Greetings, and welcome to X4 Pharmaceuticals' Third Quarter Financial and Operating Results Conference Call. 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 is being recorded. It is now my pleasure to introduce your host, Dan Ferry, from LifeSci Advisors. Thank you, Mr. Ferry. You may begin.
Thank you, operator, and good morning, everyone. Presenting on today's call will be X4's Chief Executive Officer, Dr. Paula Ragan; Chief Commercial Officer, Mark Baldry; and Chief Medical Officer, Dr. Christophe Arbet-Engels. Following prepared remarks by each, we will open up the call to your questions and we'll be joined by Chief Financial Officer, Adam Mostafa; Chief Scientific Officer, Art Taveras; and Chief Operating Officer, Mary DiBiase.
As a reminder, on today's call, the company will be making forward-looking statements regarding regulatory and product development plans, as well as research activities. 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 this quarter's Form 10-Q, which is expected to be filed after market close today. I'd now like to turn the call over to X4's President and CEO, Dr. Paula Ragan. Paula?
Thanks, Dan. Good morning, everyone, and thank you for joining us on the call today. To start, I'll be reviewing our third quarter events and recent highlights, the first two of which will be the primary focus of our discussion today. As you can see here, it's again been a very busy and productive period at X4, which culminated in the great news last week that our NDA, seeking approval of our lead drug candidate, mavorixafor, for the treatment of WHIM syndrome, was accepted by the FDA for priority review. The FDA has set a PDUFA, or action date, of April 30th, 2024, which sets us up for a potential second quarter 2024 launch in the U.S., if approved. In anticipation of this, we have continued to build out our fit-for-purpose commercial organization.
Mark Baldry, our Chief Commercial Officer, will run through some of the details of our launch readiness and pre-launch activities to date, all of which aim to further the understanding of WHIM syndrome and educate both patients and physicians on the importance and benefits of early diagnosis. We do want to remind everyone here that due to mavorixafor's rare pediatric disease designation for WHIM syndrome in the U.S., X4 is eligible to receive a priority review voucher, or PRV, if mavorixafor is approved, that we can use to either obtain priority review for a subsequent application or to sell to another drug sponsor. Turning now to our mavorixafor chronic neutropenia program, we've continued to advance our phase II study and have now enrolled more than 15 participants in the trial.
Importantly, key learnings from this ongoing study, along with the data from previous trials and input from the FDA, have enabled us to finalize the design of what we believe will be the global pivotal phase III clinical trial that will be used as the basis for seeking approval of mavorixafor in certain chronic neutropenic disorders. As discussed on our last quarterly call, Dr. Christophe Arbet-Engels has joined X4 as our new Chief Medical Officer. We thought it would be a good opportunity to hear from him today as we approach some key milestones in our development of mavorixafor in chronic neutropenia. He will be providing an update on this program later in the call. During the quarter, we also strengthened our Board of Directors with the addition of Mr. Keith Woods.
Keith brings significant global commercial strategy and operations experience, most recently at argenx, where as Chief Operating Officer, he helped build a sustainable enterprise and successfully launch a rare disease product globally. His meaningful experience adds to our Board as X4 aims to transition to a global R&D and commercial enterprise as well. As also discussed on our last quarterly call, we completed an expanded loan facility with Hercules Capital. In addition to the possible PRV next year, this facility provides us with financial flexibility and, importantly, non-dilutive financing options. Lastly, we believe our current cash and equivalence balance of just over $140 million at the end of the third quarter is sufficient to fund our operations into 2025. Note that this does not include the debt options or the potential proceeds of the sale of the PRV if NDA approval is obtained.
Before I turn it over to Mark, I'd like to present a quick review of WHIM Syndrome as a reminder to those not familiar with our lead indication. WHIM Syndrome is a rare primary immunodeficiency caused by the over-signaling of the CXCL12/CXCR4 pathway, which in many cases results from genetic variants in the CXCR4 receptor gene. This pathway is a key regulator of normal trafficking of white blood cells, which include neutrophils, lymphocytes, and monocytes. In WHIM Syndrome, white blood cells become trapped in the bone marrow, which leads to chronic neutropenia and lymphopenia.
These immunodeficiencies lead to a number of clinical manifestations, most notably an increased risk of infection in the lungs, skin, and other key organs, and an increase in the incidence of HPV-related cancers, greatly affecting the quality and length of life of the estimated 1,000 people diagnosed with WHIM in the U.S. With earlier diagnosis, we believe the damage from these infections could be mitigated. As you recall, we were able to show in our successful phase III trial on WHIM syndrome, statistically significant increases in both neutrophil and lymphocyte counts, as well as clinically significant reductions in the rate, frequency, and severity of infections versus placebo. If approved next spring, mavorixafor would be the first therapy available for those individuals diagnosed with WHIM syndrome and the only disease-modifying treatment.
I'm now going to turn it over to Mark to give you some of our insights and details on our progress and plans for commercializing mavorixafor for WHIM syndrome. Mark?
Thanks, Paula, and thanks to all of you on the call today. We thought it would be helpful to start by highlighting some of the ways that we've been engaging with the WHIM community, developing an understanding of the market landscape, and establishing a foundation for a successful introduction of mavorixafor to physicians, patients, and payers. As Paula mentioned, there are currently no approved therapies for WHIM syndrome. Physicians are limited to managing their patients' symptoms using repurposed symptomatic treatments. For example, immunoglobulin replacement therapy, which is injected or infused into the body, and/or prophylactic antibiotics, all of which carry their own challenges and do not address the underlying cause of the disease.
Diagnosing WHIM syndrome is also a challenge, given that the clinical presentation of the disease is so variable, and in fact, only about 20% of patients present with all four of the hallmark symptoms and manifestations of WHIM. Some physicians use genetic testing to help identify WHIM or confirm its diagnosis, while other physicians rely more on the clinical presentation of the patient. Due to the rarity of the disease and lack of investment in treatment innovation, there is limited awareness of WHIM syndrome in the general physician community, and the patient journey often includes visits to multiple types of doctors as they try to uncover what might be causing the symptoms.
Given this lack of historical attention and investment, we recognize the significant opportunity to not only address the unmet needs of currently diagnosed WHIM patients, but also to use our resources to identify additional patients in what we believe to be a larger population of undiagnosed people living with WHIM syndrome who could potentially benefit from mavorixafor treatment. The next slide provides an overview of our efforts to date, which have focused on partnering with the WHIM community, driving earlier diagnosis of WHIM, ensuring broad access for eligible patients, and delivering on the promise of mavorixafor. I'll highlight a few of our key successes here. First, with regard to partnering with the WHIM community, we've successfully identified KOLs with expertise and treatment experience in WHIM.
We've assembled an advisory panel, several of whom you've met during our previous data presentation events, and we've worked with numerous experts to author many of our publications. We've also developed and continue to foster collaborations with immunodeficiency patient advocacy groups and disease-focused professional societies in the U.S. and abroad. Through all of these partnerships and collaborations, we believe X4 is playing a significant role in uniting the WHIM community around a common mission to advance the understanding and management of this rare disease. Next, to drive earlier diagnosis of WHIM, we've initiated a number of programs and websites to increase disease awareness, including our most recently launched "What If It's WHIM?" campaign.
As you can see here, through the campaign, we're aiming to educate on the variable clinical presentation of WHIM syndrome, driving home the importance and urgency of early diagnosis, which can lead to better patient outcomes, and providing easy access to additional resources, including direct contact with our field diagnostic team, as well as to free genetic testing. We've also been analyzing medical databases and have completed a significant physician mapping exercise to build our prioritized list of target immunologists and hematologists. We've strengthened our presence at key medical conferences, hosting peer-to-peer disease education symposia, and launching our new Congress exhibition booth, which pulls through our "What if It's WHIM?" campaign, all of which has significantly increased the visibility of X4 and WHIM syndrome and has connected us directly with a broader group of physicians and patients.
In fact, our patient-finding efforts have been accelerating nicely and continue to give us confidence in our prevalence estimate of at least 1,000 patients living with WHIM syndrome in the U.S. To ensure that eligible patients have access to therapy after approval, we've engaged with payers and are developing materials to communicate the compelling value proposition of mavorixafor. Our published clinical data from the phase III 4WHIM trial supports our value messaging by providing evidence of improved neutrophil and lymphocyte counts, and importantly, reductions in the rate, severity, and duration of infections in trial participants treated with mavorixafor. We will be leveraging these points as we begin to discuss pricing and reimbursement going forward. Finally, we've been building an organization that's truly fit for purpose.
We've made key leadership hires across medical and commercial functions, all of whom have significant rare disease and launch experience, and supports the ramp-up of our go-to-market efforts as we head towards an anticipated second quarter 2024 launch in the U.S. I would like to conclude by highlighting the investment synergies here between the WHIM syndrome and chronic neutropenia commercial opportunities. We believe that if we're successful in developing mavorixafor for the treatment of chronic neutropenia, we expect to leverage much of the work and many of the relationships developed through commercializing in WHIM with a future potential commercial launch in CN. And now I'll hand it back to Paula to discuss more about our ongoing development program in CN. Paula?
Thanks so much, Mark. Really great summary of all of our efforts to increase disease awareness, enable earlier diagnosis, and support patients in need as we head towards our anticipated launch in WHIM. Let's now change gears a bit and focus on our chronic neutropenia program. We thought it would be helpful here as well to remind everyone what would make the biggest difference to people living with chronic neutropenia and their physicians. That is, where are the greatest needs in those that we serve? If you recall, a little over a year ago, we held an investor event focused on CN and our initial phase I-B trial results. At that time, we shared that 100% of the 25 patients enrolled achieved robust ANC responses from a single dose of mavorixafor, regardless of background therapy.
Additionally, from our interviews, we gathered valuable insights from patients and physicians, where they highlighted their needs as related to the product profile of a potential new treatment for CN. Their preferred attributes included an oral formulation that is safe, well-tolerated, and easy to administer or take, and one that durably increases ANCs over time. Mavorixafor has already demonstrated these attributes across a number of populations. Additionally, patients and clinicians' criteria for a new treatment includes a therapy that decreases infection burden and also decreases the toxicities and challenges associated with taking lifelong G-CSF. Our successful WHIM phase III trial, where infection rate, severity, and duration were meaningfully reduced, provides evidence that this benefit of mavorixafor could also translate into helping the CN community.
Most importantly, our CN phase II study is beginning to build direct evidence in chronic neutropenic patients across the key metrics of durable ANC increases, safety, and reduction of G-CSF use. I'll now pass it over to Christophe Arbet-Engels, our Chief Medical Officer, to share an update on the status of the promising CN program. Christophe?
Thanks so much, Paula. As you know, following the successful completion of our phase I-B clinical trial, evaluating the ability of a single dose of mavorixafor to raise absolute neutrophil counts, or ANC, in CN patients, we launched a phase II trial to evaluate chronic use of mavorixafor in the same patient population with or without concurrent G-CSF treatment. The study is designed to assess mavorixafor ability to durably raise ANC within the first few months and to see whether patients could possibly reduce or eliminate their G-CSF dose longer term. Importantly, we also assess for safety of mavorixafor in combination with G-CSF. We have seen a nice acceleration in trial enrollment and now have more than 15 participants receiving treatment in the trial.
On our last quarterly conference call, the team presented initial data from the first three participants in the study, all of whom were on G-CSF treatment at study initiation but had identified unmet needs. All achieved large, durable increases in ANC, and the two neutropenic patients achieved normal ANC level despite having low ANC while on G-CSF therapy as they entered the study. Notably, the ANC increase seen would meet the responder criteria that we've established for the phase III study design, which I will go into in more detail shortly. Importantly, the robust ANC response in the early months of the trial enabled the successful dose decrease or complete withdrawal of the G-CSF in some patients. Mavorixafor has also demonstrated an acceptable safety profile in combination with G-CSF.
The full data set on these three subjects will be presented in a poster at the annual ASH meeting in December. In addition, we expect to be able to present a comprehensive update from at least 15 of the participants in the phase II trial during the first half of 2024. Most importantly, we are pleased to rapidly move forward towards our pivotal phase III trial in CN, given all the positive data to date supporting this population. The success we've seen thus far across the CN study, as well as the positive impact that mavorixafor has demonstrated in WHIM patients, has inspired us to advance as quickly as we can by delivering an innovative oral therapy.
With our current insight and input from the FDA, I'll now provide a little more detail on our global pivotal phase III clinical trial, which we remain on track to initiate in the first half of 2024. We've now completed multiple interactions with the FDA and have aligned on the current study design. The trial will be a global, double-blind, randomized, and placebo-controlled trial in participants with or without concurrent G-CSF treatment. The dosing of mavorixafor will be the same as our phase III for WHIM clinical trial. Participants on G-CSF at baseline will remain on a stable dosing regimen during the 52-week study duration, and adjustments in G-CSF dosing will be allowed only for safety reasons.
Patients included in the study are required to have confirmed idiopathic, autoimmune, or congenital chronic neutropenia, with an ANC less than 1,500 cells per microliter and to have demonstrated a history of infection requiring medical intervention in the last 12 months prior to trial entry. The trial is currently designed with a two-component primary endpoint, assessing annualized infection rate and ANC response. Secondary endpoints will include severity and duration of infection, antibiotic use, fatigue, quality of life assessment, and safety. Infections will be assessed by a centralized, blinded adjudication committee, the same approach used for infection assessment in the successful WHIM phase III pivotal study.
Working with Professor Tom Fleming, an FDA Biostatistics Advisor, we've determined the trial size to be 150 participants globally to achieve a power of greater than or equal to 90% for each of the annualized infection rate and ANC response endpoint. With this protocol finalized, we are confident that we will be able to initiate the trial in the first half of 2024. Further adding to our confidence, we have selected our global CROs, who have already begun advancing towards site activation and identifying potential participants for the trial. By leveraging the existing CN patient advocacy groups and global patient registry, and through our own efforts to align a large pool of existing patients with the identified sites, we believe we are now well positioned to achieve full enrollment in the phase III pivotal trial in approximately 12 months. I'll now pass it back to Paula.
Thanks so much, Christophe. We hope it's clear from our discussions today why we are so excited about the future of our company. We have a robust data set maturing in our CN phase II study. Given all the positive data in hand and clear FDA guidance, we are gearing up for a potential launch of our global CN phase III pivotal trial. F inally, we are headed for a potential first approval of mavorixafor in WHIM syndrome in the U.S. and subsequent launch. We look forward to the potential of delivering mavorixafor, a much-needed treatment option, to this underserved group of patients. We very much look forward to the continued periods of growth and accomplishment in the coming years. And with that, we'll now open up the call for your 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. A confirmation tone will indicate your line is in the question queue. You may press star two if you would like to remove your questions 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 hold for questions. The first question comes from the line of Eva Privitera with TD Cowen. Please go ahead.
Hi, good morning, and, thanks for taking our questions. I, I was wondering if you could talk about the CN patients who are on G-CSF but still neutropenic. What are the reasons for their inadequate response? Is it mostly due to intolerance, or are there reasons they're refractory or have inadequate response? I guess I'm trying to get a sense of how heterogeneous this patient population is.
Yeah, that's an excellent question. Christophe, our CMO, will provide some commentary on that.
Yes, thanks. Yes, it is indeed a very heterogeneous population. We do have the plan to have congenital and idiopathic neutropenic patients in our phase III study. There are different reasons, obviously, if there's the genetic mutations in those congenital patients, and the response with G-CSF varies within these patients. So some have a defect in the maturation of the neutrophils, and G-CSF may not address all the aspects of the different phenotypes you can see in these patients.
Maybe just to add, we had an excellent, recent, recent clinical meeting, and what we've heard even from some of our clinicians is how happy some of these patients are to be considered for a study around an oral therapy, given the very difficult tolerance level. Even when they can respond to G-CSF, it's a tough treatment to take.
Great. Thanks for that a nd the second on WHIM, just based on all your commercialization and market-building efforts in WHIM, how do you expect the launch trajectory to look? Do you expect a bolus of patients at launch?
Yeah, we've actually been really pleased with the level of engagement we've had with physicians and patient groups to date. I t's actually given us continuing confidence in our prevalence estimates of about 1,000 patients with WHIM in the U.S. So we're building the foundations for a launch that will get the product to patients as quickly as possible after approval.
Great. Thank you so much for taking my question.
Thank you. Next question comes from the line of Stephen Willey with Stifel. Please go ahead.
Yeah, good morning. Thanks for taking the question. Can you say whether or not, I guess, you've brought any additional sites online in the phase II? I think you had previously said you were at around six. T hen how many sites are you planning for, for the phase III?
Sure. Thanks, Steve. So we have some good lessons learned on the phase II. We did have those six, we maintained it at six, which is why there was a little bit of a hiccup in enrollment, but we're very pleased with the 15+ patients now enrolled, so we'll have a robust data set. But it's a great lesson learned to prepare us for the phase III, and Christophe will share some more on that.
Yeah. So we've learned a lot from the phase II study and from the PIs of the phase II study. We're gonna apply those to the phase III. We are evaluating exactly the number of sites that we're gonna have ex-U.S. and in the U.S. at this time. We are confident we've identified a large proportion of the patients that we plan to enroll in the U.S. and ex-U.S., and feasibility seems to really match with what our expectations are. So we're confident we can enroll this phase III study.
Yeah, and maybe just to give you a little bit of a benchmark, Steve, I think we had around 20 sites, just using rough numbers for WHIM. We have about three times as many patients in CN, so you should expect about three times as many sites for the phase III trial. We're still, you know, finalizing those exact numbers, but it's definitely gonna be a robust number to make sure we hit that one-year enrollment.
Okay, that's helpful. Then, with respect to the phase III protocol in chronic neutropenia, I guess, how are you standardizing G-CSF reduction withdrawal? Is that just investigator subjectivity? And then is there a way that you're gonna try to statistically quantify that at all?
Right. So, G-CSF, as you know, is prescribed individually to patients, and there is a lot of variability. So we're asking our patients in the phase III to remain stable. We want to establish the effectiveness of mavorixafor versus placebo, where we're gonna maintain this. We're gonna allow only modifications of this under a blinded, adjudicated committee for safety reasons. So for example, if their ANC goes too high or if there's a safety event, we're gonna be trying to enforce stable treatment of G-CSF for the 52 weeks of the trial.
Okay. Thanks for taking my questions.
Thank you. Next question comes from the line of Edward Tenthoff with Piper Sandler. Please go ahead.
Thank you very much, and good morning, everyone. Two questions, if I may. Firstly, I think I should know this, but is there any open-label extension work being done in WHIMs? In other words, are there currently any patients on mavorixafor? T hen the second question, you, I think you mentioned, if I heard you correctly, that there will be the three patients, more data on those three patients from ASH. Could there be more patients just because of additional time between abstract submission and the actual presentation? Thank you.
Sure. So I'll just do a quick summary on that. The WHIM OLE is ongoing. We expect to present data on that in the first half of next year. So we'll look forward to sharing some updates on the patients who are on placebo, moving into mavorixafor and those on long-term mav. So stay tuned for that. T hen, in terms of the three patients in the ASH poster, our intent is to really make sure we have a robust data set around those 15 patients in the first half of next year. I think that'll tell the fulsome story around how the drug's acting, but we're really looking forward to presenting the six-month data on those three patients on G-CSF. There's a lot of exciting new information amongst those three patients, so stay tuned for that as well, Ted.
Great. Awesome. I'm looking forward to that. Can you remind me, I know the majority of patients rolled over, how many patients are in the OLE for WHIM? Thanks.
So we haven't given any cut updates, but I think 90%+ of them rolled over, and I'm actually not sure where the cut is at this point, but we'll certainly be disclosing that when we get the data updates. Yep.
Thanks, Paula.
Thanks, Ed.
Thank you. Next question comes from the line of Kalpit Patel with B. Riley Securities. Please go ahead.
Hey, good morning. This is Andy Fleszar on for Kalpit. Thank you for taking the questions. Are you able to give any additional granularity on the timing of the next comprehensive update for the 15 patients, or if there's a certain amount of follow-up that you'll be looking for before presenting the results?
Sure, Christophe, go ahead. Yep.
Yeah, no, there will be... We will have all of those, those data in a more comprehensive manner in the first half of, of next year. The ASH poster will help, share some preliminary information, and we hope to continue this. We want to remind you that the phase II study is a six-month study. It's an open label, so we could do different analysis, but it is important that we see those six months data, and we see this in the, in, the largest number of patients to, raise the confidence of the preliminary data which we are going to be seeing at ASH.
Okay, and then as a follow-up to that, do you anticipate the phase II data to be enriched for cyclic, congenital, or idiopathic CN?
No, I think what we're doing for the phase II, of course, is all comers, because there's two questions we're trying to answer. Obviously, making sure that our phase III study design is robustly incorporating any responses that we're seeing. T hen secondly, there's the longer-term question around G-CSF. So both of those questions need to be answered. That's why we are including a diverse population with no pre-specified numbers in any of those categories.
That makes sense and then one final question from us. From your market research, can you speak about what level of G-CSF reduction is clinically meaningful to patients and prescribers? Obviously, more reduction is better, but any color that you can provide on what success might look like would be helpful.
That's an excellent question, and our answer is come to the ASH poster, 'cause we'll be actually providing some very meaningful market research around that exact question.
Sounds great. We look forward to seeing you there. Thank you.
Thank you.
Thank you. Next question comes from the line of Kristen Kluska with Cantor Fitzgerald. Please go ahead.
Hi, good morning, everyone. Thanks for taking the question. When I look at your finalized phase III CN study design, in a lot of ways, as you mentioned, it really does mirror what the trial looked like for WHIM, which of course, was successful. So can you talk about how you think that gives you the confidence in this particular study, and any differences we should really be thinking about related to the mechanism in these subset of patients versus WHIM?
No, so we have worked with one of our consultants, Professor Tom Fleming, from the FDA, in designing that study very closely with him. We are confident that we have the power for our primary endpoint to be achieved, and we have the right sample size. We have two population that we're going to be including in that study, which is the monotherapy and the patients on G-CSF. Because it's a randomized, double-blind, controlled trial versus placebo, we will be able to establish the effectiveness, the efficacy of mavorixafor. We also incorporated in that study the requirements from, and the feedback from, the FDA that we received. So we believe we have all the elements to recruit and see positive data in this phase III study.
Maybe just to remind folks, in the WHIM phase III, we saw about a 60% reduction across those patient populations. We actually took a fairly conservative approach, even reducing that to plan for the stats on the phase III, which is why we feel very confident.
Okay, thanks for that and it's nice to see the uptick in enrollment in this update. Can you maybe talk about what you think some of the factors are that, you know, it was originally slower than expected, and now that you've seen this uptick? So was it things like seasonality? And again, how this also helps for planning for phase III. Thanks again.
Yeah, sure. I mean, I think trial enrollment is a challenge for every biotech out there, especially in a new field such as chronic neutropenia. But I think what we just learned is, you know, the summer is tough for some the younger patients. You know, we actually have fairly sick patients in this trial, but they certainly wanted to have some downtime, as is expected. The trial enrollment's picked up nicely. I mean, more importantly, phase IIIs are very different from phase IIs. They're a very heavy operational lift. You want to create a large pool of patients waiting that are co-localized with your sites. We actually have a very nice start to that with quite a robust patient pool already identified to the sites that are coming on Board.
We look forward to certainly hitting that 12, 12-month enrollment with high confidence.
Are you done with the question, Ms. Kluska?
Yes. Thank you.
Thank you. Next question comes from the line of Swayampakula Ramakanth with H.C. Wainwright. Please go ahead.
Thank you. Good morning, Paula and Adam. A lot of my questions have been answered. Just looking into the phase III study in CN disorders, you know, what's the label you're looking for, or, you know, once we get into the application stage? And also, do you foresee either increasing the size of the trial as the trial progresses so that you maintain a certain number of patients in these individual populations so that you can get a broad label?
So, regarding the label, based on the previous label for G-CSF, for example, we should see a label that addresses the reductions of infections in the patient populations that we include in this trial. S o we haven't finalized the label yet with the FDA, obviously, but this is what we think could be happening, and we're looking at this reduced infection rate. With regards to...
Yeah, I think he was just asking about subpopulations. I mean, I-
Oh, yeah.
Similar to G-CSF, subpopulations were kind of catch as you catch can, but it's based on the inclusion, and then the label covered all the variable patient populations.
So right. Yes, and the trial, again, is powered 90% for our primary endpoint. So we're planning to have this study executed with the sample size, and we're confident that we should be able to hit our primary endpoint with the current sample size.
Yep.
Okay. Thank you. Thanks for taking my question.
Thank you. Next question comes from the line of David Bautz with Zacks Small- Cap Research. Please go ahead.
Hey, good morning, everyone. Thanks for the update this morning. Just one question for me. I'm curious what type of doctor ends up typically making the diagnosis for WHIM? And I guess what I'm trying to get at is how are you sure that you're reaching the correct physician population with your outreach program?
That's a great question. Because of the variable presentation of the disease, these patients have a different, you know, journey, diagnostic journey, depending on their symptoms. So some of the patients end up under the care of immunologists, and some of the patients end up under the care of a hematologist or a hem-onc. So, this is what we've been focused on for the past few months, is really looking at the data, engaging with key institutions, and learning about who are the physicians most likely to have these patients, so that once we get approved, we know where to prioritize our efforts. We've also been complementing this with, you know, our digital marketing, which I talked about earlier in the presentation, and this is an efficient way of extending our reach.
Okay, great. Thanks for that.
Thank you. This concludes today's question and answer session. I would now like to turn the floor over to Paula Ragan for closing comments.
Well, thank you very much again today for joining the call. We look forward to providing continued updates in the future as we make our exciting progress as a company. Have a great day.
Thank you. This concludes today's teleconference. You may disconnect your lines at this time. Thank you for your participation.