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GlycoMimetics Inc  (NASDAQ:GLYC)
Q4 2018 Earnings Conference Call
March 06, 2019, 8:30 a.m. ET

Contents:

  • Prepared Remarks
  • Questions and Answers
  • Call Participants

Prepared Remarks:

Operator

Good morning, and thank you all for joining the GlycoMimetics call. At this time, all participants are in listen-only mode. Following management's remarks we will hold a brief question-and-answer session. And at that time the lines will be open for you. (Operator Instructions)

I would now like to turn the call over to Shari Annes of the Investor Relations group at GlycoMimetics. Please go ahead.

Shari Annes -- Investor Relations

Good morning. Today, we will highlight the key achievements of 2018, provide a summary of our financial results for the year end and comment on the milestones we anticipate in the next few months. The press releases we issued this morning on our year-end and Q4 financials and yesterday on our Board transition are available in the News section of the Company's website at www.glycomimetics.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 in the Investor Relations section of the Company's website for 30 days.

Joining me on the call today from GlycoMimetics are Rachel King, Chief Executive Officer; Brian Hahn, Chief Financial Officer; and Dr. Helen Thackray, Senior VP of Development and Chief Medical Officer. We will start today's call with comments from Rachel, and after that, Brian will provide an overview of the Company's financial position. We will then open the call for Q&A.

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 development plan for uproleselan, formerly known as GMI-1271; and for rivipansel, GlycoMimetics' product candidate licensed to our collaborator, Pfizer; and as well as our other pipeline programs. 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. Please refer to GlycoMimetics' filings with the SEC, which are available from the SEC, or on the GlycoMimetics website for information concerning the risk factors that could affect the Company.

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

Rachel K. King -- Chief Executive Officer

Thank you. Shari, and thank you all for joining our call. This morning I'd like to cover three topics: first, upcoming Phase 3 results for rivipansel in patients experiencing sickle cell crisis; next the major accomplishments of 2018; and finally, the milestones we expect to achieve in the coming months.

I believe we're poised for important accomplishments in 2019, all of which were made possible by the achievements of last year. As you may recall, Pfizer has said they intend to announce top line results from the Phase 3 RESET trial of rivipansel at the end of the second quarter of this year. The anticipated timing of this readout has not changed. With less than four months until the Phase 3 readout, our enthusiasm is high. The Phase 3 readout is an important milestone that very few drugs in development reach.

Since our discovery of rivipansel, we've focused chemistry, preclinical and clinical development resources to derisk and advance the program. Today, we believe we're on the cusp of delivering a potential therapy for sickle cell crisis that could transform the way patients with this devastating disease are treated. This has been our goal for the program since its inception. While the pivotal data will tell the ultimate story, we do believe that the rivipansel study has a high probability of success and if positive that this novel on demand agent could be utilized extensively in the major markets. We base this confidence on a number of factors: first, the data from our own Phase 2 trial, which among other things demonstrated an 83% reduction in opioid use throughout the duration of hospitalization, a finding that gives us confidence that the drug is having its intended biological effect; second, the fact that selected inhibition has now been clinically validated as a therapeutic target based on data from our controlled trial as well as other programs that have demonstrated the selected inhibition can reduce the incidence of VOC; third, the sample size and powering of the RESET trial, which includes 350 patients, approximately four-fold larger than our Phase 2 trial. We believe this more than adequately powers the trial to demonstrate the statistically significant result on the primary endpoint as well as on key secondary endpoints. The fact that this is the only on demand therapy for sickle cell crisis that is in late stage development and also importantly the subject of a special protocol assessment by the FDA that's the fourth reason. And finally, last but not least, as part of the Phase 3 program, clinical and pharmacoeconomic outcomes are being rigorously assessed allowing this data to service the basis for regulatory pricing and reimbursement discussions.

We believe that reducing the hospital stay would be meaningful to patients, physicians, regulators and payors. Furthermore, we expect this clinical benefit along with reductions in opioid use to strongly position rivipansel for regulatory and commercial success. As you could imagine rivipansel's market potential was a key consideration as we began our work in sickle cell disease. But commercialization and details of market planning are not Pfizer's responsibility. So I'd like to reiterate Pfizer's recent public statements describing rivipansel as a potential blockbuster. They estimate possible annual peak sales of greater than $1 billion.

What would a positive outcome in the Phase 3 rivipansel trial mean for us. First and foremost, we would be deeply grateful if we can contribute to transforming and improving the lives of people with sickle cell disease, that would be personally meaningful to each of us and it's been a long time goal of ours that has been motivating us since the program began.

Second, a successful outcome with rivipansel, a drug discovered, synthesized and developed through Phase 2 by GlycoMimetics which serve as validation for our unique GlycoMimetics discovery platform. In particular, it would confirm the value of targeting selectin and our ability to translate our understanding of carbohydrate chemistry into novel therapeutics. We believe this would reinforce our leadership in the field of Glycobiology, an emerging space and an untapped source of novel therapeutics.

Third, I'd like to remind you of the potential financial benefits to GlycoMimetics. Under our agreement with Pfizer, GlycoMimetics is entitled to receive development, regulatory and commercial milestone payments of up to $285 million in the aggregate, with the next milestone payment due to us on acceptance of an NDA. After that we're entitled to a further milestone payment upon the first commercial sale in the United States. We're also eligible for similar regulatory and commercial milestone payments based on progress in the EU.

The royalties are also healthy and based on rivipansel product sales. They begin in the low double-digits and go to the low-teens. The very meaningful milestone payments together with potential royalties from sales of the drug would allow us to accelerate the development of our novel GlycoMimetics pipeline.

I'd now like to recap the achievements of 2018. If there is one overriding theme that characterizes our focus and progress, it is execution. Execution across all fronts; clinical, preclinical and financial. Our wholly owned investigational drug for AML, uproleselan, reflects once again the commitment of our cross-functional teams in chemistry, preclinical and importantly now focusing in clinical development.

Early last year, we announced a comprehensive Phase 3 clinical program in AML. Our vision for uproleselan is to position it as a foundational therapy that could work across the AML spectrum, whether a patient has relapsed refractory disease, is newly diagnosed and fit for chemotherapy or not fit for chemotherapy. Based on the strength of the clinical data, we opted to move forward on a Company-sponsored pivotal trial in the relapse-refractory setting, which is the indication where we have the breakthrough therapy designation.

And in early November, we announced the dosing of the first patient in that Phase 3 study. That trial is now well under way with good enrollment momentum. In parallel, we announced collaborations with two consortia, one here in the US with the Alliance for Oncology, a National Cancer Institute, and one in Europe with the prestigious HOVON Group, to study uproleselan in two additional patient populations.

The NCI study is active and open for enrollment with dosing of a first patient expected in the next quarter. In December, at the annual ASH meeting in San Diego, we reported in an oral presentation final Phase 1/2 data that included extended follow-up for survival in patients in both relapsed refractory and the newly diagnosed population. This data has increased our confidence in the key efficacy endpoint to underpin our GMI and NCI sponsored registration programs.

Importantly, we also presented for the first time data on measurable residual disease or MRD, over 50% of the evaluable patients in both the relapse-refractory and newly diagnosed cohorts achieved the stringent level of disease response. We believe uproleselan selectively disrupts the relationship between leukemic cells and the bone marrow microenvironment. Therefore, we had hoped to see a higher percentage of those patients who achieved complete remission to be MRD negative. That's exactly what we saw and this finding suggests that uproleselan is indeed enhancing the depth of remission.

I'd also like to remind you of the trials correlative data on E-selectin ligand expression on leukemic blasts and leukemic stem cells. That data showed our target to be highly relevant, particularly in the relapsed/refractory population with higher levels of E-selectin ligand expression correlating with both response and survival in this cohort of patients treated with uproleselan.

In short, the data on expression combined with the data on MRD negativity further support our underlying hypothesis that E-selectin is a driver of resistance in AML. If we can disrupt binding of cancer cells to this target, we believe the result will be improved clinical outcomes. Across our GMI sponsored, NCI sponsored, and HOVON sponsored program, we expect to have over 850 patients treated in Phase 2, 3 clinical programs evaluating uproleselan in North America, Europe and Australia. If the results from each of these studies are positive, we expect that the totality of the data could support a broad label across the continuum of care in AML.

I'd now like to spend a few moments highlighting the significant progress we've made in discovery research and preclinical. The result of which is a robust pipeline of novel agents, all of which come from the specialized chemistry expertise that underlies our GlycoMimetics discovery platform here at the Company.

In December, we presented data on two compounds -- two new compounds; GMI-1687 and GMI-1757, which both have their debut in the public arena at ASH. Of particular note, our poster on GMI-1687, a highly potent antagonist of E-selectin points to the potential of GMI-1687 as an important follow-on drug candidate to uproleselan with the potential advantage of self-administration in the outpatient setting. We believe this could enable us to realize the value of an E-selectin antagonist outside of AML.

Our poster on GMI-1757, a novel dual function Galectin-3 E-selectin antagonist shared data on the first of a series of Galectin-3 antagonist, which we believe could have broad applicability in the variety of cancers and for broader conditions. We will be rolling out more preclinical data with these agents as well as other Galectin-3 antagonist in 2019. We're proud of the platform developed here and excited about the opportunities that these novel drug candidates represent.

One final comment. We were pleased in the third quarter to receive a patent issuance for uproleselan in Japan. This patent covers composition of matter as well as pharmaceutical formulations and expires in December 2032. This patent extends intellectual property coverage for uproleselan across all seven of the major markets and it solidifies an important component of our continually expanding intellectual property portfolio.

At this time, I'd like to look forward across 2019 to the milestones we anticipate. In the second quarter of this year, we expect to announce dosing of the first patient in the NCI sponsored Phase 3 trial in AML patients who are newly diagnosed and fit for chemotherapy. Later this year, we also expect to announce the initiation of the HOVON sponsored trial in patients with newly diagnosed AML who are unfit for intensive chemotherapy.

As highlighted in the beginning of this call, at the end of the second quarter, we are expecting to announce top line data from the Phase 3 pivotal trial of rivipansel and sickle cell disease. Again, we believe a positive outcome will transform the way patients with sickle cell disease are treated as well as bring other tangible and intangible benefits to our organization.

Later this year, we also expect to be in a position to announce the initiation of an exploratory clinical trial of GMI-1359 in a solid tumor indication. We've continued to make progress with this drug candidate and have decided to move into solid tumor setting in which we will be able to evaluate biomarker and PK data in patients with cancer that has metastasized to bone. We will be providing additional details about this trial later in the year, and this will be our first trial with our potent dual function antagonist in cancer patients.

Finally, before turning the call to Brian for a review of our financials, we issued a Board release yesterday in parallel with the filing of our proxy that the Chairman of our Board of Directors, Jim Barrett; and our Chief Scientific Officer, John Magnani will not be returning for reelection to the Board when their terms expire later this year.

Jim, since 2001 was a general partner at NEA. He led NEA's founding venture investment in our Company, and as a representative of GlycoMimetics' largest shareholder has chaired our Board since its founding. His decision not to run for reelection comes at a time when he's announced his retirement both from NEA and from a number of Boards. We are all deeply grateful to him for the support and guidance he has provided through the years.

John Magnani, as many of you know, co-founded GlycoMimetics with me and has been our Chief Scientific Officer since the Company's inception. He remains in that position, even with the transition from Board Director. All of the work here at GlycoMimetics is a product of a specialized chemistry platform for which John has provided exceptional creativity and leadership. His key position on our senior leadership team does not change with this announcement.

Stepping into the role of Board Chair will be Tim Pearson, a GlycoMimetics Director since 2014, and until recently, Chief Financial Officer and Executive Vice President for TESARO. This change will be effective at our annual meeting of stockholders on May 17. Tim brings broad industry experience from his senior roles at a number of leading biotech companies and we very much look forward to working with him as our chair. I might add we have an impressive Board of Directors here at GlycoMimetics and these changes are part of the natural maturation process.

Let me now turn the call over to Brian, who will review our financials with you. Brian?

Brian Hahn -- Chief Financial Officer and Senior Vice President

Thank you, Rachel. As of December 31, 2018, GlycoMimetics had cash and cash equivalents of $209.9 million compared to $123.9 million as of December 31, 2017. The Company in the first quarter of the year successfully completed a follow-on public offering netting proceeds of $128.4 million.

The Company's research and development expenses increased to $12 million for the quarter ended December 31, 2018 as compared to $6.7 million for the fourth quarter of 2017. Research and development expenses increased by $16 million to $40 million for the year ended December 31, 2018, from $24.1 million in the year ended December 31, 2017. These increases were primarily the result of higher manufacturing costs to scale up production of uproleselan in clinical supplies for the Company's Phase 3 clinical trial and for clinical trials conducted by or in collaboration with third parties. Personnel-related and stock-based compensation increased due to an increase in clinical head count.

The Company's general and administrative expenses increased to $2.9 million for the quarter ended December 31, 2018 as compared to $2.8 million for the fourth quarter of 2017. General and administrative expenses for the year ended December 31, 2018 increased to $11.4 million as compared to $9.8 million in the prior year. These increases were primarily due to an increase in legal and patent expenses as well as personnel-related costs and stock-based compensation expense. Patent expenses were higher due to an increase in the number of patent applications filed. Personal-related and stock-based compensation expenses increased due to additional headcount in 2018.

In summary, GlycoMimetics is in an excellent financial position to carry out its planned initiatives and to advance applications for its unique technology platform.

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

Rachel K. King -- Chief Executive Officer

Thank you, Brian. I hope you'll agree that we've been uniquely successful in progressing truly differentiated drug candidates from discovery well into late-stage development. Starting with rivipansel pivotal results at the end of the second quarter of this year, we now anticipate what we hope will be a series of value creating milestones, which I hope will reward patients and their shareholders for years to come.

With that, I'd like to open the call for your questions. Operator?

Questions and Answers:

Operator

Thank you. (Operator Instructions) Our first question comes from the line of Jotin Marango of Roth Capital. Your line is now open.

Jotin Marango -- Roth Capital -- Analyst

Good morning, team, and congratulations on all good progress.

Rachel K. King -- Chief Executive Officer

Hi. Good morning, Jotin.

Jotin Marango -- Roth Capital -- Analyst

Good morning. Two questions. First about with upro. I know it's certainly early, it's still early in the process, but do you have any qualitative commentary or site feedback about the enrollment of patients, such as granularity on screening or dark attitude. And importantly, whether or not this may be competing for patients with other trials in the same leukemia silos.

Rachel K. King -- Chief Executive Officer

Let me make a comment, and then I'll turn it over to Helen for some more detail. I will say we're getting enthusiastic responses from the sites. We have sites open now in the United States and Australia and we will be shortly opening in Europe. So we're enrolling widely and well. And the study has good -- has good momentum. Let me turn to Helen for comments on more granularity around the reaction from some of the investigators.

Helen Thackray -- Senior Vice President, Clinical Development and Chief Medical Officer

Yeah. So we have had, as we announced, we had our first patient enrolled in November. We have sites coming online at a good pace in the US and outside the US, as Rachel said. And multiple sites are excited about the trials. We've had good momentum with investigators and good momentum with start-up with sites. So we're very pleased with that momentum so far. And we've not had much feedback in terms of competing trials for patients. We think that this was a good space in the relapsed refractory setting. There is not very much available for patients or available to investigators and pairing uproleselan with the standard of care is a very attractive option in this clinical trial setting. So we are very optimistic with the feedback we've had so far.

Jotin Marango -- Roth Capital -- Analyst

Thank you. And second is the early pipeline. So thank you for the overview, Rachel, the data at ASH about the antithrombin effect is interesting. And I remember the data from -- I think it was ASCO where you showed white cell mobilization. How are you thinking about prioritizing the early programs at this point, because it seems like you can do a lot.

And then second, in -- some of these agents do share a common target with upro. How do you think about the possibility of just advancing your agent versus expanding the upro program, if that's already clinical?

Rachel K. King -- Chief Executive Officer

Yeah. Well, so, I think that you raise a number of important points and actually highlight some of the great opportunities that we think we have going forward. 1687 is the compound that you referenced, which is the highly potent E-selectin antagonist and, as far as the follow-on pipeline is concerned, that would be our next priority of among the compounds that are still preclinical. And I would say that that first of all offers a chance for us to take the indications in which we develop upro into a self-administered setting potentially.

For example, we know that some of the -- we were currently dosing uproleselan intravenously for patients who were hospitalized while they received their chemotherapy. But you could envision over time wanted to be able to move in AML therapy into the outpatient setting, and 1687 gives us the potential to do that because we believe it will be subcutaneously available. It also, as you indicate gives us a potential to move into some other indications. I would say our initial focus is going to be to look for other areas within the hematology, oncology space that would allow us to build on the expertise and the relationships that we've built in that area, also focusing as we have in the past on areas where we feel that the use of the drug could be potentially transformative and could also lead to streamline development as well as potentially targeted commercial opportunities. So that's the reference to the 1687.

We also have as you indicated, you briefly alluded to the fact there were other molecules, there's also the Galectin program, which is also preclinical. We introduced one of those molecules that E-selectin Galectin combination molecule at ASH, that has a broad potential range of indications and there are some very exciting indications for the Galectin-3 antagonist alone. So, we will be saying more about what we intend to do with those as we continue to develop the preclinical story. But I think, at a high level what I'd say is, you'll see focused clinical programs to build on the expertise that we have but then continued to build out the pipeline based on what really is a unique very differentiated specialized chemistry platform that we have here at the Company.

Jotin Marango -- Roth Capital -- Analyst

Thank you very much. And I look forward to the rivipansel data.

Rachel K. King -- Chief Executive Officer

Thanks, so do we.

Operator

Thank you. Our next question comes from the line of Stephen Willey of Stifel. Your line is now open.

Stephen Willey -- Stifel, Nicolaus & Company -- Analyst

Yeah. Good morning (inaudible) for taking the questions.

Rachel K. King -- Chief Executive Officer

Good morning.

Stephen Willey -- Stifel, Nicolaus & Company -- Analyst

Good morning. So maybe just a couple of sickle cell questions. Just curious as to -- how you see the potential approval of crizanlizumab perhaps impacting the addressable market opportunity. And I guess, second to that, if a patient was taking that drug prophylactically and how to break through, would there be hesitancy at all within the acute care setting to try to address the VOC with an asset that has somewhat similar mechanism of action?

Rachel K. King -- Chief Executive Officer

Yeah. So let me take the second question first. We actually think that there would not be hesitancy to use it and no reason not to use it. As you remember, may remember crizanlizumab targets P-selectin. Whereas, rivipansel is a Pan-selectin antagonist with specifically greater on activity against E-selectin. And there's a lot of data in the literature that actually speaks to the dominance of E-selectin in the context of a vaso-occlusive crisis. So we think that as a breakthrough therapy, or as a breakthrough crisis would occur in the patient on crizanlizumab that there would be every reason actually to give rivipansel to that patient in the acute care setting.

As far as the addressable market is concerned, I do believe that with all the drugs that are in development now there will be -- there'll be a number of different therapies potentially available for treatment of sickle cell disease, which is good news for patients, but I think these are going to be complementary. You may note that the data from crizanlizumab is very similar to the data that was generated a number of years ago with hydroxyurea. And hydroxyurea has been on the market for about 15 years, it had an excellent clinical trial output showing that it also significantly reduced the hospitalizations for crisis, but had very little impact -- has had very little impact over the years on the actual hospitalizations for crisis.

So I think fundamentally, although treatment of the disease will improve, I don't think vaso-occlusive crisis is going away, and I'll remind you that rivipansel is the only drug in late stage development for treatment of crisis. So we think that where there currently is a lot of competition right now in the prophylaxis setting, crizanlizumab, the global blood product hydroxyurea, et cetera, there is no other late-stage development, no other drug in late-stage development currently for the treatment of crisis, so we think that rivipansel can really own that portion of the market.

Stephen Willey -- Stifel, Nicolaus & Company -- Analyst

Got it. And then, maybe just a clarification question -- the dual-function antagonist. Did you say that goes into the clinic later this year?

Rachel K. King -- Chief Executive Officer

No. The dual-function antagonist which is preclinical -- I'm sorry, we have two dual-function antagonist. Let me clarify, there's -- there's 1359, which has already been in the clinic that's completed the Phase 1 trial and that's the drug that we intend to take into a solid tumor trial later this year. There is a second dual-function antagonist which targets E-selectin and the Galectin, which is still at the preclinical stage. But 1359 is already in the clinic, and we intend to continue with the clinical development of that program with the initiation of a solid tumor trial later this year.

Stephen Willey -- Stifel, Nicolaus & Company -- Analyst

Understood. And then, just lastly, I know that there was some prior mention of potentially seeing some myeloma data this year. I guess, is that still -- is that still part of the plan and, is the objective again really just to show changes in N-protein? Thank you.

Rachel K. King -- Chief Executive Officer

So, we have deprioritized the myeloma program, largely because of the changes in the myeloma landscape particularly with the approval of Daratumumab and the way that that treatment landscape has continued to develop with more options for patients. So we actually have decided to discontinue enrollment in the myeloma trial. We will continue to gather data on those patients and we'll see if there is anything that we want to report out about that, but we've decided that we see a -- feel there are greater opportunities for uproleselan in AML and potentially in other areas, which we are continuing to explore. But we don't think that -- we think that given the changing treatment landscape in the myeloma field, we don't see as good an opportunity for uproleselan there as we think we could develop in other areas.

Stephen Willey -- Stifel, Nicolaus & Company -- Analyst

Got it. Thanks for taking my questions.

Operator

Thank you. Our next question comes from line of Peter Lawson of SunTrust Robinson. Your line is now open.

Peter Lawson -- SunTrust Robinson Humphrey, Inc -- Analyst

Thanks for taking my questions. Rachel, just following up on the 1359 moving into solid tumors. Which tumors are you thinking?

Rachel K. King -- Chief Executive Officer

Yes. So we haven't -- we haven't announced specifically what we'll be doing in that trial, we do expect to say that later this year. What I can say though is that we've decided to move into a solid tumor setting in which the tumor has metastasized to the bone, and that's because the preclinical data that we've generated with that molecule demonstrates particular effect in that setting, where we've shown that both CXCR4 and E-selectin are involved in both trafficking and maintenance of metastatic sites within the bone. So, what we've designed is a study that's going to be looking at both PK in the cancer patients as well as some biomarkers that are going to allow us to begin to explore the potential use of 1359 in the setting of solid tumor metastasized to bone. So we'll be saying a bit more about that later in the year, but that's general contours of what we expect to do with that trial.

Peter Lawson -- SunTrust Robinson Humphrey, Inc -- Analyst

Got it. Thank you. And then, just as we think about follow-on molecules, for rivipansel, is that completely under the control of Pfizer. Are you thinking about follow-on molecules there and whether you need to move beyond IV into subcu and -- kind of what else we should be expecting on that? Thank you.

Rachel K. King -- Chief Executive Officer

Yes. So the license with Pfizer is for rivipansel and for drugs which are very likely rivipansel, so centrally rivipansel and its family of molecules. We are not continuing work with other rivipansel like molecules here at GlycoMimetics, however, Pfizer could take rivipansel into other indications if they would choose to do that.

Here at GlycoMimetics, what we are doing is, we are developing our own suite of molecules, which are more focused and more potent than rivipansel. They're more -- they're newer molecules and have the benefit of what we've learned through the early days of the discovery and development of rivipansel, but they are being taken forward in different indications from sickle cell here at GlycoMimetics. So we're not specifically working on follow-ons to rivipansel here.

Peter Lawson -- SunTrust Robinson Humphrey, Inc -- Analyst

Perfect. Thank you. And then, just as we think about the number of trials that are enrolling for upro, are there any that are enrolling better or worse than expected, just anything can you talk about the -- the major innings (ph) of patients?

Rachel K. King -- Chief Executive Officer

And so, I'll now turn that over to Helen.

Helen Thackray -- Senior Vice President, Clinical Development and Chief Medical Officer

I think the question is about sites enrolling with uproleselan in Phase 3. And I think, at this point our comment is simply that enrollment has started, enrollment is proceeding well, demonstrating good early momentum and we have also the NCI program is under way. NCI has listed their trial uproleselan also on clinicaltrials.gov. And so we're looking forward to seeing that trial and progress with that trial over the course of the coming time.

Peter Lawson -- SunTrust Robinson Humphrey, Inc -- Analyst

Great. Okay. Thank you so much.

Helen Thackray -- Senior Vice President, Clinical Development and Chief Medical Officer

Sure. Thank you.

Operator

Thank you. (Operator Instructions) Our next question comes from the line of Biren Amin of Jefferies. Your line is now open.

Biren Amin -- Jefferies -- Analyst

Yeah. Hi, guys. Thanks for taking my question.

Rachel K. King -- Chief Executive Officer

Good morning.

Biren Amin -- Jefferies -- Analyst

So, Rachel, just couple of questions on rivipansel Phase 3 design as it relates to the Phase 2 data that you guys generated. So on the Phase 3, I think you're enrolling pediatric patients along with adults and given the pediatric patients were not enrolled in the Phase 2, how do you think it would impact the Phase 3? And then also, I think adult patients in the Phase 3 are receiving a fixed-dose, which is equivalent to the lower weight-based dose used in the Phase 2. So, a question on what led to the choice of that fixed dose in the adults in the Phase 3 and how you think that would impact it?

And then lastly, I guess, what are your thoughts on site to site variability and how that would impact Phase III data?

Rachel K. King -- Chief Executive Officer

I think I'll turn those questions over to Helen. If you can answer this in more detail.

Helen Thackray -- Senior Vice President, Clinical Development and Chief Medical Officer

Yeah, sure. So with regards to pediatric patients, you're correct, this Pfizer trial is enrolling patients down to age six. In our Phase 2 trial we enrolled patients down to age 12, the adolescent and adult populations. Pfizer has included that adolescent and adult population and also I think quite appropriately extended the trial down to the younger group. As you know, sickle cell disease vaso-occlusive crisis occurs across the age ranges, the pediatrics and adults. So that seems to be very appropriate for the trial and we'll allow for a data set that informs the whole population. And so we think that's quite -- that's quite appropriate.

In terms of the dosing, there's the fixed dose. The transition from weight-based dosing in the Phase 2 to a fixed-dose in the Phase 3, it's a standard transition. And adults dose is based on the exposure levels demonstrated in the Phase 2. Those exposure levels are looking at exposure on a population PK-based analysis, and so, the fixed dose is simply selected to establish the exposure on a fixed-dose level for the adult population. We have taken this approach also with uproleselan where in our Phase 1/2, we had weight-based dosing. In the Phase 3, we've been able to select a fixed-dose built on population PK data. So that's a pretty standard approach. And I think we're quite comfortable with the way Pfizer has approach that.

And then your third question, I think it was on this site to site variability. It is expected that there is variability at a patient level as well as at a site level in any clinical trial and that's the reason for expanding the number of sites and number of patients in our Phase 3 program. So that's fairly standard. Pfizer has a large trial with a good distribution of sites that represents the standard of care. They're also very similar to the nature of sites that were included in the Phase 2, and so we expected the standard of care treatment as well as the outcomes that will be seen should be representatives, certainly as what we saw in the Phase 2 as well.

It's also a randomized trial, which allows you to control for any potential sources of variability. The Pfizer trial is also sized substantially larger than our Phase 2. And as you'll remember, there were some outcomes in our Phase 2 trial that either achieved statistical significance or trended toward significance even in that small trial. So we think that the Pfizer trial is well designed and well-sized to address any variability that might be seen.

Biren Amin -- Jefferies -- Analyst

Okay. And then, I just have a one follow-up on upro. Given the Phase 2 data at ASH, it seems to suggest better outcomes in patients that have favorable and intermediate risk compared to adverse risk. And just, I think, it is a trend that you saw on both the refractory trial and the newly diagnosed trial. So my question is, would you attempt to more heavily recruit favorable and intermediate risk patients in the Phase 3 refractory study to have better odds of success?

Rachel K. King -- Chief Executive Officer

I'll let Helen take that one also.

Helen Thackray -- Senior Vice President, Clinical Development and Chief Medical Officer

Yeah. Actually, we were very pleased with the data that we saw in adverse outcomes in both the relapsed/refractory group in our trial and in the newly diagnosed group. There were substantial levels of patients with adverse cytogenetics enrolled as well as high risk disease. And they did quite well, especially compared to what you'd expect for these populations. These populations have a -- have a poor remission rate and poor survival. We had patients in those groups in that adverse risk group achieve remission and have long survival outcomes with reduction and -- sorry, with negativity in terms of measurable residual disease. And so we believe that that has demonstrated a deep durable remission even in patients with adverse risk at rates higher than we would expect from historical controls.

We also know from the work that Pam Becker has done and that John Magnani has done, that the adverse risk in those patients who are most likely to relapse early and have refractory disease have a higher proportionately E-selectin ligand expressed on those leukemic blasts, illustrating that the cells that are most likely to be the cause of relapsed or the cause of resistant disease are in fact the cells of uproleselan that's best designed to target. And so our data in the average population was very, very much in line with what we expected to see and hoped to see in terms of targeting those patients with the highest risk and also the highest proportion of the E-selectin ligand on their blasts and seeing negativity in terms of measurable residual disease and durable responses.

Biren Amin -- Jefferies -- Analyst

Great. Thank you.

Rachel K. King -- Chief Executive Officer

Thanks, Biren.

Operator

Thank you. And I'm showing no further questions at this time. I would now like to turn the call over to Ms. Rachel King for closing remarks.

Rachel K. King -- Chief Executive Officer

Great. Thank you, operator, and thank you everyone for your questions and for taking the time to join the call. In 2018, GlycoMimetics delivered achievements on all fronts, in clinical development, in preclinical research discovery and in finance. We put into place a comprehensive Phase 3 clinical program for uproleselan that if successful could position this -- investigational drug as a foundational therapy across the spectrum of AML. This is an accomplishment that reflects the enthusiasm worldwide of clinicians who've seen our data and its impact to date on patient outcomes as presented at top oncology congresses.

In discovery preclinical research, our specialized chemistry expertise produced new potential drug candidates to expand our pipeline opportunities into indications that go beyond sickle cell disease and hematologic cancers. Looking forward to 2019, we're in a strong financial position to pursue clinical and preclinical opportunities ahead. Importantly, we look to the rivipansel top line readout expected late in the second quarter that could represent the first commercial success of our pipeline and the key financial resource going forward. Thank you very much.

Operator

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

Duration: 39 minutes

Call participants:

Shari Annes -- Investor Relations

Rachel K. King -- Chief Executive Officer

Brian Hahn -- Chief Financial Officer and Senior Vice President

Jotin Marango -- Roth Capital -- Analyst

Helen Thackray -- Senior Vice President, Clinical Development and Chief Medical Officer

Stephen Willey -- Stifel, Nicolaus & Company -- Analyst

Peter Lawson -- SunTrust Robinson Humphrey, Inc -- Analyst

Biren Amin -- Jefferies -- Analyst

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