X4 Pharmaceuticals Q3 2023 Earnings Call Transcript

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Provided by Quartr
November 9, 2023

There are 12 speakers on the call.

Operator

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.

Operator

Thank you, Mr. Ferri. You may begin.

Speaker 1

Thank you, operator, Good morning, everyone. Presenting on today's call will be Exforge's Chief Executive Officer, Doctor. Paula Regan Chief Commercial Officer, Mark Baldry and Chief Medical Officer, Doctor. Christophe Arbat 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 Tavares and Chief Operating Officer, Mary DiBiase.

Speaker 1

As a reminder, on today's call, the company will be making forward statements regarding regulatory and product development plans as well as research activities. These statements are subject to risks and uncertainties that may 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 X Force President and CEO, Doctor. Paula Redican.

Speaker 1

Paula?

Speaker 2

Thanks, Dan. Good morning, everyone, and thank you for joining us on the call today. To start, I'll be reviewing our Q3 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, maverixafor, for the treatment of WIM syndrome was accepted by the FDA for priority review. The FDA has set a PDUFA or action date of April 30, 2024, which sets us up for a potential second quarter 2024 launch in the U.

Speaker 2

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 the WIM 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 mavericks for rare pediatric disease designation for WIM syndrome in the U.

Speaker 2

S, X4 is eligible to receive a priority review voucher or PRB if Maverick's 4 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 mavrixiphora chronic neutropenia program. We have continued to advance our Phase 2 study and have now enrolled more than 15 have enabled us to finalize the design of what we believe will be the global pivotal Phase 3 clinical trial that will be used as the basis for seeking approval of MAVERICK for in certain chronic neutropenic disorders. As discussed on our last quarterly call, Doctor. Christophe Arbat Engels has joined X4 as our new Chief Medical Officer.

Speaker 2

We thought it would be a good opportunity to hear from him today as we approach some key milestones And our development of MAVERICK's before 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 Wood. He brings significant global commercial The transition to a global R and D and commercial enterprise as well.

Speaker 2

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 An equivalent balance of just over $140,000,000 at the end of the 3rd quarter is sufficient to fund our operations into 2025. And note that this does not include the debt options or the potential proceeds of the sale of the PRD if NDA approval is obtained. Before I turn it over to Mark, I'd like to present a quick review of WIM syndrome as a reminder to those not familiar with our lead indication.

Speaker 2

Limb syndrome is a rare primary immunodeficiency caused by the over signaling of the CXCL12, CXCR4 pathway, which in many cases results from genetic variance 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 WIM syndrome, white blood cells become trapped in the bone marrow, which leads to 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 and the incidence of HPV related cancers, greatly affecting the quality and length of life of the estimated 1,000 people diagnosed with WIM in the U. S. With earlier diagnosis, we believe the damage from these infections could be mitigated.

Speaker 2

As you recall, we were able to show in our successful Phase 3 Frequency and severity of infections versus placebo. If approved next spring, MAVERICK-four would be the first therapy available for those individuals diagnosed with limb 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 MAVERICK's forum for women syndrome. Mark?

Speaker 3

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 WIM community, Developing an understanding of the market landscape and establishing a foundation for a successful introduction of maverixifor to physicians, patients and payers. As Paula mentioned, there are currently no approved therapies for WIMS 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 WIMS syndrome is also a challenge given that the clinical presentation of the disease is so variable.

Speaker 3

And in fact, only about 20% of patients present with all 4 of the hallmark symptoms and manifestations of WIM. 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 WIM 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 WIM 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 WIMM syndrome who could potentially benefit from MAVERICK's for treatment. The next slide provides an overview of our efforts to date, which have focused on partnering with the WIM community, Driving earlier diagnosis of WIM, ensuring broad access for eligible patients and delivering on the promise of maverixafor.

Speaker 3

I'll highlight a few of our key successes here. First, with regard to partnering with the WIM community, We've successfully identified KOLs with expertise and treatment experience in WIM. We've assembled an advisory panel, several of whom you've met during our 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 Through all of these partnerships and collaborations, we believe is playing a significant role in uniting the WIM community around a common mission to advance the understanding and management of this rare disease. Next, to drive earlier diagnosis of WIM, we've initiated a number of programs and websites to increase disease awareness, including our most recently launched What If It's Wim campaign.

Speaker 3

As you can see here, Through the campaign, we're aiming to educate on the variable clinical presentation of WIM 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 to build our prioritized list of target immunologists and hematologists. We 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 Wim campaign, all of which has significantly increased the visibility of X4 and WIMS 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 WIMM syndrome in the U. S.

Speaker 3

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 mavericksof4. Our published clinical data from the Phase 3 4 WIM 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 MAVERICK support. We will be leveraging these points as we begin to discuss pricing and reimbursement going forward. And 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 2nd quarter 2024 launch in the U.

Speaker 3

S. I would like to conclude by highlighting the investment synergies here between the WIM syndrome and chronic neutropenia commercial opportunities. We believe that if we're successful in developing MAVERICK's 4 for the treatment We expect to leverage much of the work and many of the relationships developed through commercializing in WIM 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?

Speaker 2

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 WIM. 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 and those that we serve?

Speaker 2

If you recall, a little over a year ago, we held an investor event focused on CN and our initial Phase 1b trial results. At that time, we shared that 100 percent of the 25 patients enrolled achieved robust ANC responses From a single dose of MAVERICK before 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 A and Cs over time. MAVERICK4 has already demonstrated these attributes across a number of populations.

Speaker 2

Additionally, patients and clinicians' criteria for new treatment includes a therapy that decreases infection burden and also decreases the Our successful LIM Phase 3 trial Where infection rate, severity and duration were meaningfully reduced provides evidence that this benefit of MAVERICK's forest could also translate into helping the CN community. Most importantly, our CN Phase 2 study is beginning to build direct evidence in chronic neutropenic patients across the key metrics of durable A and C increases, safety and reduction of G CSF use. I'll now pass it over To Christophe Arbat Engels, our Chief Medical Officer, to share an update on the status of the promising CN program. Christophe?

Speaker 4

Thanks so much, Paula. As you know, following the successful completion of our Phase 1b clinical trial, Evaluating the ability of a single dose of MAVERICKZA 4 to raise absolute neutrophil counts or ANC in CN patients, We launched a Phase 2 trial to evaluate chronic use of maverick support in the same patient population with or without concurrent G CSF treatment. The study is designed to assess MAVERICKA-four ability to durably raise ANC within the 1st few months and to see whether patients could possibly reduce or eliminate their GCSS dose longer term. Importantly, we also assess for safety of MAVERICKI4 in combination with DCFS. We have seen a nice acceleration in trial enrollment and now have more than 15 participants receiving treatment in the trial.

Speaker 4

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 2 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 3 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. Mavilyxophore has also demonstrated an acceptable safety profile in combination with GTSS.

Speaker 4

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 2 trial during the first half of twenty twenty four. Most importantly, We are pleased to rapidly move forward towards our pivotal Phase 3 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 Mavorexo4 has demonstrated in wound 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 3 clinical trial, which we remain on track to initiate in the first half of twenty twenty four.

Speaker 4

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 participant with or without concurrent G CSF treatment. The dosing of maverick's 4 will be the same as our Phase 3 for William Clinical Trial. Participants from G CSF at baseline will remain on a stable dosing regimen during the 52 week Study duration and adjustments in GCSS dosing will be allowed only for safety reasons. Patients included in the study are required to have confirmed idiopathic autoimmune or congenital chronic neutropenias With an ANC less than 1500 cells per microliter, NC have demonstrated a history of infection requiring medical intervention in the last 12 months prior to trial entry.

Speaker 4

The trial is currently designed with the 2 component Primary endpoint assessing annualized infection rates 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 win Phase 3 pivotal study. Working with Professor Tom Fleming, An FDA Biostatistics adviser, we 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 endpoints.

Speaker 4

With these protocols finalized, we are confident that we will be able to initiate the trial in the first half of twenty twenty four. And 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 We align a large pool of existing patients with the identified site. We believe we are now well positioned to achieve full enrollment in the Phase 3 pivotal trial in approximately 12 months. I'll now pass it back to Paula.

Speaker 2

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 2 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 3 pivotal trial. And finally, we are headed for a potential 1st approval of MAVERICK's foreign and women syndrome in the U.

Speaker 2

S. And subsequent launch, we look forward to the potential of delivering MAVERICK's for a much needed treatment option to this underserved group of patients. We very much look forward to the continued periods of growth and accomplishments in the coming years. And with that, we'll now open up the call for your questions. Operator?

Operator

Thank you. We will now be conducting a question and answer session. One moment please while we poll for questions. The first question comes from the line of Eva Privaterra with TD Cowen. Please go ahead.

Speaker 5

Hi, good morning and thanks for taking our questions. I was wondering if you could talk about the Seeing patients who are on G CSF, but still neutropenic, what are the reasons for their inadequate response? Is it mostly due to Comments or other reasons they're refractory or have inadequate response? I guess I'm trying to get a sense of how heterogeneous this patient population is.

Speaker 2

It's an excellent question. Christophe, our CMO will provide some commentary on that.

Speaker 6

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 is 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 But the neutrophils and G CSF may not address all the aspects of the different phenotypes you can see in this patient.

Speaker 2

Maybe just to add, we have an excellent 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 levels, Yes. And even when they can respond to G CSF, it's a tough treatment to take.

Speaker 5

Great. Thanks for that. And second on WIM, Just based on all your commercialization and market building efforts in WIM, how do you expect the launch Trajectory to look, do you expect a bolus patient at launch?

Speaker 3

Yes, we've actually been really pleased with The level of engagement we've had with physicians and patient groups to date and it's actually given us continuing confidence in our prevalence Estimates of about 1,000 patients with WIM 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.

Speaker 5

Great. Thank you so much for taking my question.

Operator

Thank you. Next question comes from the line of Stephen Willey with Stifel. Please go ahead.

Speaker 7

Yes, 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 2, I think you had previously said you were at around 6. And then how many sites are you planning for the Phase 3?

Speaker 2

Sure. Thanks, Steve. So we have some good lessons learned on the Phase 2. We did have those 6. We maintained it at 6, which why there's a little bit of a hiccup in enrollment, but we're very pleased with the 15 plus patients now enrolled.

Speaker 2

So we'll have a robust data set, but it's a great lesson learned to prepare us for the Phase 3 and Safo will share some more on that.

Speaker 6

Yes. So we've learned a lot from the Phase 2 study and from the Yes. It's a Phase II study. We're going to apply those to the Phase III. We are evaluating exactly the number of sites that we're going to have ex U.

Speaker 6

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.

Speaker 6

S. And ex U. S. And feasibility seems to really match with Tracking with what we are our expectations are. So we're confident we can enroll this Phase 3 study.

Speaker 2

Yes. 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 WIM. We have about 3 times as many patients in Yes. So you should expect about 3 times as many sites for the Phase 3 trial. We're still finalizing those exact numbers, but it's definitely going to be a robust number to make sure we hit that 1 year enrollment.

Speaker 7

Okay. That's helpful. And then, with respect to the Phase The re protocol in chronic neutropenia. I guess, how are you standardizing G CSF reduction withdrawal? Is that just Investigator subjectivity.

Speaker 7

And then is there a way that you're going to try to statistically quantify that at all?

Speaker 6

Right. So G CSF, as you know, is prescribed individually to patients and there is a lot of variability. So we are Asking our patients in the Phase III to remain stable, we want to establish the effectiveness of mavericksof4 versus placebo, And we're going to maintain this. We're going to allow only modifications of that under blinded adjudicated committee For safety reasons, so for example, if their ANC goes too high or if there is a safety event, we're going to be Trying to enforce stable treatment of G CSF for the 52 weeks of the trial.

Speaker 7

Okay. Thanks for taking my questions.

Operator

Thank you. Next question comes from the line of Edward Tynanoff with Piper Sandler. Please go ahead.

Speaker 8

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 wins. In other words, are there currently any patients on mavarixa for? And then the second question, I think you mentioned, if I heard you correctly, that there will be the 3 patients, more data on those 3 patients from ASH.

Speaker 8

Could there be more Just because of additional time between abstract submission and the actual presentation. Thank you.

Operator

Sure. So I'll just do a

Speaker 2

quick summary on this. The WAM OLE is ongoing. We expect to present data on that on the first half of next year. So we'll look forward to sharing some updates on the patients who are on placebo moving into MAVERICK-four and those on long term MAV. So stay tuned for that.

Speaker 2

And then, in terms of the 3 patients in the ASH poster, our intent is to really make sure we have a robust data set around 15 patients in the first half of next year, I think that will tell the fulsome story around how the drug is acting. But we're really looking forward to presenting the 6 month data on those 3 patients on G CSF. There's a lot of exciting new information amongst those 3 patients, so stay tuned for that as well, Ted.

Speaker 8

Great. Awesome. I'm looking forward to that. And can you remind me, I know the majority of patients rolled over, how many patients are in the OLE for when? Thanks.

Speaker 2

So we haven't given any cuts updates, but I think 90 plus percent of them rolled over and I'm actually not sure where the cut is at this But we'll certainly be disclosing that when we get the data updates. Yes.

Speaker 4

Thanks, Colin.

Speaker 2

Thanks, Ted.

Operator

Thank you. Next question comes from the line of Talpreet Patel with B. Riley Securities. Please go ahead.

Speaker 9

Hey, good morning. This is Andy Pleasure on for Kelvin. 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?

Speaker 2

Sure, Christophe, go ahead.

Speaker 6

Yes. No, there will be we will have all of those data in a more comprehensive manner in the first half of next year. The ASH poster will help share some preliminary information And we hope to continue this. I want to remind you that the Phase 2 study is a 6 month study. It's an open label, so we could do Different analysis, but it is important that we see those 6 months data and we see this in the largest number of patients to raise Of the preliminary data we are going to be seeing at ASH.

Speaker 9

Okay. And then as a follow-up Do you anticipate the Phase 2 data to be enriched for cyclic, congenital or idiopathic again?

Speaker 2

No, I think what we're doing for the Phase 2, of course, is all comers because there's 2 questions we're trying to answer. Obviously, making sure that our Phase 3 study design is robustly incorporating any responses that we're seeing. And then secondly, there's the longer term question around GCSF. 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.

Operator

That makes sense. And then

Speaker 9

one final question from us. From your market research, can you speak about what level of GCF 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.

Speaker 2

That's an excellent question and our answer is come to the ASH poster because we'll be actually providing some very meaningful market research around that exact

Speaker 9

Sounds great. We look forward to seeing you there. Thank you.

Speaker 2

Thank you.

Operator

Thank you. Next question comes from the line of Kristin Klipska with Cantor Fitzgerald. Please go ahead.

Speaker 5

Hi, good morning, everyone. Thanks for taking the question. When I look at your finalized Phase 3CN study design, In a lot of ways, as you mentioned, it really does mirror what you 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 the subset of patients versus when?

Speaker 6

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 2 population that we're going to be included in that study, which is the monotherapy and the patients on G CSF. And because it's a randomized double blind controlled trial versus placebo, we will be able to establish The effectiveness, the efficacy of MAVERICK's support. We also incorporated in that study the requirements from and the feedback from the FDA that we received.

Speaker 6

And so we believe we have all the elements to recruit And see positive data in this Phase 3 study.

Speaker 2

And maybe just to remind folks, in the WIM Phase 3, we thought about a 60% Reduction across the patient populations, we actually took a fairly conservative approach even reducing that to plan for the stats on the Phase 3, which is why we feel very confident.

Speaker 5

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 it was originally slower than expected now that you've seen this uptick? So were things like seasonality and again how this also helps for planning for Phase 3?

Speaker 5

Thanks again.

Speaker 2

Yes, sure. I mean, I think trial enrollment is a challenge for every biotech out there, especially in a new field such as chronic neutropenia. I think what we just learned is the summer is tough for some of the younger patients. We actually have fairly sick patients in this trial, but they certainly wanted to Have some downtime as is expected. The trial enrollments picked up nicely.

Speaker 2

I mean, more importantly, Phase 3s are very different from Phase 2s. They're 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 a quite a robust patient pool already identified to the sites that are coming on board. So we look forward to certainly hitting that 12 month enrollment with high

Operator

Are you done with the question, Ms. Kluska?

Speaker 5

Yes. Thank you.

Operator

Thank you. Next question comes from the line of Swayampakula Ramakanth with H. C. Wainwright. Please go ahead.

Speaker 10

Thank you. Good morning, Paula and Adam. A lot of my questions have been answered. Just looking into the Phase 3 study in CNS disorders, what's the label you're looking for 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 There's certain number of patients in this individual populations, so that you can get a broad label.

Speaker 6

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. And so 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 And Liz?

Speaker 2

Yes, I think he was just asking about subpopulations. I mean, I similar to GCSF, 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 population.

Speaker 4

So right,

Speaker 6

yes. And we're not we're The trial again is powered 90% for our primary endpoint. So we're not planning we're Planning to have this study executed as with the sample size and we're confident that we should be able to hit our primary endpoint with the current sample size.

Speaker 9

Okay. Thank

Speaker 10

you. Thanks for taking my question.

Operator

Thank you. Next question comes from the line of David Borge with Zacks Small Cap Research. Please go ahead.

Speaker 11

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 WIM. 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?

Speaker 3

Yes, it's a great question. And because of the variable presentation of the disease, these patients have a different 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 hemonc. 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 our digital marketing, which I talked about earlier in the presentation.

Speaker 3

And this is an Efficient way of extending our reach.

Speaker 11

Okay, great. Thanks for that.

Operator

Thank you. This concludes today's question and answer session. I would now like to turn the floor over to Paula R. Reagan for closing comments.

Speaker 2

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.

Operator

Thank you. This concludes today's teleconference. You may disconnect your lines at this time. Thank you for your participation.

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