X4 Pharmaceuticals Inc (XFOR) 2024 Q3 法說會逐字稿

Greetings, and welcome to the X4 Pharmaceuticals call and webcast. (Operator instructions) As a reminder, this conference call is being recorded.

您好，歡迎收聽 X4 Pharmaceuticals 的電話和網路廣播。（操作員說明）謹此提醒，本次電話會議正在錄音中。

It is now my pleasure to introduce your host, Dan Ferry from LifeSci Advisors. Please begin.

現在我很高興向您介紹主持人，來自 LifeSci Advisors 的 Dan Ferry。請開始。

Thank you, operator, and good morning, everyone. Thank you for joining us today for the special earnings call, during which X4 will focus on providing a business update and importantly, data and results from its now completed Phase 2 study of mavorixafor in chronic neutropenia.

謝謝接線員，大家早安。感謝您今天參加我們的特別財報電話會議，在此期間，X4 將重點提供業務更新，重要的是，其現已完成的 mavorixafor 治療慢性中性粒細胞減少症的 2 期研究的數據和結果。

As a reminder, on today's call, the company will be making forward-looking statements regarding regulatory and product development and commercialization plans, as well as research activities and financial projections.

提醒一下，在今天的電話會議上，該公司將就監管、產品開發和商業化計劃以及研究活動和財務預測發表前瞻性聲明。

These statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted. Description of these risks can be found in X4's most recent filings with the SEC, including this year's Form 10-K, which was filed on March 21, 2024, and in the company's Form 10-Q for the third quarter, which is expected to be filed shortly.

這些陳述存在風險和不確定性，可能導致實際結果與預測不同。這些風險的描述可以在 X4 最近向 SEC 提交的文件中找到，包括今年於 2024 年 3 月 21 日提交的 10-K 表格，以及該公司第三季的 10-Q 表格，預計該表格很快就會被歸檔。

Presenting on today's call will be Dr. Paula Ragan, X4's President and CEO; and the company's Chief Medical Officer, Dr. Christophe Arbet-Engels.

X4 總裁兼執行長 Paula Ragan 博士將出席今天的電話會議。以及公司首席醫療官 Christophe Arbet-Engels 博士。

I'll now turn it over to Paula Ragan to run through today's agenda. Paula?

現在我將把今天的議程交給保拉·拉根 (Paula Ragan)。保拉？

Thank you, Dan, and thank you all for joining us. Today, we will provide an update on our operational achievements, including activities supporting the launch of XOLREMDI in the US and the substantial progress to date with our ongoing Phase 3 clinical trial of mavorixafor in people with chronic neutropenia or CN.

謝謝丹，也謝謝大家加入我們。今天，我們將提供有關我們營運成就的最新信息，包括支持XOLREMDI 在美國上市的活動，以及我們正在進行的mavorixafor 在慢性中性粒細胞減少症或CN 患者中進行的3 期臨床試驗迄今為止取得的重大進展。

In addition, we're excited to present the positive data from our recently completed Phase 2 study of mavorixafor in CN. These results include the final mavorixafor monotherapy data, which remains very encouraging and consistent with the data we presented in June from the interim Phase 2 analysis.

此外，我們很高興地展示我們最近在中國完成的 mavorixafor 2 期研究的積極數據。這些結果包括最終的 mavorixafor 單藥治療數據，該數據仍然非常令人鼓舞，並且與我們 6 月提供的中期 2 期分析數據一致。

We will also be presenting new data from the study. Results showing that most of the participants who were treated with mavorixafor and G-CSF ended up having their G-CSF dose adjusted downward during the study, while the mean neutrophil count for this group remained in the normal range. These data provide a first glimpse into how mavorixafor could be used in the real world if ultimately approved in CN.

我們還將展示該研究的新數據。結果顯示，大多數接受 mavorixafor 和 G-CSF 治療的參與者最終在研究期間下調了 G-CSF 劑量，而該組的平均中性粒細胞計數仍保持在正常範圍內。這些數據讓我們初步了解如果最終在 CN 獲得批准，mavorixafor 將如何在現實世界中使用。

Equally exciting are the new insights we've gained from assessing the functionality of circulating neutrophils in a subset of the Phase 2 study participants, as well as healthy donors for a comparison. And we'll briefly review this updated safety data from the study as well.

同樣令人興奮的是，我們透過評估第二階段研究參與者的一部分以及用於比較的健康捐贈者的循環中性粒細胞的功能而獲得了新的見解。我們也將簡要回顧該研究中更新的安全數據。

We'll then conclude and open it up for your questions, then we will be joined by our Chief Commercial Officer, Mark Baldry; our Chief Financial Officer, Adam Mostafa, our Chief Operating Officer, Dr. Mary DiBiase; our Chief Scientific Officer, Dr. Art Taveras; and our Head of Corporate and Patient Affairs, Jose Juves.

然後我們將結束並開放供您提問，然後我們的首席商務官 Mark Ba​​ldry 將加入我們；我們的財務長 Adam Mostafa、我們的營運長 Mary DiBiase 博士；我們的首席科學官 Art Taveras 博士；以及我們的公司和病患事務主管 Jose Juves。

So let's quickly run through the operational update as we know you're all eager to hear about the CN Phase 2 study results and analysis. As you know, we received US FDA approval in late April for our first product, mavorixafor, branded XOLREMDI, for the treatment of WHIM syndrome, a rare primary immunodeficiency disease.

因此，讓我們快速瀏覽一下操作更新，因為我們知道你們都渴望聽到 CN 第二階段的研究結果和分析。如您所知，我們的首款產品 mavorixafor（品牌為 XOLREMDI）於 4 月下旬獲得美國 FDA 批准，用於治療 WHIM 症候群（一種罕見的原發性免疫缺陷疾病）。

As with ultra-rare disease launches, our initial commercial strategy has been focused on disease awareness to support patient identification and diagnosis. While we do recognize that sales appear to be flat quarter-over-quarter, we want to remind everyone that some of the sales in the second quarter were products stocking with our specialty pharma as is typical during initial launch.

與推出超罕見疾病一樣，我們最初的商業策略一直專注於疾病意識，以支持患者識別和診斷。雖然我們確實意識到銷售額似乎與上一季持平，但我們想提醒大家，第二季的部分銷售額是我們的專業製藥公司庫存的產品，這在最初推出期間是典型的情況。

I'm pleased to report this past quarter, we hit our most important launch goal, having engaged with all 3,400 of the initial immunologists and hematologists targeted for the first phase of our launch. And these visits and digital engagements are bearing fruit, as you can see here. Recently completed market research comparing pre and post-launch metrics indicate that knowledge of WHIM syndrome and these likely XOLREMDI prescribers has increased and is now at more than 75%.

我很高興地向大家報告，上個季度，我們與第一階段啟動的全部 3,400 名初始免疫學家和血液學家進行了接觸，實現了最重要的啟動目標。正如您在此處所看到的，這些訪問和數位互動正在取得成果。最近完成的市場研究比較了發布前和發布後的指標，顯示對 WHIM 綜合徵和這些可能的 XOLREMDI 處方者的了解有所增加，目前已超過 75%。

In addition, an increasing number of physicians are screening their patients for WHIM, and 80% of HCP surveyed report that they would consider prescribing XOLREMDI for their WHIM patients. Over the past six months, we've also stepped up our conference attendance, launched a peer-to-peer speaker program and initiated a patient focused campaign, which includes a website, new materials and an HCP discussion guide. Reimbursement and access continue to go well with favorable policy decisions resulting in more than 150 million lives now covered. So very good progress there.

此外，越來越多的醫生正在對患者進行 WHIM 篩檢，接受調查的 80% 的 HCP 表示，他們會考慮為 WHIM 患者開立 XOLREMDI 處方。在過去的六個月中，我們還增加了會議出席率，啟動了點對點演講者計劃，並發起了以患者為中心的活動，其中包括網站、新材料和 HCP 討論指南。報銷和取得繼續與有利的政策決定配合良好，現已覆蓋超過 1.5 億人的生命。所以那裡的進展非常好。

Importantly, the activities we are conducting in WHIM are yielding insights that may support our efforts to advance mavorixafor into the much broader indication of chronic neutropenia. And while our commercial efforts are exclusively focused on WHIM today, we do see a significant overlap between the WHIM and CN communities.

重要的是，我們在 WHIM 中開展的活動正在產生見解，這些見解可能支持我們努力將 mavorixafor 推向更廣泛的慢性中性粒細胞減少症適應症。雖然我們今天的商業努力完全集中在 WHIM 上，但我們確實看到 WHIM 和 CN 社區之間存在顯著的重疊。

Based on our claims analysis and experience in the field, we observed that a majority of hematologists that we're calling on for WHIM also see chronic neutropenia patients. And of course, there's overlap within the patient advocacy communities we engage with as well.

根據我們的索賠分析和該領域的經驗，我們觀察到我們要求進行 WHIM 的大多數血液科醫生也診治慢性中性粒細胞減少症患者。當然，我們參與的患者倡導社群內部也存在重疊。

If mavorixafor is approved for use in CN, we certainly expect to be able to leverage our commercial infrastructure and relationships within the WHIM community to support a potential launch into chronic neutropenia. As you all know, success in CN will be dependent on the results of our global pivotal Phase 3 trial, which is progressing well and currently participants are being screened and dosed across multiple countries.

如果 mavorixafor 被批准用於 CN，我們當然希望能夠利用我們的商業基礎設施和 WHIM 社區內的關係來支持潛在的慢性中性粒細胞減少症治療。眾所周知，CN 的成功將取決於我們全球關鍵 3 期試驗的結果，該試驗進展順利，目前正在多個國家對參與者進行篩選和給藥。

Our 4WARD, as we've named it, is a global trial that will engage patients and physicians across 20 to 25 countries. We can report today that we have already received health authority authorizations to initiate the trial in approximately 85% of these countries. We can also now report that approximately 40% of our planned sites worldwide are initiated and we expect to have the majority of sites initiated in early '25.

正如我們所命名的，我們的 4WARD 是一項全球試驗，將吸引 20 至 25 個國家的患者和醫生參與。今天我們可以報告說，我們已經獲得衛生當局的授權，可以在其中約 85% 的國家啟動試驗。我們現在還可以報告，我們在全球範圍內規劃的站點中約有 40% 已啟動，我們預計大多數站點將在 25 年初啟動。

This trial is expected to enroll 150 participants at 90 to 110 sites with a planned enrollment of one to two participants per site, which is typical in a rare disease clinical trial. Most importantly, we are on track and continue to expect full trial enrollment in mid-2025.

該試驗預計將在 90 至 110 個地點招募 150 名參與者，每個地點計劃招募 1 至 2 名參與者，這在罕見疾病臨床試驗中是典型的。最重要的是，我們正在步入正軌，並預計在 2025 年中期實現全面試驗註冊。

With that, I'll now turn it over to Christophe to get to the heart of our call today, data results from the completed Phase 2 study in chronic neutropenia. Christophe?

現在，我將把它交給 Christophe，讓他了解我們今天電話會議的核心內容，即已完成的慢性中性粒細胞減少症第二階段研究的數據結果。克里斯托夫？

Thanks so much, Paula, and hello to everyone on the call. Let me begin with a quick refresher on chronic neutropenia. The hallmark of chronic neutropenia is abnormally low levels of circulating neutrophils that are the defense mechanism our body uses to fight pathogens. As we've presented previously, there is a well established correlation between circulating levels of neutrophils or absolute neutrophil count, ANC, and the risk of frequent and, or serious infection.

非常感謝，寶拉，並向通話中的每個人問好。首先讓我快速回顧一下慢性嗜中性白血球減少症。慢性中性粒細胞減少症的特徵是循環中性粒細胞水平異常低，這是我們身體用來對抗病原體的防禦機制。正如我們之前所介紹的，中性粒細胞的循環水平或中性粒細胞絕對計數、ANC 與頻繁和/或嚴重感染的風險之間存在明確的相關性。

Note that the lower limit of normal for neutropenia diagnosis is 1,500 cells per microliter. Below that, grades are divided based on the infection risk in increments of 500 cells per microliter from mild to moderate to severe. As you can see, severely neutropenic patients have ANC of less than 500, and this corresponds to the highest risk of infection.

請注意，中性粒細胞減少症診斷的正常下限是每微升 1,500 個細胞。在此之下，根據感染風險以每微升 500 個細胞為增量，從輕度到中度再到重度劃分等級。如您所見，嚴重中性粒細胞減少症患者的 ANC 低於 500，這對應於最高的感染風險。

To reduce the risk of infection in these patients, it is critical to move them from the most severe grade towards less severe grade to achieve as normal a level of neutrophil as possible. Therefore, an increase of 500 cells per microliter is an important clinical practice objective. Through its mechanism of action, mavorixafor has been shown to do precisely that, increase the number of circulating neutrophils.

為了降低這些患者的感染風險，至關重要的是將他們從最嚴重的級別轉移到不太嚴重的級別，以盡可能達到正常的中性粒細胞水平。因此，每微升增加500個細胞是重要的臨床實務目標。透過其作用機制，mavorixafor 已被證明可以增加循環中性粒細胞的數量。

This ability is clearly stated in the mavorixafor label from the FDA approval in WHIM syndrome based on results from a pivotal Phase 3 trial. These results demonstrated that mavorixafor sustainably raised ANC over the 52 weeks of the trial and consequently reduced the annualized rate of infection along with the duration and severity of infections in trial participants. This infection benefit was achieved through an increase in mean ANC of more than 500 cells per microliter.

根據一項關鍵 3 期試驗的結果，FDA 批准用於治療 WHIM 症候群的 mavorixafor 標籤中明確說明了這種能力。這些結果表明，mavorixafor 在試驗的 52 週內可持續提高 ANC，從而降低了試驗參與者的年感染率以及感染的持續時間和嚴重程度。這種感染益處是透過每微昇平均 ANC 增加超過 500 個細胞來實現的。

These results and the WHIM approval in the US provide confidence in our plan to potentially bring mavorixafor to those with other chronic neutropenic conditions, where significant unmet needs remain. The gap in addressing these unmet needs results from the significant lack of innovation in the field.

這些結果和 WHIM 在美國的批准為我們的計劃提供了信心，該計劃有可能將 mavorixafor 帶給患有其他慢性中性粒細胞減少症的患者，這些患者的需求仍然存在重大未滿足。解決這些未滿足需求的差距是由於該領域嚴重缺乏創新所造成的。

The only currently available therapy is G-CSF, which was approved almost 30 years ago. It's an injectable drug that is associated with dose dependent and often treatment limiting side effects, as well as long-term risk of certain malignancy.

目前唯一可用的療法是 G-CSF，它在大約 30 年前獲得批准。它是一種注射藥物，與劑量依賴性、通常限制治療的副作用以及某些惡性腫瘤的長期風險有關。

As you've heard in our previous investor presentations, CN patients and the physicians that treat them are dissatisfied with G-CSF and its side effects, especially fatigue and bone and muscle pain. Unfortunately, these side effects are often dose dependent and can limit the use of G-CSF or prevent its use at a fully effective dose.

正如您在我們之前的投資者演講中所聽到的，CN 患者和治療他們的醫生對 G-CSF 及其副作用（尤其是疲勞、骨骼和肌肉疼痛）不滿意。不幸的是，這些副作用通常是劑量依賴性的，並且可能限制 G-CSF 的使用或阻止其以完全有效的劑量使用。

We believe there is an urgent need for a new innovation like mavorixafor, which data now suggests could be used, if approved as a monotherapy or in combination with G-CSF, while also allowing for reduced doses of G-CSF to limit side effects and long-term cancer risk.

我們認為，迫切需要像 mavorixafor 這樣的新創新，現在的數據表明，如果批准作為單一療法或與 G-CSF 聯合使用，則可以使用該創新，同時還可以減少 G-CSF 的劑量以限制副作用和長期癌症風險。

Our market research indicates there are approximately 50,000 people diagnosed with CN in the US. Of this population, it's estimated that about 15,000 have high unmet need, including recurrent infection despite the available standard of care. Our Phase 3 4WARD trial focuses on this population with the highest unmet need, enrolling adolescents and adults with ANC below 1,500 cells per microliter and a history of recurrent and, or serious infection.

我們的市場研究表明，美國大約有 50,000 人被診斷出患有 CN。據估計，在這一人群中，約有 15,000 人的需求未得到滿足，其中包括儘管有現有標準護理，但仍存在反覆感染的情況。我們的 3 期 4WARD 試驗重點關注未滿足需求最高的人群，招募 ANC 低於 1,500 個細胞/微升且有復發和/或嚴重感染史的青少年和成人。

To meet the multiple needs of this population, we envision two potential opportunities for oral once daily mavorixafor within this market. One, it could be used as a monotherapy in newly diagnosed CN patients or to replace G-CSF treatment. And two, in combination with G-CSF, enabling a meaningful reduction in G-CSF dosing, lessening pain, discomfort and long-term risk of malignancy.

為了滿足該族群的多種需求，我們設想了該市場中每日一次口服 mavorixafor 的兩個潛在機會。第一，它可以作為新診斷 CN 患者的單一療法或替代 G-CSF 治療。第二，與 G-CSF 結合，可以顯著減少 G-CSF 劑量，減輕疼痛、不適和長期惡性腫瘤風險。

Our Phase 2 CN study set out to evaluate those opportunities. And as you will see in just a minute, we believe the data from this study supports the potential for mavorixafor to transform the chronic care of this neutropenic population.

我們的第二階段 CN 研究旨在評估這些機會。正如您稍後將看到的，我們相信這項研究的數據支持 mavorixafor 改變中性粒細胞減少症人群長期護理的潛力。

As shown here, the main goal of the Phase 2 study were to confirm the durability of the Phase 1b results that showed 100% response to a single dose of oral mavorixafor plus or minus G-CSF across multiple CN types.

如圖所示，2 期研究的主要目標是確認 1b 期結果的持久性，該結果顯示，在多種 CN 類型中，單劑量口服 mavorixafor 加或減 G-CSF 有 100% 的反應。

We also aim to assess the long-term safety and tolerability of mavorixafor use with and without G-CSF in people with CN. In addition, we wanted to explore the feasibility and the willingness of physicians to safely reduce G-CSF with mavorixafor, and a key goal was also to inform the design of our Phase 3 pivotal trial and in doing so de-risk the trial.

我們也旨在評估 CN 患者在合併或不合併 G-CSF 的情況下使用 mavorixa 的長期安全性和耐受性。此外，我們希望探索醫生使用 mavorixafor 安全減少 G-CSF 的可行性和意願，一個關鍵目標也是為我們 3 期關鍵試驗的設計提供信息，並以此降低試驗風險。

The Phase 2 study was a six month study with monthly visits and comprehensive assessment at baseline in months one, three and six. Two quick notes on the design of this study. First, since the neutrophil life cycle is about 10 to 14 days. We believe that a study of this length provides a good indication of the bone marrow's long-term ability for stable neutrophil production.

第二階段研究是一項為期六個月的研究，每月進行一次訪問，並在第一、第三和六個月的基線進行全面評估。關於本研究設計的兩個簡短說明。首先，中性粒細胞的生命週期約為10至14天。我們相信，這種長度的研究可以很好地顯示骨髓穩定產生中性粒細胞的長期能力。

And second, modifications to G-CSF dosing were permitted in the combination group, following participants month two visit. So the first assessment of those on an adjusted dose would have been at the month three visit. These adjustments were decided between physicians and their patients and were not protocol mandated.

其次，在參與者兩個月就診後，聯合組允許修改 G-CSF 劑量。因此，對調整劑量的患者的第一次評估將在第三個月就診時進行。這些調整是由醫生和病人共同決定的，並不是協議規定的。

Let's start by looking at the final participant disposition in the study. A population typical of those with chronic neutropenia and high unmet need. We enrolled a total of 23 participants in the six month, single arm, open-label study with the mix of idiopathic and congenital CN and even a few with cyclic presentation. There was a typical gender distribution in the study and a mean age of 34 years.

讓我們先看看研究中最終參與者的分佈。患有慢性中性粒細胞減少症和高度未滿足需求的典型人群。我們總共招募了 23 名參與者參加為期 6 個月的單組開放標籤研究，其中包括特發性和先天性 CN，甚至還有一些患有週期性表現的參與者。研究中存在典型的性別分佈，平均年齡為 34 歲。

We also note here that there was a good mix of genetic backgrounds with the congenital group, also representative of what you might see across the full CN population. We analyzed the data in two groups, mavorixafor monotherapy comprising 10 participants, and the mavorixafor plus G-CSF group with 13 participants, 9 of whom ended up having their G-CSF dose reduced in the study.

我們也注意到，先天群體的遺傳背景有很好的混合，這也代表了你在整個 CN 群體中可能看到的情況。我們分析了兩組數據，mavorixafor 單藥治療組有 10 名參與者，mavorixafor 加 G-CSF 組有 13 名參與者，其中 9 名最終在研究中減少了 G-CSF 劑量。

In addition, we evaluated neutrophil functionality pre and post-treatment in a sub-study population of nine evaluable participants and compared those results to a group of healthy individuals. To address the unmet needs existing with current chronic CN therapy, our objective is to sustainably increase circulating functional neutrophils using oral mavorixafor. Thus, the results presented today are focused on answering the following four questions.

此外，我們評估了由九名可評估參與者組成的子研究族群治療前和治療後的中性粒細胞功能，並將這些結果與一組健康個體進行比較。為了解決目前慢性 CN 治療中存在的未滿足的需求，我們的目標是使用口服 mavorixafor 持續增加循環功能性嗜中性球。因此，今天提出的結果集中在回答以下四個問題。

One, in the monotherapy group, did mavorixafor increase and durably sustained absolute neutrophil count at clinically meaningful levels? Two, in the combination group, were physicians willing and able to adjust their patients G-CSF dose when adding mavorixafor? Three, could G-CSF be reduced while maintaining clinically meaningful ANC levels? And four, are the neutrophils that are mobilized into the peripheral blood by mavorixafor functional throughout the six month study?

第一，在單一治療組中，mavorixafor 是否增加了嗜中性球絕對數量並持久維持在有臨床意義的水平？第二，在合併治療組中，醫師在添加 mavorixafor 時是否願意並且能夠調整病患的 G-CSF 劑量？第三，能否在減少 G-CSF 的同時維持有臨床意義的 ANC 水準？第四，在整個六個月的研究中，mavorixafor 動員到週邊血液中的嗜中性球是否發揮作用？

First, let's look at the monotherapy results. Consistent with our data presentation in June, mavorixafor durably and meaningfully increased mean ANC, bringing levels into normal range at month three and month six. As an oral once-daily treatment delivering this level of ANC increase, you can begin to appreciate the potential for mavorixafor as monotherapy.

首先，讓我們來看看單一療法的結果。與我們 6 月的數據一致，mavorixafor 持久且有意義地增加了平均 ANC，使第三個月和第六個月的水平恢復到正常範圍。作為每日一次的口服治療，可提供這種水平的 ANC 增加，您可以開始認識到 mavorixafor 作為單一療法的潛力。

In addition, mavorixafor also durably and meaningfully increased mean ANC level in the severe CN study participants. Those starting with levels below 500 cells per microliter had more than twofold increase in mean ANC versus baseline throughout six months of mavorixafor treatment.

此外，mavorixafor 也持久且有意義地提高了嚴重 CN 研究參與者的平均 ANC 水平。在 mavorixafor 治療期間，那些起始水平低於 500 個細胞/微升的患者的平均 ANC 與基線相比增加了兩倍以上。

As a reminder, such an increase in ANC in our Phase 3 WHIM trial led to a decrease of 60% in infection rate. So this similar increase in ANC here is not only clinically meaningful, but also helps increase our confidence in achieving a positive infection rate readout for this monotherapy population in the ongoing pivotal Phase 3 trial in CN.

提醒一下，我們 3 期 WHIM 試驗中 ANC 的增加導致感染率下降了 60%。因此，ANC 的這種類似增加不僅具有臨床意義，而且有助於增強我們在 CN 正在進行的關鍵 3 期試驗中實現該單一療法人群的陽性感染率讀數的信心。

Let's move now to the combination results. In this group, we started with 13 participants entering the study on G-CSF at baseline. One dropped out almost immediately, so we had 12 eligible to have their G-CSF dose reduced following their month two visit. Notably, clinicians chose to reduce the G-CSF in 9 of these 12, with 3 being fully taken off G-CSF prior to their six month visit, finishing the study on mavorixafor monotherapy.

現在讓我們來看看組合結果。在該組中，我們從 13 名參與者開始進入基線 G-CSF 研究。其中一名患者幾乎立即退出，因此我們有 12 名患者有資格在第二個月就診後減少 G-CSF 劑量。值得注意的是，臨床醫師選擇減少這 12 例中 9 例的 G-CSF，其中 3 例在六個月就診前完全停用 G-CSF，完成 mavorixafor 單一藥物治療的研究。

Taking back to the market opportunity and unmet need slide, you can now see why we're so excited about the data. This is the first look at how physicians could potentially use mavorixafor in their CN patients who are already on G-CSF should it gain approval.

回到市場機會和未滿足的需求投影片，您現在可以明白為什麼我們對這些數據如此興奮。這是首次探討如果 Mavorixafor 獲得批准，醫生如何在已經接受 G-CSF 治療的 CN 患者中使用 mavorixafor。

Today, we're not presenting the data from the three participants who remain on stable dose G-CSF. But we know that the final data set from this group was consistent with the results we shared this past June, showing robust and sustained increases in mean ANC from baseline levels.

今天，我們不會提供繼續使用穩定劑量 G-CSF 的三位參與者的數據。但我們知道，該組的最終數據集與我們今年 6 月分享的結果一致，顯示平均 ANC 較基線水平強勁且持續增長。

Let's look now only at participants who were dose adjusted. As shown here, the G-CSF reductions were quite substantial, and mean ANC were able to be maintained at normal levels throughout the study. At month three, where eight of the nine have been adjusted, physicians were comfortable lowering G-CSF by 52% on average. This average increased to 70% reductions by month six, with three of the nine adjusted dose participants taken completely off G-CSF.

現在讓我們只看調整劑量的參與者。如圖所示，G-CSF 減少量相當大，並且在整個研究過程中平均 ANC 能夠維持在正常水平。在第三個月，九個中的八個已被調整，醫生對 G-CSF 平均降低 52% 感到滿意。到第六個月，這一平均減少量增加到 70%，九名調整劑量的參與者中有三名完全停止使用 G-CSF。

Anecdotally, physicians and academic experts have told us that they believe a 25% reduction in G-CSF would be clinically meaningful to their patients. So seeing this level of G-CSF reduction reinforces the potential benefit of mavorixafor and role in the treatment of CN.

有趣的是，醫生和學術專家告訴我們，他們相信 G-CSF 減少 25% 對病人來說具有臨床意義。因此，看到這種程度的 G-CSF 減少強化了 mavorixafor 的潛在益處及其在 CN 治療中的作用。

We now turn our attention to assessing the functionality of the increased levels of circulating neutrophil in the Phase 2 sub-study. As mentioned, the purpose of this sub-study was to assess the percentage of functional neutrophils in people with CN, including those with congenital genetic variations associated with neutrophil maturation arrest.

我們現在將注意力轉向評估第二階段子研究中循環中性粒細胞水平增加的功能。如前所述，這項子研究的目的是評估 CN 患者中功能性嗜中性球的百分比，包括那些具有與嗜中性球成熟停滯相關的先天性遺傳變異的患者。

We used two well accepted functionality assays. The phagocytosis assay, which assesses the neutrophil's ability to engulf pathogens, and an assay for ROS production. ROS is reactive oxygen species or the ability to damage or kill pathogens that have been engulfed.

我們使用了兩種廣為接受的功能測定。吞噬作用測定，評估嗜中性球吞噬病原體的能力，以及ROS產生的測定。ROS 是活性氧或破壞或殺死已被吞噬的病原體的能力。

These neutrophil function studies were conducted on the sample from nine eligible participants with a mix of CN types and monotherapy and combination use. We also recruited five healthy individuals to provide a benchmark for comparison.

這些中性粒細胞功能研究是對九名合格參與者的樣本進行的，混合使用 CN 類型、單一療法和聯合療法。我們也招募了五位健康人作為比較基準。

For simplicity today, we are only showing the phagocytosis data, but we note that the ROS assay results closely mirrored the phagocytosis results. While participants with chronic neutropenia have fewer neutrophils than healthy donors, you can see here that those neutrophils that do make it into the circulation are, in fact, functional, with a similar percent of functionality between the neutropenic and healthy donor population.

為了簡單起見，今天我們僅顯示吞噬數據，但我們注意到 ROS 測定結果密切反映了吞噬結果。雖然患有慢性中性粒細胞減少症的參與者的中性粒細胞比健康捐贈者少，但您可以在此處看到，那些確實進入循環的中性粒細胞實際上是有功能的，中性粒細胞減少症和健康捐贈者群體之間的功能百分比相似。

Note that the bars here represent the percentage of neutrophils that demonstrated phagocytosis after challenge with opsonized bacteria. We've also broken out the congenital CN subset to see if genetic variant affects functionality differently than in the other groups.

請注意，此處的條形代表在用調理細菌攻擊後表現出吞噬作用的中性粒細胞的百分比。我們也對先天性 CN 子集進行了分析，看看遺傳變異對功能的影響是否與其他群體不同。

Looking now at the full results, we see that the percentage of functional neutrophil in the healthy donors, the full group of participants and the congenital CN subset are essentially the same following six months of mavorixafor therapy. And note, while we are showing you here the data for fibrocystic function, we generated very similar data on the percentage of neutrophils generating ROS after bacterial challenge.

現在查看完整的結果，我們發現在接受 6 個月的 mavorixafor 治療後，健康捐贈者、整個參與者組和先天性 CN 亞群中功能性中性粒細胞的百分比基本上相同。請注意，雖然我們在這裡向您展示纖維囊性功能的數據，但我們產生了關於細菌攻擊後產生 ROS 的中性粒細胞百分比的非常相似的數據。

Recall in our 52 week WHIM Phase 3 clinical trial, we saw that an increase in circulating mature neutrophils corresponded with a 60% reduction in the annualized infection rate as well as reduction in the severity and duration of infection.

回想一下，在我們為期 52 週的 WHIM 3 期臨床試驗中，我們發現循環成熟嗜中性球的增加與年化感染率降低 60% 以及感染嚴重程度和持續時間的降低相對應。

By increasing the number of functional circulating neutrophils, we believe that mavorixafor treatment could correspondingly reduce the infection rate in the larger chronic neutropenia population. And we are hopeful to see similar results in our ongoing registrational Phase 3 chronic neutropenia trial.

透過增加功能性循環中性粒細胞的數量，我們相信 mavorixafor 治療可以相應降低較大慢性中性粒細胞減少症人群的感染率。我們希望在正在進行的註冊 3 期慢性中性粒細胞減少症試驗中看到類似的結果。

Mavorixafor with or without concurrent G-CSF therapy was generally well tolerated throughout the study. The overall safety profile was consistent with previous studies and the most frequent treatment-related, treatment emergent AEs were GI-related, nausea and diarrhea, which were mild to moderate and typically resolved over time.

在整個研究過程中，無論是否同時進行 G-CSF 治療，Mavorixafor 的耐受性都普遍良好。整體安全性與先前的研究一致，最常見的治療相關、治療中出現的不良事件是胃腸道相關、噁心和腹瀉，這些不良事件是輕度至中度，通常會隨著時間的推移而消失。

As we noted in our June presentation, we did see three discontinuations in the study. These occurred early in the execution of the study. And after implementing an education program on possible GI side effects, there were no further discontinuation. Rest assured, we have incorporated this education into the Phase 3 CN trial.

正如我們在 6 月的報告中指出的那樣，我們確實看到了該研究的三個中斷。這些發生在研究執行的早期。在實施了有關可能的胃腸道副作用的教育計劃後，沒有進一步停藥。請放心，我們已將此教育納入 CN 3 期試驗中。

In summary, we could not be more pleased with the results of this Phase 2 study having met all of the goals we set forth. We have shown that mavorixafor can durably and meaningfully increase mean ANC as a monotherapy, even in the most severe CN population and can do likewise when dosed in combination with G-CSF. We show that mavorixafor enabled clinicians and their patients to meaningfully lower the use of G-CSF while maintaining mean ANC at normal levels.

總之，我們對第二階段研究的結果感到非常滿意，它達到了我們設定的所有目標。我們已經證明，即使在最嚴重的 CN 族群中，mavorixafor 作為單一療法也可以持久且有意義地增加平均 ANC，並且與 G-CSF 聯合用藥時也能起到同樣的作用。我們表明，mavorixafor 使臨床醫生及其患者能夠有意義地降低 G-CSF 的使用，同時將平均 ANC 維持在正常水平。

Critically, we showed that mavorixafor increased total count of functional circulating neutrophils even in the most difficult to treat individuals with CN. And importantly, we demonstrated that mavorixafor was generally well tolerated on its own and in combination with G-CSF. These results give us great confidence in the chance of success of our ongoing Phase 3 trial and mavorixafor's potential to reduce infection rate.

至關重要的是，我們發現，即使在最難治療的 CN 患者中，mavorixafor 也能增加功能性循環中性粒細胞的總數。重要的是，我們證明 mavorixafor 單獨使用以及與 G-CSF 聯合使用通常具有良好的耐受性。這些結果讓我們對正在進行的 3 期試驗的成功機會以及 mavorixafor 降低感染率的潛力充滿信心。

They also provide hope that mavorixafor could be the innovation that the CN community is looking for. As you've heard previously, through our interviews with physicians and CN patients, what they need and want is a well tolerated oral option, one that can enable them to safely reduce the use of injectable G-CSF. And so far, our results have shown that mavorixafor fits that profile well as either a monotherapy or in combination use.

他們也希望 mavorixafor 能夠成​​為 CN 社群正在尋找的創新。正如您之前所聽到的，透過我們對醫生和 CN 患者的採訪，他們需要和想要的是一種耐受性良好的口服選擇，使他們能夠安全地減少注射 G-CSF 的使用。到目前為止，我們的結果表明，mavorixafor 無論是作為單一療法還是聯合使用都非常符合這項要求。

I'll now turn it back to Paula to conclude. Paula?

現在我將把它轉回保拉來結束。保拉？

Thanks so much, Christophe. Great job sharing our exciting update on mavorixafor's potential in CN. To conclude, over the last six months, we have made significant progress as a company in bringing innovation to the immunodeficiency community.

非常感謝，克里斯托夫。非常高興分享我們關於 mavorixafor 在中國的潛力的令人興奮的最新消息。總而言之，在過去的六個月裡，我們作為一家公司在為免疫缺陷社群帶來創新方面取得了重大進展。

By gaining approval for and launching XOLREMDI in the US for WHIM syndrome and by exploring additional geographies for mavorixafor's approval and distribution in WHIM, we are seeking to maximize the global opportunity to help those with WHIM.

透過在美國批准並推出用於治療 WHIM 綜合徵的 XOLREMDI，以及探索 mavorixafor 在 WHIM 中的批准和分銷的其他地區，我們正在尋求最大限度地利用全球機會來幫助 WHIM 患者。

And by completing and reporting out these positive Phase 2 results and initiating our pivotal Phase 3 trial of mavorixafor in those with chronic neutropenia, we are seeking to further expand the market opportunity for mavorixafor and have a positive impact on a much larger potential patient population.

透過完成並報告這些積極的2 期結果並啟動mavorixafor 在慢性中性粒細胞減少症患者中的關鍵3 期試驗，我們正在尋求進一步擴大mavorixafor 的市場機會，並對更大的潛在患者群體產生積極影響。

In terms of our financial results, I direct you to our press release from this morning and our 10-Q to be issued shortly. We ended the third quarter of 2024 with cash and equivalents of almost $136 million. We continue to believe this gives us the runway into late 2025, and note that this does not include the expected ramp up of sales of XOLREMDI throughout next year.

關於我們的財務業績，我請您閱讀我們今天上午的新聞稿以及即將發布的 10-Q 報告。截至 2024 年第三季末，我們的現金及等價物接近 1.36 億美元。我們仍然相信這為我們提供了到 2025 年底的機會，並注意到這不包括 XOLREMDI 明年全年銷售的預期成長。

As always, we thank you for your continued support and would now be happy to take your questions. Operator?

一如既往，我們感謝您的持續支持，現在很樂意回答您的問題。操作員？

Thank you. (Operator instructions)

謝謝。（操作員說明）

Kristen Kluska, Cantor.

克里斯汀·克魯斯卡，坎托。

Hi, good morning, everybody. Congrats on these new great data you shared with us. Really appreciate it. So questions that I have are related to the new data today. So I wanted to ask what the biggest criteria were in the trial that made the physicians comfortable to reduce and lower the G-CSF usage? And while I recognize it wasn't a formal endpoint measured, I'm curious if the doctors had any anecdotes to share about any early signals of reduced pain or better convenience benefits for patients.

嗨，大家早安。恭喜您與我們分享了這些新的重要數據。真的很感激。所以我的問題與今天的新數據有關。所以我想問一下，試驗中讓醫生放心減少和降低 G-CSF 使用量的最大標準是什麼？雖然我意識到這不是一個正式的測量終點，但我很好奇醫生是否有任何軼事可以分享，以減輕疼痛或為患者帶來更好的便利好處的任何早期信號。

Yes, Kristen, thank you for the question. I'll start and then Christophe will add some color. So I mean the important thing about the Phase 2 study is we really put the choice in the physicians and patients hands. So there's a wide variety of patients. They have real world experience with them.

是的，克里斯汀，謝謝你的提問。我先開始，然後 Christophe 會添加一些顏色。所以我的意思是，第二階段研究的重要之處在於我們真正將選擇權交給了醫生和病人。所以，患者的種類很多。他們擁有現實世界的經驗。

So really, what gave them comfort to adjust G-CSF dosing was the high and robust ANC responses due to the addition of mavorixafor. That is confidence in data, and of course, they acted on that. But Christophe, why don't you give some more color on that?

事實上，讓他們放心調整 G-CSF 劑量的是由於添加 mavorixafor 而產生的高而強勁的 ANC 反應。這就是對數據的信心，當然，他們也據此採取了行動。但是克里斯托夫，為什麼不對此給予更多的色彩呢？

Sure. Yes, no, we are really happy with the results and the choice that 75% of those physicians with their patients have decided to decrease G-CSF. This is a really high percentage of those decisions without any mandates from the protocol or any prescriptions that we ask them to do this.

當然。是的，不，我們對結果以及 75% 的醫生及其患者決定減少 G-CSF 的選擇感到非常滿意。在沒有任何協議授權或我們要求他們這樣做的任何處方的情況下，這些決定的比例非常高。

With regard to the choice to do it is, as mentioned, a choice between patients and the physician. And it's a balance between the level of ANC, as Paula just mentioned. And so they were very comfortable with the level of ANC and the clinical manifestations that these patients were having. So we could not identify a specific pattern, but this is really what drives most of those decisions.

如同前面所提到的，關於選擇這樣做，是病人和醫生之間的選擇。正如 Paula 剛才提到的，這是 ANC 水平之間的平衡。因此，他們對 ANC 水平和這些患者的臨床表現非常滿意。因此，我們無法確定特定的模式，但這確實是大多數決策的驅動因素。

Okay. Thanks. And then any early anecdotes or it's a little too early to make that assessment?

好的。謝謝。還有什麼早期的軼事嗎？

I think it's early to make that assessment. And of course, what we're most pleased with is the broad applicability, the broad reduction in G-CSF across the 75% of patients and even some of them completely coming off. So certainly, a lot more study to be done, Kristen, but thanks for the highlights.

我認為現在做出這樣的評估還為時過早。當然，我們最滿意的是其廣泛的適用性，75% 的患者 G-CSF 廣泛減少，甚至其中一些完全消失。當然，克里斯汀，還有很多研究要做，但感謝您的重點。

Yes. And if I may ask one more question then. Can you talk a little bit more in terms of what lower G-CSF usage actually means? Is it that physicians are assessing it every other day for usage? Is it the amount of G-CSF that's administered is lowered? And then do we have a general sense to give us whether, what correlation of G-CSF usage could ultimately like truly lead to alleviating some of the dangerous side effects and the inconvenient ones?

是的。那我可以再問一個問題嗎？您能否多談談 G-CSF 使用率降低的實際意義？醫生是否每隔一天評估一次它的使用情況？是 G-CSF 的給藥量減少了嗎？然後，我們是否有一個普遍的認知來告訴我們，G-CSF 使用的相關性是否最終能夠真正減輕一些危險的副作用和不便的副作用？

Yes. So we'll take the second part first. So what we've been reporting on from patients and physicians in some of our early research is that about 25% reduction either in dose and, or frequency seems to be the threshold for something that's clinically meaningful for them. And obviously, the lower, the better in terms of the short and long-term toxicities experienced by dosing.

是的。所以我們先來看第二部分。因此，我們在一些早期研究中從患者和醫生那裡得到的報告是，劑量和/或頻率減少約 25% 似乎是對他們有臨床意義的閾值。顯然，就給藥所經歷的短期和長期毒性而言，該值越低越好。

And then just in terms of percent reductions, that's always based off of the patient's entry criteria. The challenge with G-CSF is the heterogeneity of each patient is different. So we treated them as such as clinicians do. So that reduction reflects their entry, changes in their entry baseline levels.

然後就減少百分比而言，這始終基於患者的進入標準。G-CSF 面臨的挑戰是每個患者的異質性都不同。所以我們像臨床醫生一樣對待他們。因此，這種減少反映了他們的進入，以及他們進入基線水平的變化。

Thanks again and congratulations.

再次感謝並恭喜。

Thank you.

謝謝。

Ted Tenzo, Piper Sandler.

特德·坦佐，派珀·桑德勒。

Okay. Thank you very much. Good morning everyone and congrats on the Phase 2 data. I had one quick follow-up question. When it comes to the Phase 3 trial, and I'm wondering how, is there a way or are you looking at benefits, kind of following up on the last question from G-CSF reduction? Is there any way to capture those in terms of improved tolerability, improved safety? And then I have a quick one on XOLREMDI.

好的。非常感謝。大家早安，祝賀第二階段的數據。我有一個快速的後續問題。當談到第 3 階段試驗時，我想知道如何，有沒有辦法或您是否正在考慮好處，有點跟進 G-CSF 減少的最後一個問題？有沒有什麼方法可以提高耐受性和安全性？然後我就 XOLREMDI 進行了快速介紹。

Sure. I'll turn that over to Christophe.

當然。我會把它交給克里斯托夫。

Yes, no, so the Phase 3 trial is designed as a registration approval trial as per the FDA requirements. So we will be looking at those aspects as a follow-on study to that Phase 3 trial.

是的，不是，所以第 3 期試驗被設計為根據 FDA 要求的註冊批准試驗。因此，我們將把這些面向作為第三階段試驗的後續研究。

Right. Okay. Yes, that makes a lot of sense. And also, I have to imagine it might take some time for some of those to emerge, but yes, that's great. And then when it comes to XOLREMDI, you guys announced a new initiative. Can you just provide a little bit more detail around that in terms of reaching out to the patients?

正確的。好的。是的，這很有意義。而且，我必須想像其中一些可能需要一些時間才能出現，但是是的，這很棒。然後，當談到 XOLREMDI 時，你們宣布了一項新舉措。您能否在接觸患者方面提供更多細節？

Sure. Ted, just to clarify, a little bit more color on our sort of patient ambassador efforts?

當然。特德，只是為了澄清一下，對我們耐心大使的努力有更多的了解嗎？

Yes. Thank you.

是的。謝謝。

Sure. Mark?

當然。標記？

Yes. Thanks, Ted. And you're right. We're laser focused right now on raising disease awareness and driving screening for WHIM and presenting XOLREMDI as a treatment that has proven to work in WHIM. So we've just launched a new patient directed campaign.

是的。謝謝，泰德。你是對的。我們現在專注於提高疾病意識和推動 WHIM 篩檢，並推出 XOLREMDI 作為一種已被證明對 WHIM 有效的治療方法。因此，我們剛剛發起了一項新的以患者為導向的活動。

This is a combination of digital campaigns with websites as well as hard copy material that we can provide to patients. And we think this will help to educate patients and encourage them to go and have a discussion with their physician about WHIM syndrome.

這是數位活動與網站以及我們可以向患者提供的硬拷貝材料的結合。我們認為這將有助於教育患者並鼓勵他們與醫生討論 WHIM 綜合徵。

Yes, great. Excellent. Well, thank you very much. Appreciate the time.

是的，太好了。出色的。嗯，非常感謝。珍惜時間。

Thanks so much, Tim.

非常感謝，提姆。

RK with HCW.

RK 與醫護人員。

Thank you. Good morning, Paula and team. I just want to focus a little bit on XOLREMDI and the uptake in the market. Can you educate us on how many patients are on the drug? And how, what is the commercialization setup right now? And I'm just trying to understand how long do you think it would take for the drug to be adopted in a decent fashion.

謝謝。早上好，寶拉和團隊。我只想稍微關心一下 XOLREMDI 和市場的採用情況。您能告訴我們有多少患者正在服用這種藥物嗎？目前的商業化設定如何？我只是想了解您認為這種藥物需要多長時間才能得到適當的採用。

Thanks, RK. I'll turn it over to Mark.

謝謝，RK。我會把它交給馬克。

Yes. Thanks, RK. As I mentioned, we are right now laser focused on raising disease awareness and driving that screening for WHIM, encouraging physicians to look for WHIM patients in their practice, helping them recognize these patients in their practice. Up to now, there's been no approved treatment for this disease.

是的。謝謝，RK。正如我所提到的，我們現在專注於提高疾病意識並推動 WHIM 篩檢，鼓勵醫生在實踐中尋找 WHIM 患者，幫助他們在實踐中識別這些患者。到目前為止，還沒有批准治療這種疾病的方法。

So everything starts with raising disease awareness and encouraging physicians to identify the patients in their practice. And then we have a discussion with them about XOLREMDI, and we share the data that shows XOLREMDI works. So that's really where we are now. And it's building the foundation for our ramping up into 2025, and we'll be able to share more details with you then.

因此，一切都始於提高疾病意識並鼓勵醫生在實踐中識別患者。然後我們與他們討論 XOLREMDI，並分享顯示 XOLREMDI 有效的資料。這就是我們現在的處境。它為我們邁向 2025 年奠定了基礎，屆時我們將能夠與您分享更多詳細資訊。

Thank you. Thanks for taking my question.

謝謝。感謝您提出我的問題。

Steven Willey, Stifel.

史蒂文威利，斯蒂菲爾。

Yeah, good morning. Thanks for taking the questions. Maybe one on the data, one on XOLREMDI. So I guess on the data side, was there any correlation between CN subtype and the rapidity and magnitude of G-CSF dose reduction? And then I guess, was there also any correlation between the G-CSF dose these patients were receiving at baseline and then the magnitude of the dose reduction they were able to achieve?

是的，早安。感謝您提出問題。也許一項關於數據，一項關於 XOLREMDI。所以我想從數據角度來看，CN 亞型與 G-CSF 劑量減少的速度和幅度之間是否有任何相關性？然後我想，這些患者在基線時接受的 G-CSF 劑量與他們能夠實現的劑量減少幅度之間是否也存在任何相關性？

Sure. I'll start and then Christophe will provide more color. But in terms of the CN subtype, the good news is the drug seems to be robustly and consistently working. We did break out the severity of those patients because those are the toughest to treat, and we saw a robust response there. But in terms of CIN or congenital, we're seeing a very consistent effect.

當然。我先開始，然後 Christophe 提供更多顏色。但就 CN 亞型而言，好消息是該藥物似乎具有強大且持續的作用。我們確實列出了這些患者的嚴重程度，因為這些患者是最難治療的，我們看到了那裡的強烈反應。但就 CIN 或先天性而言，我們看到了非常一致的效果。

In terms of G-CSF percent reduction from their baseline, again, we're not really seeing any variability in response based on their G-CSF profiles. But I know Christophe has had a lot of data analysis, and Christophe can provide some more color there.

就 G-CSF 相對於基線的減少百分比而言，我們並沒有真正看到基於他們的 G-CSF 概況的任何反應變化。但我知道 Christophe 有很多數據分析，Christophe 可以在那裡提供更多的顏色。

Yes. No, thank you. So the CN Phase 2, the sample size and the number of patients in that study is too small to start drawing directions where we're going with the different type of patients. So we know that in all of the type of patients that were included in the Phase 2 study decrease, any type of those patients decreased G-CSF and chose to decrease G-CSF voluntarily.

是的。不，謝謝。因此，CN 2 期研究中的樣本量和患者數量太小，無法開始為不同類型的患者制定方向。所以我們知道，在 2 期研究中納入的所有類型的患者中，任何類型的患者都會減少 G-CSF，並選擇自願減少 G-CSF。

That, we know for sure. The Phase 3 trial will help us in having that bigger picture of how those different subtypes, the congenital, the different mutations in the congenital, et cetera, all this will be analyzed in the Phase 3 study with much more detail.

對此，我們確信無疑。第三階段試驗將幫助我們更全面地了解這些不同的亞型、先天性、先天性的不同突變等，所有這些都將在第三階段研究中進行更詳細的分析。

Okay. And then on XOLREMDI, I know you've hit 100% of your target accounts. I think you also mentioned 60% of HCPs have increased their screening. But I guess, do you have a sense of how many of these HCPs that you're targeting are indeed actively screening? Just wondering, I guess, how low of a base you're starting at here and what kind of runway you might have? Thanks.

好的。然後在 XOLREMDI 上，我知道您已達到 100% 的目標帳戶。我想您也提到 60% 的 HCP 增加了篩檢。但我想，您是否知道您所針對的這些 HCP 中有多少人確實在積極篩選？我想只是想知道你從這裡開始的基地有多低以及你可能擁有什麼樣的跑道？謝謝。

Yes. So again, the way we identify physicians that we think have a WHIM patient in their practice is really using claims analysis and our market research. And these are the physicians that we're going out to really make them aware of WHIM syndrome and encourage them to start screening for the patients in their practice. And this is really where we're getting the most engagement at this point.

是的。再說一次，我們識別在實踐中擁有突發奇想患者的醫生的方式實際上是使用索賠分析和我們的市場研究。我們要讓這些醫生真正了解心血來潮綜合症，並鼓勵他們在實踐中開始對患者進行篩檢。這確實是我們目前參與度最高的地方。

And the other thing that we're really trying to do is reach the physicians where they are. And so we've really doubled down on our conference presence this year. As you know, these physicians come from different specialties. So we mainly focus on hematology and immunology and try to really meet those physicians where they are.

我們真正想做的另一件事是聯繫醫生。因此，今年我們確實加倍了參加會議的強度。如您所知，這些醫生來自不同的專業。因此，我們主要關注血液學和免疫學，並嘗試真正與這些醫生見面。

All right. Thanks for taking the questions.

好的。感謝您提出問題。

David Bautz, Zachs.

大衛鮑茨，扎克斯。

Hey, good morning, everyone. For XOLREMDI, I'm curious if you will be able to provide any type of sales guidance numbers moving into 2025. And then for the new Phase 2 data this morning, I realize the numbers are small, but was there any data collected on infections, number of infections or types and then particularly for those patients where G-CSF was reduced?

嘿，大家早安。對於 XOLREMDI，我很好奇您是否能夠提供進入 2025 年的任何類型的銷售指導數字。然後，對於今天早上新的第二階段數據，我意識到數字很小，但是否收集了有關感染、感染數量或類型的數據，特別是對於那些 G-CSF 減少的患者？

Sure. So sales guidance for 2025, we're not yet providing that. We certainly, as Mark mentioned, we'll provide some more robust insights into our commercial launch as we head into the year. In terms of infection data, I mean, as you can appreciate, this is a Phase 2 study.

當然。因此，我們尚未提供 2025 年的銷售指導。當然，正如馬克所提到的，我們將在今年進入商業發佈時提供一些更強有力的見解。就感染數據而言，我的意思是，正如您所理解的，這是一項第二階段研究。

It was not designed to assess infection rates, no randomized comparator or comparator arm, no history collected. Importantly, though, we'd really like to remind everybody, in this population, ANC is proven to correlate with infection risk that is based on the G-CSF clinical trials and everyday use. So we're extremely pleased that with the fact that our ANCs are robustly increasing and the neutrophils are functional, we've actually gone one step further beyond the data with G-CSF in our own study.

它的目的不是評估感染率，沒有隨機比較或比較組，也沒有收集病史。但重要的是，我們真的想提醒大家，在這個人群中，基於 G-CSF 臨床試驗和日常使用，ANC 被證明與感染風險有關。因此，我們非常高興地看到，我們的 ANC 正在強勁增長，中性粒細胞也具有功能性，我們實際上比我們自己的研究中 G-CSF 的數據更進一步。

So we really hope we can remind everybody as it relates to infections, of course, that was part of our safety database, which will be published at a future point. But really, we're incredibly excited about the totality of evidence and are locked and loaded for the Phase 3.

因此，我們真的希望能夠提醒大家，因為它與感染有關，當然，這是我們安全資料庫的一部分，該資料庫將在將來發布。但實際上，我們對全部證據感到非常興奮，並已鎖定並加載第三階段。

Okay, great. Thanks for taking the questions.

好的，太好了。感謝您提出問題。

Thank you. And this concludes our Q&A session. I will now turn the call over to Paula Ragan for closing comments.

謝謝。我們的問答環節到此結束。我現在將把電話轉給保拉·拉根 (Paula Ragan) 徵求結束意見。

Well, thank you so much for joining us on the call today. As you can appreciate, we are very excited about these data and we are on track to deliver value. So from all of us at X4, stay classy and have a great day.

非常感謝您今天加入我們的電話會議。正如您所理解的，我們對這些數據感到非常興奮，我們有望實現價值。因此，X4 的所有人，請保持優雅並祝您度過愉快的一天。

And this does conclude today's program. Thank you for your participation. You may disconnect at any time.

今天的節目到此結束。感謝您的參與。您可以隨時斷開連線。