about the effects of this protein for those treated early in their acute VOC.

Yes. And I think another perspective that we could add to this is that there is a -- as we think about this, there's a potential for the development of a new paradigm for how patients are counseled with respect to VOC. Currently, many times, patients are told to stay home. They are counseled to do all they can to manage the pain at home. And there's -- and of course, many people understand that they are reluctant to go to the emergency room where the standard of care is the delivery of opioids. If there were an alternative that could provide relief, now that could actually change the paradigm of how patients are advised. They may be advised to go earlier to the emergency room and to seek an earlier intervention in the crisis, which I think is an interesting opportunity in something that's also -- something that's being recognized by our -- by the KOLs. So we certainly also look forward to more KOL engagement as the data becomes even more widely known through the oral presentation at ASH.

And then just the other follow-up here. Excited to see that the upro plus HMA data will be an oral presentation as well. Even though the preclinical study outcome, I got excited about this one when I saw some early data at ASH last year, been pounding the table on it, so quite excited. And just thinking, because these are huge studies and these are going to be long studies, if this is something that you're going to explore clinically as well. So are you thinking about potentially partnering this? And then at what stage would you want to kind of get it as far as you can before partnering? Or would you be open to some kind of partnership as early as possible?

Yes. So thank you. And we -- yes, we are also very excited about the combination data with the -- with upro, venetoclax and the HMA. As you know, venetoclax has really come in as an important new treatment option for patients with AML. But sometimes the -- although the response rates are good, the CR -- full CR as a percentage of total responses is not what you'd like to see. And sometimes, the responses are not as durable as one might like to see. So the preclinical data really is exciting to us in that we do think that it does support moving this combination into the clinic.

So we remain very focused in terms of our operations on getting the Phase III trial finished with upro in the relapsed/refractory setting, as you know. But we have had some very strong interest from investigators in pursuing this combination therapy. So we are looking at how we might, in a very cost-effective way, take this forward in the clinic. And when we have specific plans with regard to that, we'll certainly let you know. And so I think -- I guess I would have to say stay tuned. What we're looking at is if -- is there a cost-effective way for us to begin to get an early look at the potential of this combination in the clinic. And as we bring together those plans, we'll certainly let you know.

At this time there are no questions. Speaker, do you have any closing remarks?

Just to thank everyone for participating in today's call. And we really -- we appreciate your questions, your interest and your support of the company. Thank you.

Ladies and gentlemen, thank you for participating in today's conference. This concludes today's program. You may all disconnect. Everyone, have a great day.