Greetings, welcome to the X4 Pharmaceuticals conference call to discuss the positive results of its phase III clinical trial in WHIM syndrome. At this time, all participants are in a listen-only mode. A question and answer session will follow the formal presentation. As a reminder, this conference call is being recorded. It is now my pleasure to introduce your host, Dan Ferry from LifeSci Advisors. Please begin.
Thank you, operator. Good afternoon, everyone. As a reminder, on today's call, the company will be making forward-looking statements regarding regulatory and product development plans. These statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted. A description of these risks can be found in X4's most recent filings with the SEC, including the third quarter 2022 10-Q, which was filed on November third. Presenting on today's call will be X4's President and Chief Executive Officer, Dr. Paula Ragan, and the company's Interim Chief Medical Officer, Dr. Murray Stewart. Following prepared remarks, we will open the call to your questions and we'll be joined by Chief Commercial Officer, Mark Baldry, Chief Operating Officer, Mary DiBiase, Chief Financial Officer, Adam Mostafa, and Chief Scientific Officer, Art Taveras. I'd now like to turn the call over to Dr. Paula Ragan. Paula?
Thanks, Dan. Thank you everyone for joining us on the call today. Needless to say, we are thrilled to start this call on a resoundingly positive note. mavorixafor has hit its primary endpoint in our global 4WHIM registration trial. As you may recall, the primary endpoint of the trial is a metric called time above threshold for absolute neutrophil count, or TAT-ANC. The TAT-ANC is a metric to assess the correction of neutropenia above a clinically relevant threshold of 500 cells per microliter, the definition of severe neutropenia. The mavorixafor treatment arm had a TAT-ANC of just over 15 hours, which was clinically and statistically significantly better than the placebo arm's TAT-ANC of 2.75 hours with a P value of less than 0.0001.
We believe the strong statistical significance demonstrated in this trial speaks to the robust design and the ability of mavorixafor to correct neutropenia in people with WHIM syndrome. We are extremely pleased and excited to share more details on the data in the coming slides. First, let's briefly review background about the patients we aim to serve and the trial design. WHIM syndrome is a combined immunodeficiency. By definition, combined means that multiple parts of the immune system are dysfunctional. More specifically, in the case of WHIM, important manifestations of the disease include low blood levels of absolute neutrophil counts, or ANC, and absolute lymphocyte counts, or ALC. These low levels of ANC and ALC, respectively known as neutropenia and lymphopenia, are the root cause of a broader clinical sequelae of the disease.
By correcting neutropenia and lymphopenia, we believe the health risks associated with WHIM syndrome diagnosis, which include increased risk of serious and sometimes life-threatening infections and certain HPV-associated cancers, can be reduced. There are no targeted therapies to treat the combined neutropenia and lymphopenia that are the drivers of WHIM syndrome. We are very proud to have championed the study of mavorixafor, which has been shown in preclinical research to target the root cause of the combined neutropenia and lymphopenia. We are very excited about the potential impact that mavorixafor could have on people with WHIM syndrome. We believe mavorixafor is a good candidate to target WHIM and other neutropenias because of the mechanism of the diseases and mavorixafor's mechanism of action.
CXCR4 is a chemokine receptor that plays a key role in the maturation and egress of a number of white blood cells, including neutrophils and various lymphocyte lineages. The human bone marrow is the organ responsible for de-producing and maintaining a reserve of these white blood cells, which then ultimately exit from the bone marrow and move into circulation. Down-regulation of CXCR4 leads to maturation and egress of neutrophils and other white blood cells, enabling these cells to perform immune surveillance and establish long-term immunity. As one can appreciate, immune surveillance and immune system memory are critical components to enable the ability to fight infections. As you know, mavorixafor is an oral once-daily investigational therapy that has been shown to antagonize the CXCR4 receptor and support the maturation and egress of immune cells from the bone marrow.
As a company, we are committed to exploring the potential benefit that mavorixafor may have on immune surveillance. Recently, we reported a successful phase I-B study in which mavorixafor improved neutropenia across various chronic neutropenic disorders. Today, we focus on our years of effort studying mavorixafor's broader potential to impact neutropenia and lymphopenia in the specific combined immunodeficiency disorder of WHIM syndrome. With that, I'll turn it over to Dr. Murray Stewart, X4's Interim Chief Medical Officer, to review our trial design and results. Murray?
Thank you, Paula.
We designed our 4WHIM global registration trial to assess the potential of mavorixafor to impact neutropenia and lymphopenia, two of the hallmarks of WHIM syndrome, as well as other clinical metrics of this chronic syndrome. Today, we will share the top-line data from the 4WHIM trial, which focus on the potential of impact of treatment on the neutropenia and lymphopenia components of the disease, while in parallel assessing the safety profile. Additional assessments from the totality of the clinical and disease metrics are ongoing and will be presented at a future point. The 4WHIM trial was a randomized, placebo-controlled, double-blind study. The study's inclusion criteria at screening required that all patients enrolled had a diagnosis of WHIM syndrome and a confirmed absolute neutrophil count, or ANC, less than or equal to 400 cells per microliter.
Remember that less than 500 cells per microliter is the definition of severe neutropenia. Participants aged 12 and above were eligible to enroll. Participants were assessed for the primary endpoint of TAT ANC at weeks 13, 26, 39, and 52, and the mean TAT ANC was calculated for each patient over the four time points spanning the 52-week trial. Similar assessments at the same time intervals were completed for the calculation of the key secondary endpoint of mean TAT -ALC to assess mavorixafor's effectiveness against lymphopenia, which is defined as 1,000 cells per microliter. At the end of the placebo-controlled portion of the study, all participants were eligible to roll over into the open-label extension, or OLE. Notably, more than 90% of eligible participants elected to continue on and receive mavorixafor treatment in the OLE. We're continuing to collect data from this ongoing portion of the trial.
We also have a robust series of metrics to assess the safety and tolerability of mavorixafor in the participants in this phase III trial. The demographics and screening metrics of the 31 subjects enrolled are shown here, broken down by treatment arm. The study included both adults and adolescents, divided roughly evenly between the two groups. 45% of the population had previous immunoglobulin use that was maintained during the trial. As you can see, there is good balance between the mavorixafor and the placebo arms of the study. Importantly, the data values at screening of the severe neutropenia and lymphopenia experienced by these patients are shown here.
Across both arms, neutrophil screening levels, as measured by absolute neutrophil count, or ANC, were below 200 cells per microliter, which is well below the clinical threshold of 500 for severe neutropenia, indicating the potential for a high-risk group of severe infections in these patients. The data values at screening on lymphopenia show that the median values were 420 for the mavorixafor group and 520 for the placebo group, where normal is considered above a value of 1,000 cells per microliter. Here we present the box plots of the mean TAT ANC and hours of mavorixafor arm in blue versus the placebo arm in red of the 4WHIM trial. Treatment with mavorixafor yielded a mean TAT ANC of 15 hours over the 24-hour assessment periods.
Put another way, during nearly two-thirds of the assessment period, neutrophil counts in the blood achieved levels above the 500 cell threshold for severe neutropenia, demonstrating a meaningful clinical improvement in severe neutropenia of these study participants treated with mavorixafor. In contrast, you'll note the placebo arm TAT ANC achieved only 2.75 hours, demonstrating that these patients likely continued to have severe neutropenia for a vast majority of their time in the study. The P value comparing the two arms was 0.0001 in favor of those dosed with mavorixafor. Additionally, there was a 5.5-fold improvement in TAT ANC with treatment compared to placebo, which is both clinically and statistically significant. These data are consistent with what we saw in the phase II WHIM trial, which showed reductions in infection rates and wart burden .
We're hopeful that these benefits will be demonstrated in this trial as well. These data analysis are ongoing. Here we share data that demonstrate the time course of changes in the TAT-ANC between the mavorixafor arm and the placebo arm over the assessment spanning the 52-week blinded portion of the study. Note that the X-axis shows the time course and the Y-axis time in hours. As you can see, the mavorixafor-treated participants experienced increased TAT-ANC at the 13-week mark that was maintained over the 52 weeks compared to either baseline or placebo TAT-ANC at week 0, demonstrating a durability of effect. This demonstration of consistent and durable TAT-ANC measurements suggests the bone marrow had an ability to draw upon its reserve and increase circulating neutrophils over the 52-week study period.
This reduction in neutropenia, seen as early as 13 weeks in WHIM patients, will be important as we review the data in the CN trial of chronic mavorixafor dosing at a similar time point. Let's turn to our first key secondary endpoint, the assessment of lymphocyte counts as opposed to neutrophils. As we mentioned, given the combined immune deficiency nature of WHIM syndrome, where low lymphocyte counts are also a challenge, the measurement of time above threshold for absolute lymphocyte counts, or TAT-ALC, is an important secondary endpoint in the 4WHIM trial. Here we present the mean TAT-ALC of the mavorixafor arm versus the placebo arm of the trial. Treatment with mavorixafor yielded a mean TAT-ALC of approximately 15.8 hours over the 24-hour assessment periods.
Therefore, for about two-thirds of the assessment period, lymphocyte counts in the blood achieved levels above the 1,000-cell threshold defining severe lymphopenia, demonstrating a clinically meaningful improvement from the severe lymphopenia baseline of these patients. In contrast, the placebo arm mean TAT-ALC was 4.5 hours, demonstrating that these participants continued to have lymphopenia for the vast majority of their days. The P value comparing the two arms was 0.0001 in favor of those dosed with mavorixafor. Additionally, there was a 3.5-fold improvement in TAT-ALC compared with placebo, which is both clinically and statistically significant. Here we share the data that demonstrate the time course of changes in TAT-ALC between the mavorixafor arm and the placebo arm over the assessment spanning the 52-week blinded portion of the study.
Similar to what was shown with multiple changes at week 13, the mavorixafor-treated participants saw an increased TAT-ALC that was maintained over the 52 weeks, demonstrating durability of effect. Again, this TAT-ALC measurement suggests that bone marrow had an ability to draw upon its reserve and increase circulating lymphocytes over the 52-week study period. Now to safety. The safety assessed over the 52-week study is summarized here. First and foremost, there was no treatment-related serious adverse events or SAEs in this study. There were seven participants who had SAEs, five in the treatment group and two in the placebo group. The SAEs included mostly infection, thrombocytopenia, and one case of glioma. Importantly, as a reminder, none of these were deemed to be treatment related, there was no treatment limiting toxicity, no patients withdrew from the double-blind 52-week study due to safety or tolerability issues.
As you can imagine, we're very pleased with these data on mavorixafor in the WHIM population, in this trial with severe chronic neutropenia lymphopenia. Importantly, the primary endpoint of TAT-ANC was met, demonstrating a clinically meaningful correction of severe chronic neutropenia. This result was maintained over the 52-week study, demonstrating durability of the treatment with a P value of less than 0.0001. Similarly, the first key secondary endpoint was met, demonstrating clinically meaningful correction of chronic lymphopenia. This effect was also maintained over the 52-week study, demonstrating durability of the treatment with a P value of less than 0.0001. Mavorixafor was generally well tolerated, with no treatment-related serious adverse events.
With our positive top line data announced now behind us, our immediate next steps are to receive and begin assessing additional phase III data, including evaluation of the large number of secondary and exploratory endpoints in the randomized portion of the trial. Additionally, with the positive top line data, we're moving forward with great enthusiasm to engage with the FDA and discuss some plans for the NDA submission, which we continue to target for early in the second half of 2023. We also look forward to publishing additional results of the 4WHIM trial at upcoming medical conferences, possibly in the first half of 2023. In addition to our NDA submission work, we will continue to ramp up our activities in preparation for possible approval in the U.S. and launch soon thereafter in the first half of 2024. With that, I'll turn back to Pa ula.
Thank you, Murray. Before putting these milestones into greater corporate context, I'd like to take just a moment to pause here and think about all we've accomplished and to acknowledge the many people who deserve our thanks for helping us achieve this important outcome. The 4WHIM trial was successfully completed only because of the committed study participants with WHIM syndrome and the dedicated healthcare professionals that spanned 12 countries across U.S., European, and Asian continents, all during a global pandemic. Additionally, I'd like to thank the X4 employees who have and continue to work tirelessly to advance mavorixafor to support the patients that we aim to serve. We really have a great group of people here at this company, and it was not easy, but we were successful in getting to this exciting moment because of all of you who support our mission.
Thank you very much from all of us at X4. Here I'd like to put our phase III WHIM data into broader perspective, not only as they relate to mavorixafor's potential to support thos P e with WHIM syndrome, also the potential of this investigational therapy to help those in the broader CN disorders community. Yes, we robustly hit the primary endpoint and the first key secondary endpoint. Yes, we demonstrated a well-tolerated safety profile throughout a year of daily dosing. Importantly, today's results further speak to the durability of mavorixafor's treatment effect on blood levels of neutrophils. As we've seen in the data presented by Murray just now, the changes in neutrophil counts, as measured by TAT-ANC, were essentially at steady-state levels by 13 weeks and remained relatively consistent over the 52 weeks of the study.
With these results, we have an even deeper confidence to press ahead with further development of mavorixafor in broader CN disorder populations. Based on prior FDA interactions, we have confidence that a placebo-controlled phase III trial in chronic neutropenia, similar to our WHIM phase III trial, is a likely path forward to support registration. Now brings us to our key upcoming corporate milestones. X4 continues to build great momentum with our September announcement of mavorixafor's as 100% response rate across a range of CN disorders, and today's announcement of the positive 4WHIM phase III trial.
With these data now in hand, we believe we are well-positioned for additional expected 2023 milestones, which are expected to include, in the first half of 2023, additional data in CN, including durability of response and hopefully regulatory clarity on the registration path and phase III trial design for mavorixafor and chronic neutropenic disorders. In the second half of 2023, we aim to submit our NDA for mavorixafor in WHIM syndrome, which could lead us to our potential first approval for mavorixafor and receipt of a priority review voucher in the first half of 2024. Building on our demonstrated success in WHIM, our hope is to rapidly advance the additional development of mavorixafor in CN disorders to potentially offer a new treatment to the approximately 50,000 people in the U.S. living with this chronic and challenging condition.
Our future vision is to deliver an effective and safe oral treatment as a new option for patients and potentially become the new standard of care. We'll now open up the call for questions. Operator?
Thank you. We will now be conducting a question and answer session. If you would like to ask a question, please press star one on your telephone keypad. The confirmation tone will indicate your line is in the question queue. You may press star two if you'd like to remove your question from the queue. For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys. One moment, please, while we poll for questions. Thank you. Our first question is from Stephen Willey with Stifel. Please proceed with your question.
Hey, good afternoon and congratulations on the data. It looks like there were some placebo patients that were given mavorixafor in advance of some of their week 52 measurements. Again, I know it doesn't impact statistical significance, but was just curious as to how long in advance those patients had received mavorixafor before those week 52 measurements were made.
Sure. Thanks, Steve. Appreciate the attention to that, and I'll pass over to Murray to give the details.
This is actually the same day. What happens is patients came in for the final 52-week visit at the same time starting their open label extension. Ideally, what should have happened is they stayed on their medication, then had their assessments, blood assessments over that time, and then started their active open label. A couple of physicians gave their patients the active therapy on that day just before the assessment. It actually shows that within the 24-hour assessment, you actually saw an increase in blood count within that period. It actually shows that the response of ANC occurred within that 24-hour period. No one got the drug before in the placebo group. It was just on that final visit.
Okay, that's helpful. With respect to the pre-NDA meeting, I know that you guys already have some guidance in hand with respect to what a label might look like. You have breakthrough. What are you hoping to clarify? What are some of the key questions that you hope to address with FDA in response to this meeting?
Yeah, I'll start, then of course, Murray and others can chime in. Steve, of course, we wanna review the totality of the data. As we look at the robust, hopefully, assessments across not only the primary but the key secondaries, we hope to be able to include as appropriate some of that information in the label. That hopefully the more robust data sets will be incorporated to help sort of create a robust label as we go out and potentially market mavorixafor. I'll ask Murray to chime in further.
Yeah. No, I'm predicting a pretty straightforward pre-NDA meeting. We've got non-clinical data, we've got our CMC package, and the clinical data will be part of that. As Paula said, it's a matter of taking them through the efficacy and the safety and seeing if they have any concerns. I think we had a good SAP agreement on what the primary endpoint was, and I'm looking forward to the meeting.
Okay. And then, just lastly, I guess how should we be thinking about, the communication from you guys with respect to some of these secondary and exploratory endpoints? Are these something that you're going to try to preserve for a medical conference presentation, or is this something that we should hear about, maybe just from you guys independently in the form of a press release or whatever?
Our intention is to communicate via a medical conference. Obviously, it's important to preserve publication rights. We haven't updated our guidance that we're likely to put that out the first half of 2023. We're looking forward to. We still have a lot of analyses to go. Anyone can see that on ClinicalTrials.gov. We have some heavy lifting ahead of us, but we'll look forward to sharing that data in the first half of 2023.
All right. Thanks for taking the questions and, congrats again.
Thanks, Steve.
Thank you. Our next question is from Mayank Mamtani with B. Riley Securities. Please proceed with your question.
Good afternoon, team. Congrats on the data, and thanks for taking our question. On the ANC data, you know, just was comparing that to what we had seen in phase II. It seems like it got better on relative terms. Could you just maybe explain why that could be, if there were any demographics differences? And I know you had pediatrics included in this study. Just, could you lay out what were maybe the differences between phase II and phase III populations?
Sure. Thanks, Mayank. Murray will address that.
Yeah. I think there's three main things. One is the phase II with just a small number of patients, eight, without a control group. Two, there were different doses used in the phase II, so not everyone got up to the 400. For me, the phase III data shows the robustness of the 400 mg dose. The fact that we had a placebo group really showing the delta compared to placebo, and actually robust numbers with a durable effect over time. I think it's the duration, the dose, and durability gives me a lot of confidence in the phase III results.
Okay. Got it. Then a couple of quick process questions. The sample size, you know, at the different time points, evaluated for both ANC and ALC is a little bit more variable for the treatment arm versus the placebo arm. Can you explain why that was? Also as you look to follow the open label data here now that, you know, more subjects will be receiving the placebo patient will be receiving drug arm, how would you look to disclose that, you know, additional follow-up from the OLE? Those are two process questions. Then I have one final follow-up.
Yes. I think I heard two questions, which I'll ask Murray to answer. One is just the variability, that you've observed between the treatment and placebo arms. The second was how we'll disclose the open label extension data as it matures. I'll turn it over to Murray, and I can also chime in if I can.
I think variability is accounted for by having enough patients. The question is the effect real or not? You want to recruit enough patients in the phase III to show that the effect of the treatment isn't due to variability. I think we know it's not due to variability because of the statistical value of P 0.0001. I think the number of subjects, the clear difference, explains the true treatment effect. There is some variability, we accounted for that by having enough subjects in a longer study. Regarding the open label extension, that study continues. We will do data cuts next year, we won't have immediate update on the open label extension, given it's an active program at present.
Got it. Just a final question on the SAEs. I know they're mostly deemed as not treatment related, but just if you could opine on the five versus two and, if, you know, infections were any part of this, would be helpful, qualitative color if you could provide.
Sure, yes. You're right, Mayank. None of them were related to treatment. Murray can give a little bit more color on the profile.
Yeah. The two adverse event related to the fact these infections result in hospitalization. There were four infections, two in the mavorixafor group, two in the placebo. The infections all resolved with continuing treatment. There was one case of someone who was COVID positive during the pandemic. They were immunosuppressed, they were admitted to hospital for prophylactic antibiotics and monoclonal antibodies, understandably so, but that was counted in SAE because hospitalization. There was one individual who had low platelets, they had a past history of low platelets when they were two years old. They had GCSF and low platelets. They had low platelets before they came into the study. Six months into the study, they had nosebleeds and were noted to have a low platelet. They were observed. The platelet count resolved on continuing treatment.
If it had been cut due to mavorixafor, I would expected the platelets to be low. The fact it recovered and was there before means, again, not related. The last SAE was an unfortunate individual who had a glioma. They had symptoms before the start of therapy, and it was actually picked up due to one of the eye assessments. They continued on treatment in the double-blind portion of the study. Not drug related. Mavorixafor doesn't cross the blood-brain barrier, the symptoms clearly were before start of therapy.
Yeah, Mike.
Got it. Go ahead.
Oh, sorry. Just to share some color too as well. All these assessments are done by independent physicians of X4 that have been monitoring these patients for years over the study. Again, the non-drug related effects are due to these physicians and experts reviewing the data, so we feel very comfortable with where we're at today.
We had an IDMC, an independent data monitoring committee, as well as individual physicians.
Got it. If I may, squeeze one last question. You know, given the totality of data you have and more data you are generating and also, the breakthrough and fast track, is a rolling submission possible where, you know, you start with some of this initial package and kind of complete it over the course of next year, kind of an option that you would look at when you go into the pre-NDA meeting? We are seeing that, you know, with a lot of other companies with, you know, indications of unmet need and breakthrough potential, you know, that this, the data has h
ere.
Thanks, Mike. I mean, we are taking advantage of the various ways of, you know, accelerating and moving us forward through our NDA discussions. I think we're very comfortable to still maintain the NDA filing in the early part of the second half of next year. Just given even with the rolling submission opportunities, there's still a number of analyses, and data gathering, as we head towards that date. We're on tra ck for that early, the early part of the second half of next year.
Thank you. Our next question is from Marc Frahm with Cowen. Please proceed with your question.
Hi. Thanks for taking my questions and congrats on the data today. maybe just one, I realize you're kind of early in the process of analyzing some of these secondary endpoints, but is there anything you can tell us on kind of early trends on things like infections or wart burden or some of the other clinical impacts of disease? just 'cause, you know, there's some question out there of, you know, are the neutrophils that are being released by mavorixafor, you know, truly fully functional.
Thanks for the question, Mark. We do not have any data in-house yet around any of these additional clinical secondaries and exploratory endpoints, where it's such a huge bolus of data with a lot more complications around these central adjudication committees and central reviews of some of the images of the warts. We are looking forward to accelerating, you know, analyzing that data as we approach the end of the year and early next year, and we'll provide a data analysis update during the first half of 2023.
Okay, thanks. That's fair. Just following up on one of the earlier questions on just kind of some of the numbers at different time points, you know, varying. Just can you remind us kind of how those data gaps that naturally happen in the trial are being filled? Just how are they treated in the primary analysis? Have you had a chance to do any kind of sensitivity analyses of the different ways that missing data can be treated?
The prime analysis was an intention to treat. Addressing one of the earlier questions, it didn't matter what happened, whether they were on placebo, the FDA wanted to look at the ITT. The ITT was highly significant. You'll notice that some people didn't always get blood drawn at that visit. The ANC curve of the 24 hours required people to obviously have blood for that 24 hours. Occasionally, either people didn't do the visit or they had a missing value. There's certain determinants where you put in a mixed model, so you can predict what's happening. You had to have enough events. We had certainly enough patients who had enough samples to make the data robust, and modeling allows for some adjustment.
Okay. Thank you.
Thank you. Our next question is from Leland Gershell with Oppenheimer. Please proceed with your question.
Hey, guys. Great to see the positive data. Thanks for taking my questions. Just a couple for me. First, given the nature of the enrollment, you probably have a few patients who are quite long-lasting in the extension. I'm wondering if, Paula, if you can comment on how long your longest patient has been on MAV in the extension phase. Also wanted to ask with respect to WHIM syndrome versus chronic neutropenia. Can you remind us, are those reviewed by the same division at FDA or are they two different divisions? Thank you.
Sure. I'll take the second question first. The, the same group at the FDA is the one that we're discussing, both the WHIMs, WHIM program as well as chronic neutropenia. In terms of the longest patient in our OLE, I think it's about two years now into the OLE, but I'm looking at the our team here to confirm that.
Yeah. I think that's about right. I think we've not got any data on the open label extension. For me, what was really exciting is that 90% of the people went into the open label extension, and they're doing well. We'll need to have a more detailed update when we do analysis.
All right. Terrific. Thank you.
Thank you. Our next question will come from RK with H.C. Wainwright & Co. Please proceed with your question.
Thank you. Good evening, Paula and team. Congratulations. Most of my questions have been asked. I was wondering about, I believe there were about 45% of the patients on the study had IgG on board, during the study period. Just trying to figure out, you know, what effect does that have on the totality of the data?
Sure. I'll start and then bring Murray in. Certainly when we designed the study, we anticipated that some patients would be on chronic immunoglobulins, given the low IG levels that are hallmark of WHIM syndrome. It's never been tested in WHIM to actually demonstrate any effect, but of course it's part of their standard of care. That was the intent of us to ensure that we randomized the study to make sure that it was equally balanced. Importantly, what we've heard, at least from our clinical experts, is these patients are still quite sick. They still get infections. I mean, certainly IVIG or IG doesn't impact wart burden. We believe our study design will certainly be able to appreciate and understand the impact of mavorixafor on their clinical profile. Murray?
Yeah. I mean, the key for me is the groups were balanced at baseline, so we will be able to do sub-analysis looking at the effect, which we will do. You know, to Paula's point, I think the key thing there is the immunoglobulins are not the complete answer. We hopefully will see an effect in people without IVIG and on top of IVIG, but that data will come in the subsequent analysis.
Perfect. Thank you very much. Thanks for taking my question.
Thank you. There are no further questions at this time. I'd like to turn the floor back over to Paula for any closing comments.
Thank you very much everyone, today for joining us on this call. We feel incredibly proud of this historic moment, for X4 with this positive phase III data, and we look forward to shar ing more to come in the future. Have a great night. Thank you, everyone.
This concludes today's conference. You may disconnect your lines at this time. Thank you for your participation.