X4 Pharmaceuticals Inc (XFOR) 2024 Q2 法說會逐字稿

Good morning, everyone, and welcome to X4 Pharmaceuticals Second Quarter 2024 Earnings Conference Call. (Operator Instructions) Also, today's call is being recorded. (Operator Instructions)

大家早安，歡迎參加 X4 Pharmaceuticals 2024 年第二季財報電話會議。（操作員說明）此外，今天的通話正在錄音。（操作員說明）

It is now my pleasure to introduce your host, Mr. Dan Ferry from Lifesci Advisors. Dan, please go ahead.

現在我很高興向您介紹主持人，來自 Lifesci Advisors 的 Dan Ferry 先生。丹，請繼續。

Thank you, operator, and good morning, everyone. Thank you for joining us today. Presenting on today's call will be Dr. Paula Ragan, X4's President and CEO; and the company's Chief Financial Officer, Adam Mostafa. Following prepared remarks, we will open up the call to your questions and we'll be joined by Chief Commercial Officer, Mark Baldry; Chief Medical Officer, Dr. Christophe Arbet-Engels; Chief Operating Officer, Dr. Mary DiBiase; Chief Scientific Officer, Dr. Art Taveras; and José Juves, Head of Corporate and Patient Affairs.

謝謝接線員，大家早安。感謝您今天加入我們。X4 總裁兼執行長 Paula Ragan 博士將出席今天的電話會議。以及公司財務長 Adam Mostafa。在準備好發言後，我們將開始電話詢問您的問題，首席商務官馬克·鮑德里（Mark Ba​​ldry）也將加入我們的行列。首席醫療官 Christophe Arbet-Engels 博士；營運長 Mary DiBiase 博士；首席科學官 Art Taveras 博士； José Juves，公司和病患事務主管。

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. 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 year's Form 10-K, which was filed on March 21, 2024, and in the company's Form 10-Q for the second quarter, which is expected to be filed later today.

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。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，以及該公司預計第二季的 10-Q 表格。 。

I'll now turn it over to Paula Ragan. Paula?

現在我將把它交給保拉·拉根 (Paula Ragan)。保拉？

Thanks so much, Dan, and welcome, everyone. I'll just start by saying what an incredible year we've had so far. Just a few weeks ago, we celebrated the tenth anniversary of X4. When we founded the company a decade ago, a small group of us had a vision to advance our lead asset, an orally active CXCR4 antagonist called mavorixafor, to help those with rare diseases who have few to no treatment options.

非常感謝丹，歡迎大家。首先我要說的是，到目前為止，我們度過了多麼令人難以置信的一年。就在幾週前，我們慶祝了 X4 十週年。十年前，當我們創立公司時，我們一小群人的願景是推進我們的主導資產——一種名為 mavorixafor 的口服活性 CXCR4 拮抗劑，以幫助那些幾乎沒有治療選擇的罕見疾病患者。

As you saw this past April, we were able to realize this vision, receiving US approval of mavorixafor, branded as XOLREMDI for the treatment of WHIM syndrome, a rare primary immunodeficiency. The US launch of XOLREMDI is now underway, and our full commercial team is in place and WHIM patients 12 years and older are now being treated with the only approved therapy targeting the underlying cause of their disease. Sales guidance on XOLREMDI will come at a future point. However, today, I'd like to highlight our strong launch progress.

正如您在去年四月看到的那樣，我們能夠實現這一願景，mavorixafor 獲得美國批准，品牌為 XOLREMDI，用於治療 WHIM 綜合徵（一種罕見的原發性免疫缺陷病）。XOLREMDI 在美國的上市正在進行中，我們完整的商業團隊已就位，12 歲及以上的 WHIM 患者現在正在接受唯一經批准的針對其疾病根本原因的療法的治療。XOLREMDI 的銷售指南將在未來發布。然而，今天，我想強調一下我們在發布過程中取得的強勁進展。

Overall, we've been very pleased with the launch to date. Our launch meeting in early May was a great success, where we completed the training and education of our field teams, enabling them to hit the ground running. We got product into our distribution channel very quickly and had our first patients on commercial drug within just a few weeks of product approval.

總的來說，我們對迄今為止的發布感到非常滿意。我們五月初的啟動會議取得了巨大成功，我們完成了現場團隊的培訓和教育，使他們能夠立即投入工作。我們很快就將產品進入我們的分銷管道，並在產品批准後短短幾週內就讓我們的第一批患者接受了商業藥物。

Coordination with our specialty pharmacy, PANTHERx Rare, has gone smoothly, and we've already received positive feedback on the patient support services provided through our X4Connect and nurse educator programs. And very encouragingly, reimbursement decisions have been coming through quickly, with almost all patients currently on therapy receiving reimbursement via prior authorizations and medical exceptions.

與我們的專業藥房 PANTHERx Rare 的協調進展順利，我們已經收到了有關透過 X4Connect 和護理教育計劃提供的患者支援服務的積極反饋。非常令人鼓舞的是，報銷決定很快就通過了，幾乎所有目前接受治療的患者都透過事先授權和醫療例外獲得報銷。

Our commercial team is fully deployed across the US, calling on key hematologists and immunologists. To date, there has been limited ability to establish centers of excellence for the WHIM community. We knew this going in and therefore, are continuing to execute on our disease awareness and education campaigns as well as leveraging our strong relationships with patient advocacy groups.

我們的商業團隊已全面部署在美國各地，召集了重要的血液學家和免疫學家。迄今為止，為 WHIM 社區建立卓越中心的能力有限。我們知道這一點，因此將繼續進行我們的疾病意識和教育活動，並利用我們與患者倡導團體的牢固關係。

We recently participated in the annual meeting of the Immune Deficiency Foundation or IDF, where we engage with physicians, patients and advocates. And members of our team visited with the Jeffrey Modell Foundation in New York just a few weeks ago. Through all of these efforts, we have become better at engaging with the treating physicians, understanding physician and patient questions and most importantly, building a community of interest around WHIM syndrome across the US.

我們最近參加了免疫缺陷基金會（IDF）的年會，與醫生、患者和倡導者進行了交流。幾週前，我們團隊的成員拜訪了位於紐約的 Jeffrey Modell 基金會。透過所有這些努力，我們能夠更好地與治療醫生互動，了解醫生和患者的問題，最重要的是，在美國各地建立一個圍繞 WHIM 綜合徵的興趣社區。

We also continue to leverage the positive data from our pivotal Phase 3 trial in WHIM that were published in April in Blood, The Journal of the American Society of Hematology. These and new data from the trial and its open-label extension phase were presented at the annual meeting of the Clinical Immunological Society, or CIS, in early May. As we reported at the time, the CIS poster revealed that longer-term treatment with XOLREMDI was associated with durable improvements in neutrophil and lymphocyte counts as well as reductions in annualized infection rates. And that no new safety signals have been observed during the OLE phase of the trial with some participants treated for more than two years.

我們也持續利用 WHIM 關鍵 3 期試驗的正面數據，這些數據於 4 月發表在《美國血液學會雜誌 Blood》上。這些數據以及該試驗及其開放標籤擴展階段的新數據已在 5 月初的臨床免疫學會 (CIS) 年會上公佈。正如我們當時報導的那樣，CIS 海報顯示，XOLREMDI 的長期治療與中性粒細胞和淋巴細胞計數的持久改善以及年感染率的降低有關。在 OLE 試驗階段，一些參與者接受了兩年多的治療，沒有觀察到新的安全訊號。

We also made tremendous progress this past quarter in our development of mavorixafor for those with chronic neutropenia, another rare immunodeficiency. At our investor event in late June, we presented positive interim clinical data from our ongoing Phase 2 clinical trial, demonstrating that once-daily oral mavorixafor was generally well tolerated and durably increased participants' absolute neutrophil counts, or ANC, both as a monotherapy and in combination with stable doses of injectable granulocyte colony-stimulating factor, or G-CSF, up to as long as six months.

上個季度，我們在針對慢性中性粒細胞減少症（另一種罕見的免疫缺陷症）患者開發 mavorixafor 方面也取得了巨大進展。在6 月下旬的投資者活動中，我們展示了正在進行的2 期臨床試驗的積極的中期臨床數據，表明每天一次口服mavorixafor 通常具有良好的耐受性，並且持久增加參與者的絕對中性粒細胞數(ANC)，無論是作為單一療法還是作為單一療法，與穩定劑量的可注射粒細胞集落刺激因子（G-CSF）聯合使用，最長可達六個月。

Importantly, 100% of the evaluable participants who had completed the six-month study as of the mid-May cutoff date achieved target ANC increases of greater than 500 cells per microliter at months three and six on once-daily mavorixafor as a monotherapy and in combination with stable G-CSF dosing. In addition, participants on mavorixafor monotherapy achieved mean ANC levels above the lower limit of normal for CN at three and six months. And mavorixafor monotherapy also durably increased ANC and participants with severe CN, defined as ANCs less than 500 cells per microliter at baseline, achieving mean ANCs of 800 to 1,000 cells per microliter, which is the ANC range targeted by experts for patients with severe CN.

重要的是，截至5 月中旬截止日期，完成為期6 個月研究的可評估參與者中，100% 在每日一次的mavorixafor 作為單一療法和在第3 個月和第6 個月實現了ANC 增加超過500 個細胞/微升的目標。此外，接受 mavorixafor 單藥治療的參與者在三個月和六個月時的平均 ANC 水準高於 CN 正常下限。mavorixafor 單藥療法也持久地增加了ANC 和患有嚴重CN 的參與者（定義為基線時ANC 低於500 個細胞/微升），實現平均ANC 為每微升800 至1,000 個細胞，這是專家為嚴重CN 患者設定的ANC 範圍。

Feedback on these interim results from the physician community has been positive, and we were pleased to see the durable impact of mavorixafor as a monotherapy and in combination with stable dose G-CSF. These interim data support our strong belief that mavorixafor may be able to address the needs of the CN patients and their physicians. Specifically, the desire for an oral treatment that does not carry short- or long-term dose-related toxicities described with G-CSF use, and for an oral treatment that also increases ANC levels and lessens the risk and severity of infections, whether used on its own or in combination with G-CSF.

醫生群體對這些中期結果的回饋是正面的，我們很高興看到 mavorixafor 作為單一療法以及與穩定劑量的 G-CSF 聯合使用的持久影響。這些中期數據支持我們堅信 mavorixafor 可能能夠滿足 CN 患者及其醫生的需求。具體來說，需要一種不帶有使用 G-CSF 所描述的短期或長期劑量相關毒性的口服治療，以及一種也能增加 ANC 水平並降低感染風險和嚴重程度的口服治療，無論是否使用單獨使用或與G-CSF 組合使用。

We're now expecting to present the full data from the Phase 2 CN trial in November, ahead of the American Society of Hematology, or ASH meeting. This data set will include results from the group of participants receiving mavorixafor and those who are allowed to dose-adjust G-CSF from baseline.

我們現在預計在 11 月的美國血液學會 (ASH) 會議之前公佈 2 期 CN 試驗的完整數據。該資料集將包括接受 mavorixafor 的參與者組和被允許從基線調整 G-CSF 劑量的參與者的結果。

We've been asked what success looks like for these data that will be presented in November. The good news is we already know what success looks like from our WHIM Phase 3 study and subsequent approval in the US.

有人問我們，這些將於 11 月發布的數據是否成功。好消息是，我們已經從 WHIM 第 3 階段研究以及隨後在美國的批准中知道了成功的樣子。

In that 52-week study, mavorixafor meaningfully increased neutrophils and lymphocytes over a sustained period, reducing infection rate, duration and severity. From the interim CN Phase 2 results we shared in June, we saw again that mavorixafor as a monotherapy and in combination with G-CSF can increase ANCs to levels expected to reduce infections.

在這項為期 52 週的研究中，mavorixafor 在持續一段時間內顯著增加了嗜中性球和淋巴球，降低了感染率、持續時間和嚴重程度。從我們 6 月分享的 CN 2 期中期結果中，我們再次看到 mavorixafor 作為單一療法並與 G-CSF 聯合使用可以將 ANC 增加到預期減少感染的水平。

In November, we'll be looking to replicate what we saw in June across all participants in the mavorixafor monotherapy and mav plus stable-dose G-CSF groups who completed the six months of the trial. From the third group, we'll be looking for data that supports physicians' ability to reduce G-CSF dosing while on mavorixafor and maintain patients' ANCs at clinically meaningful levels.

11 月，我們將在完成 6 個月試驗的 mavorixafor 單藥治療組和 mav 加穩定劑量 G-CSF 組的所有參與者中複製我們在 6 月看到的情況。從第三組中，我們將尋找支持醫生在使用 mavorixafor 時減少 G-CSF 劑量並將患者的 ANC 維持在有臨床意義的水平的能力的數據。

Finally, we'll be also looking for continued tolerability and safety of mavorixafor as seen in all of our other clinical programs to date. In June, we also announced the initiation of our global pivotal Phase 3 clinical trial, branded the 4WARD study evaluating the efficacy, safety and tolerability of oral once-daily mavorixafor with and without stable doses of G-CSF in people with congenital acquired primary autoimmune or idiopathic CN who are experiencing recurrent and/or serious infections.

最後，我們還將尋找 mavorixafor 的持續耐受性和安全性，正如我們迄今為止所有其他臨床項目所看到的那樣。6 月，我們也宣布啟動全球關鍵3 期臨床試驗，名為4WARD 研究，評估每日一次口服mavorixafor 聯合或不聯合穩定劑量G-CSF 對先天性獲得性原發性自體免疫疾病患者的療效、安全性和耐受性或正在經歷反覆和/或嚴重感染的特發性 CN。

The 52-week 4WARD trial is a randomized, double-blinded placebo-controlled multi-center study aiming to enroll 150 participants. The clinical operations team is in full swing, opening up sites, and we are on track to fully enroll the trial in mid-2025.

為期 52 週的 4WARD 試驗是一項隨機、雙盲、安慰劑對照的多中心研究，旨在招募 150 名參與者。臨床營運團隊正在緊鑼密鼓地進行試驗，我們預計在 2025 年中期全面啟動試驗。

We believe that the interim Phase 2 CN data presented to date strongly support our decision to initiate the Phase 3 trial as well as this design. Importantly, we believe that the interim results derisk the Phase 3 trial given that the ANC increases seen in CN patients to date mirror those in our successfully completed 4WHIM Phase 3 trial, where similar levels of ANC increases led to clinical benefit by reducing the frequency, duration and severity of infections.

我們相信，迄今為止提供的中期 2 期 CN 數據強烈支持我們啟動 3 期試驗以及該設計的決定。重要的是，我們認為中期結果會危及3 期試驗的風險，因為迄今為止在CN 患者中觀察到的ANC 增加與我們成功完成的4WHIM 3 期試驗中的情況相似，其中類似水平的ANC 增加通過降低頻率而帶來臨床益處，感染的持續時間和嚴重程度。

As we focus our efforts on the 4WARD CN trial, we are also looking to maximize the global opportunity in WHIM syndrome. We expect an MAA submission to the European Medicines Agency for mavorixafor in WHIM syndrome by early 2025, while we also explore additional potential opportunities in geographies where we may be able to efficiently leverage our US FDA approval.

在我們專注於 4WARD CN 試驗的同時，我們也尋求最大限度地利用 WHIM 綜合徵的全球機會。我們預計到 2025 年初，將向歐洲藥品管理局提交 mavorixafor 用於治療 WHIM 綜合徵的 MAA，同時我們也可能能夠有效利用美國 FDA 批准的地區探索其他潛在機會。

With that, I'll now turn it over to our CFO, Adam Mostafa, to review the second quarter financials. Adam?

現在，我將把它交給我們的財務長 Adam Mostafa，以審查第二季的財務狀況。亞當？

Thanks, Paula, and thanks to all of you for being on the call with us today. As we had previously discussed, upon FDA approval of XOLREMDI, we were awarded a priority review voucher, or PRV, based on mavorixafor's rare pediatric disease designation. We monetized this PRV very shortly after receipt. And in May, we recognized a gain on the sale to a third party for $105 million in cash.

謝謝保拉，也謝謝大家今天接受我們的電話。正如我們之前討論的，在 FDA 批准 XOLREMDI 後，我們根據 mavorixafor 的罕見兒科疾病資格獲得了優先審評券（PRV）。我們在收到此 PRV 後不久就將其貨幣化。5 月份，我們確認以 1.05 億美元現金出售給第三方的收益。

So as of the end of the second quarter, X4 had $169.5 million in cash, cash equivalents, restricted cash and short-term marketable securities. We believe these funds are sufficient to support our company operations into late 2025. I want to note that this projected runway does not include potential future XOLREMDI revenue.

因此，截至第二季末，X4擁有1.695億美元的現金、現金等價物、限制性現金和短期有價證券。我們相信這些資金足以支持我們公司營運至 2025 年末。我想指出的是，這個預計的跑道不包括未來潛在的 XOLREMDI 收入。

For the three months ended June 30, we reported revenues of $0.6 million and cost of revenue of $0.3 million related to the sale of XOLREMDI. This cost of revenue includes approximately $0.2 million of license costs, including sales-based royalties and operational milestones capitalized as an intangible asset and amortized over the life of the underlying intellectual property. We would like to note that there was a small amount of inventory stocking at our specialty pharmacy during the quarter, and this is reflected in the sales number.

截至 6 月 30 日的三個月，我們報告與 XOLREMDI 銷售相關的收入為 60 萬美元，收入成本為 30 萬美元。該收入成本包括約 20 萬美元的授權成本，包括基於銷售的特許權使用費和資本化為無形資產並在基礎智慧財產權生命週期內攤銷的營運里程碑。我們想指出的是，本季我們的專業藥局有少量庫存，這反映在銷售額上。

Research and development expenses were $20.9 million for the second quarter compared to $15.6 million for the comparable period in 2023. R&D expenses for the second quarter of 2024 included $1.2 million of noncash expense. Selling, general and administrative expenses were $13.3 million for the second quarter of 2024. This compares to $10.2 million for the comparable period in 2023. SG&A expenses for the second quarter also included $1.2 million of noncash expense.

第二季的研發費用為 2,090 萬美元，而 2023 年同期的研發費用為 1,560 萬美元。2024 年第二季的研發費用包括 120 萬美元的非現金費用。2024 年第二季的銷售、一般及管理費用為 1,330 萬美元。相較之下，2023 年同期為 1,020 萬美元。第二季的 SG&A 支出還包括 120 萬美元的非現金支出。

Finally, we reported net income of $90.8 million for the second quarter of 2024 compared to a net loss of $55.7 million for the comparable period in 2023. Note that net income in the current period included the sale of the PRV for $105 million as well as a noncash gain of $20.2 million related to the fair value remeasurement of the company's Class C warrant liability.

最後，我們報告 2024 年第二季淨利為 9,080 萬美元，而 2023 年同期淨虧損為 5,570 萬美元。請注意，本期淨利潤包括以 1.05 億美元出售 PRV 以及與公司 C 類認股權證負債公允價值重新計量相關的 2,020 萬美元非現金收益。

I'll now turn it back to Paula for some concluding remarks.

現在我將把它轉回給保拉，讓其做一些總結性發言。

As you can see, all of these accomplishments mark a significant step forward for X4, now a fully integrated biopharmaceutical company. As we continue to explore additional uses for and maximize the global potential of mavorixafor for patients, we look forward to reporting on our sales, regulatory and clinical progress over the coming 6 to 12 months.

正如您所看到的，所有這些成就都標誌著 X4 向前邁出了重要一步，X4 現在是一家完全一體化的生物製藥公司。隨著我們繼續探索 mavorixafor 的其他用途並最大限度地發揮 mavorixafor 對患者的全球潛力，我們期待在未來 6 至 12 個月內報告我們的銷售、監管和臨床進展。

We'll now open up the call for your questions. Operator?

我們現在將開始電話詢問您的問題。操作員？

(Operator Instructions) Kristen Kluska, Cantor.

（操作員說明）Kristen Kluska，Cantor。

Congratulations on reaching 10 years. It's a great milestone and how better to celebrate with the drug approval this year. So first, I wanted to ask how you believe physicians are going to measure success differently in a commercial setting versus the clinic for XOLREMDI. Will it be primarily on patient anecdotes, reports around infections or anything else to consider?

恭喜你已經滿10歲了。這是一個偉大的里程碑，也是如何更好地慶祝今年的藥物批准。首先，我想問您認為醫生在商業環境中與在診所中衡量 XOLREMDI 成功的方式有何不同。主要是患者軼事、感染報告還是其他需要考慮的內容？

Thanks, Kristen. I'll turn it over to Mark to give some perspectives on what they're hearing from the field.

謝謝，克里斯汀。我會將其交給馬克，讓他對他們從現場聽到的情況發表一些看法。

Yes. We're -- the launch is well underway, Kristen. And what's exciting is that we're hearing that physicians and patients have been waiting for a treatment that targets the underlying cause of the disease. And so now we have the approval of XOLREMDI, it's really opening up these conversations and allowing us to help physicians become aware of the disease, recognize the patients in their practice and then think about how they can help these patients with XOLREMDI.

是的。克里斯汀，我們的發布正在順利進行中。令人興奮的是，我們聽說醫生和患者一直在等待針對該疾病根本原因的治療方法。現在我們獲得了 XOLREMDI 的批准，這確實開啟了這些對話，讓我們能夠幫助醫生了解這種疾病，在實踐中認識患者，然後思考如何幫助這些 XOLREMDI 患者。

We know from the market research that it's really the impact on infections that drives the value of XOLREMDI for physicians, for patients and payers. So that's really what I think physicians will be looking for as they get more experience with the product.

我們從市場研究中得知，正是對感染的影響推動了 XOLREMDI 對醫生、病人和付款人的價值。因此，我認為這確實是醫生在獲得更多產品使用經驗後所需要的。

And can you give us a sense of how partnership discussions ex US are going at this stage? And just curious, your press release noted some ways you're looking to maximize the value of mavorixafor beyond WHIM syndrome and CN. So curious in these conversations, this has come up with potential partners about ways that they can also potentially utilize this drug in other indications as well.

您能否向我們介紹一下現階段美國以外的合作夥伴關係討論的進展？只是好奇，你們的新聞稿指出了你們希望在 WHIM 綜合徵和 CN 之外最大化 mavorixafor 價值的一些方法。在這些對話中，我們感到非常好奇，這讓潛在的伴侶想到了他們也可以在其他適應症中使用這種藥物的方法。

Yes. So thanks, Kristen. I mean when we currently think about the FDA approval, there are certain regions in the world that can leverage that existing approval. So there's some exploration there currently ongoing.

是的。謝謝，克里斯汀。我的意思是，當我們目前考慮 FDA 的批准時，世界上某些地區可以利用現有的批准。目前正在進行一些探索。

Adam can comment more on the more strategic side of potential collaborations.

Adam 可以對潛在合作的更具策略性的方面進行更多評論。

Yes. Thanks, Kristen. So certainly, there is interest and we have discussions, as Paula mentioned, ongoing with potential partners. We'll certainly come back and update the market as appropriate if we're going to consummate a transaction. But I think, given the recent approval and our recent positive chronic neutropenia Phase 2 data, there's only growing interest in this area.

是的。謝謝，克里斯汀。因此，正如保拉所提到的，我們當然有興趣，並且正在與潛在合作夥伴進行討論。如果我們要完成交易，我們肯定會回來並酌情更新市場。但我認為，鑑於最近的批准和我們最近的慢性中性粒細胞減少症第二階段的積極數據，人們對這一領域的興趣只會越來越大。

Ed Tenthoff, Piper Sandler.

艾德·騰索夫，派珀·桑德勒。

Yes, it's exciting to see some revenues at a company that a lot of work has gone to get that first couple of hundred thousand dollars, and I know it will grow from here. My question had to do with sort of characterizing the patients that are on drug. Can you give us a sense for how many patients are on drug? And anything about sort of the backlog? And if you're not able to give us direct patient numbers, maybe tell us sort of a little bit about the journey of patients that you're seeing as you sort of get them into commercial use of XOLREMDI.

是的，看到一家公司付出了很多努力才獲得了最初的幾十萬美元的收入，這是令人興奮的，而且我知道它會從這裡開始成長。我的問題與描述服用藥物的患者有關。您能否告訴我們有多少患者正在接受藥物治療？還有關於積壓的事情嗎？如果您無法向我們提供直接的患者人數，也許可以告訴我們一些有關您所看到的患者在將 XOLREMDI 投入商業用途時的經歷。

Thanks so much, Ted. So I think we won't be breaking down any details and continue to report on revenue. But I mean, we're overall extremely pleased with just the cascade of commitment that the company has made and then now are experiencing the receipt of that commitment in the communities as we head out.

非常感謝，泰德。所以我認為我們不會分解任何細節並繼續報告收入。但我的意思是，總的來說，我們對公司所做的一系列承諾感到非常滿意，現在我們在出發時正在社區中體驗到這項承諾的兌現。

But Mark can add some more color in kind of how they're seeing the journey from the diagnosis and education support all the way through patients on drug.

但馬克可以為他們如何看待從診斷和教育支持一直到患者用藥的整個過程添加更多色彩。

Yes. And I think the first thing to remember, this is an ultra-rare disease. And so the key here is to begin to really raise awareness, both through the -- our conversations we're having with physicians, our engagement with patient advocacy groups and also through our digital marketing campaigns because that's where we can get extended reach, and we've refreshed our disease awareness campaign. We've also launched our branded XOLREMDI digital campaigns. So we're really creating this kind of momentum around the conversations.

是的。我認為首先要記住的是，這是一種極為罕見的疾病。因此，這裡的關鍵是開始真正提高意識，無論是透過我們與醫生的對話，我們與患者倡導團體的接觸，還是透過我們的數位行銷活動，因為這是我們可以擴大影響力的地方，而我們更新了疾病意識活動。我們也推出了我們的品牌 XOLREMDI 數位行銷活動。所以我們確實在圍繞對話創造這種勢頭。

And the patients -- as Paula mentioned on the call, all of the patients that were in the open-label extension have been prescribed XOLREMDI here in the US. And so from there, we're just learning more and more about how the patients have been waiting for a treatment that targets their disease, and they're now having those conversations with physicians.

正如 Paula 在電話中提到的，所有參與開放標籤擴展的患者都已在美國開出 XOLREMDI 處方。因此，從那時起，我們只是越來越了解患者如何等待針對其疾病的治療，而他們現在正在與醫生進行這些對話。

RK, H.C. Wainwright.

RK、H.C.溫賴特。

Congratulations on the 10 years and also the launch. In terms of -- I know it's early stages of the launch. But when you go and attend these conferences, such as the Immunodeficiency Conference, how are -- what is it that you're hearing from physicians and patients at this point in terms of getting ready to take -- to adopt the drug. And also have you learned anything else that needs to be looked at in terms of improving -- I'm not saying improving, getting adoption to a better level from the get-go?

祝賀成立 10 週年，也祝賀發布。就我而言，我知道現在還處於發布的早期階段。但是，當你去參加這些會議時，例如免疫缺陷會議，你從醫生和病人那裡聽到的關於準備服用藥物的資訊是如何採用這種藥物的。另外，您是否也學到了任何其他需要在改進方面考慮的內容——我不是說改進，從一開始就將採用提高到一個更好的水平？

Thanks so much, RK. I think we're very pleased with kind of the cascade of thinking and development. And certainly, what we're hearing, and Mark will add to this is, number one, they're excited about a targeted therapy, and I'll even add the oral-targeted therapy. It's been a field of injectable treatment with varying degrees of study and rigor. Our Phase 3 was phenomenally successful showing infection burden reduction across rate, severity and duration. So that is a great story to begin with and a great data set to share.

非常感謝，RK。我認為我們對一系列的思考和發展感到非常滿意。當然，我們聽到的，馬克將補充的是，第一，他們對標靶治療感到興奮，我甚至會添加口服標靶治療。這是一個具有不同程度研究和嚴格程度的注射治療領域。我們的第三階段非常成功，顯示感染率、嚴重程度和持續時間的感染負擔有所減輕。所以這是一個很棒的故事，也是一個值得分享的很棒的數據集。

And then in terms of the clinicians, of course, they're all on their own journey even understanding the disease and then learning about the benefits of XOLREMDI. But I'll turn it over to Mark to add some color to that as well.

當然，就臨床醫生而言，他們都在自己的旅程中，甚至了解這種疾病，然後了解 XOLREMDI 的好處。但我會把它交給馬克來添加一些顏色。

Yes, exactly. I think it's really now that we have an approved product, it's really energized physicians to go back and really look in their practice for these patients who, before now, have really had no treatment available for their disease. And so now we've actually given them a reason to go back, look for these patients, make the diagnosis and then have a conversation with the patient about XOLREMDI.

是的，完全正確。我認為現在我們有了一個獲得批准的產品，這確實激勵了醫生回去並真正研究這些患者的實踐，而這些患者在此之前確實沒有針對其疾病的治療方法。所以現在我們實際上給了他們一個回去的理由，尋找這些患者，做出診斷，然後與患者就 XOLREMDI 進行對話。

Well said. And then for the SCN indication, so between now and if there is an interim analysis for the 4WARD study, is there any other data that will be presented so that we can understand how this program is moving forward?

說得好。然後對於 SCN 指示，從現在到 4WARD 研究進行中期分析，是否會提供任何其他數據，以便我們了解該計劃的進展？

Sure. So as we shared today, we'll be giving the full Phase 2 data set in November of this year. So of course, we were extremely pleased with the data set already, but it was interim. So we've already seen durable responses of the drug as monotherapy, and then sustained and durable responses in addition to stable G-CSF. So the final puzzle piece is just exploring in what circumstances G can get reduced.

當然。正如我們今天分享的，我們將在今年 11 月提供完整的第 2 階段資料集。當然，我們已經對數據集非常滿意，但它是臨時的。因此，我們已經看到該藥物作為單一療法的持久反應，然後除了穩定的 G-CSF 之外，還看到了持續且持久的反應。所以最後的難題就是探索在什麼情況下G可以減少。

So that full data set will come in November, which is going to be certainly well-timed, just before ASH. We'll be at the ASH meeting to help support the CN community and also get the word further out on our trial enrollment. So it's an excellent timing perspective, and then we'll look forward as appropriate to giving updates on how the whole study is going. But certainly, that will be a 2025 update.

因此，完整的數據集將在 11 月發布，這無疑是恰逢其時，就在 ASH 之前。我們將參加 ASH 會議，幫助支持 CN 社區，並進一步宣傳我們的試驗註冊情況。因此，這是一個很好的時機視角，然後我們將酌情提供有關整個研究進展的最新資訊。但可以肯定的是，這將是 2025 年的更新。

Leah Cann, Brookline Capital Markets.

Leah Cann，布魯克林資本市場。

I have a financial question. So Adam, could you help us with the number of fully diluted shares at the end of the quarter, including the prefunded warrants?

我有一個財務問題。那麼，亞當，您能否幫助我們了解本季末完全稀釋的股票數量，包括預先融資的認股權證？

Sure. Thanks. So there's about 167 million basic shares, regular shares outstanding, 33 million prefunded warrants. So $200 million of share equivalents, if you will. And then there are 75 million warrants that are cash only. Those are divided into two tranches, 130 million tranche, 145 million tranche. So overall, 275 million in fully diluted shares. There's some historical warrants you'll see later today in our 10-Q that are significantly out of the money, that are typically not included, but that accounts for a small piece.

當然。謝謝。因此，約有 1.67 億股基本股、已發行普通股和 3,300 萬股預售認股權證。如果你願意的話，那就是 2 億美元的股票等價物。還有 7,500 萬張認股權證只能以現金支付。這些分為兩部分，1.3億部分，1.45億部分。總體而言，完全稀釋後的股份為 2.75 億股。今天晚些時候，您將在我們的 10-Q 報告中看到一些歷史認股權證，這些認股權證明顯處於價外，通常不包括在內，但只佔一小部分。

Stephen Willey, Stifel.

史蒂芬威利，斯蒂菲爾。

Could you maybe just talk a little bit about -- and again, I understand that it's early, but the kinetics of the reimbursement process? I know the paperwork that is required sometimes for these higher-priced rare disease drugs can be a somewhat lengthy process. And just curious if you have an early read just based on the limited experience thus far as to what that process might look like from just a timing perspective.

您能否稍微談談—我知道現在還為時過早，但報銷流程的動態？我知道這些價格較高的罕見疾病藥物有時所需的文書工作可能是一個有點漫長的過程。只是好奇您是否基於迄今為止有限的經驗而提前閱讀了從時間角度來看該過程可能是什麼樣子的。

Sure, Steve. I mean, I'll turn it over to Mark. I think classic ultra-rare disease typically has barriers that a lot of these insurance companies naturally provide, given the magnitude of the drug and its options for patients. But the good news is, I think we've had a very experienced team well ahead of this.

當然，史蒂夫。我的意思是，我會把它交給馬克。我認為，考慮到藥物的規模及其為患者提供的選擇，典型的超罕見疾病通常會遇到許多保險公司自然提供的障礙。但好消息是，我認為我們已經擁有一支經驗豐富的團隊。

So I'll turn it over to Mark to give some insights there.

所以我會把它交給馬克來提供一些見解。

Yes. Steve. Yes, as Paula mentioned during the call, payers are using their standard methods for new drugs. And so they're looking at PAs and exceptions process. But these are working quite smoothly, and patients have been able to access XOLREMDI.

是的。史蒂夫.是的，正如保拉在電話中提到的那樣，付款人正在使用他們的新藥標準方法。因此他們正在研究 PA 和例外流程。但這些工作進展相當順利，患者已能使用 XOLREMDI。

Of course, we also have our full suite of patient service programs through our X4Connect, which is our patient hub. And this ensures that patients are quickly -- get quick access to the product while we work through the reimbursement. But it's good to see the process working smoothly.

當然，我們也透過 X4Connect（我們的患者中心）提供全套患者服務計劃。這確保了患者在我們處理報銷時能夠快速獲得產品。但很高興看到這個過程進展順利。

Okay. That's helpful. And maybe a guidance question, not one related to actual sales. But just curious how you're thinking about communicating enrollment progress in the Phase 3 4WARD trial going forward? Should we expect updates in conjunction with certain milestones, say, at like 50%? Or is this just going to be kind of a moving target and we'll hear about it when we hear about it?

好的。這很有幫助。也許是一個指導性問題，而不是與實際銷售相關的問題。但只是好奇您如何考慮在未來 3 期 4WARD 試驗中傳達註冊進度？我們是否應該期待與某些里程碑相關的更新，例如 50%？或者這只是一個移動目標，當我們聽到它時我們就會聽到它？

Yes. Thanks, Steve. I think we'll always aim to communicate meaningful progress. Of course, we don't want to make any commitments just because it's a 2025 target, and it's almost a year away at this point. However, certainly, as we get confidence under our belt here, we'll be able to provide some more specifics. But again, I would say that's a 2025 zone to share.

是的。謝謝，史蒂夫。我認為我們將始終致力於傳達有意義的進展。當然，我們不想因為這是 2025 年的目標就做出任何承諾，而且距離現在已經快一年了。然而，當然，當我們在這裡獲得信心時，我們將能夠提供更多細節。但我想說的是，這是一個值得分享的 2025 年區域。

Okay. And then maybe just another financial question for Adam. SG&A just looks to be down about 25% sequentially, which I guess I wouldn't have been anticipated in the context of undertaking a launch. So can you just remind us if there's anything either unique to the 1 quarter number or this quarter number that impacts that SG&A line item?

好的。對亞當來說，這可能只是另一個財務問題。SG&A 看起來比上一季下降了約 25%，我想在進行產品發佈時我是沒有預料到的。那麼，您能否提醒我們，第一季數字或本季數字是否有任何獨特之處會影響 SG&A 行項目？

Thanks, Steve. Yes. So the second quarter number is probably more representative of a typical recurring figure. The first quarter, as we mentioned last time, had a number of onetime nonrecurring sort of start-up-related costs to building a sales force and getting the launch underway early in the year ahead of approval. So that quarter was a little more inflated, if you will, in the SG&A line than I would expect going forward. So that's why the drop looks as it does.

謝謝，史蒂夫。是的。因此，第二季的數字可能更能代表典型的經常性數字。正如我們上次提到的，第一季有一些一次性的非經常性啟動相關成本，用於建立銷售團隊並在年初批准之前啟動啟動。因此，如果你願意的話，該季度的 SG&A 專案比我預期的未來要膨脹一些。這就是水滴看起來如此的原因。

Congrats on the progress.

祝賀取得的進展。

David Bautz, Zacks Small-Cap Research.

David Bautz，Zacks 小型股研究公司。

Question on the mechanism of how prescriptions are filled. Are they filled on a monthly basis? Is it multiple monthly? And then if you could talk a little bit about how revenue is recognized in relation to when those prescriptions are filled?

關於處方配藥機制的問題。它們是按月填寫的嗎？是每月多次嗎？那麼您是否可以談談如何根據這些處方的配藥時間來確認收入？

Sure. So I mean I don't think we have intended to disclose kind of the play-by-play for our patients. I mean, as we mentioned with our current revenue line, it does blend, obviously, both an inventory build as well as revenue recognition for individual patients that are now on commercial therapy.

當然。所以我的意思是，我不認為我們打算向我們的患者透露具體情況。我的意思是，正如我們在當前的收入線中提到的那樣，顯然，它確實融合了庫存建設和目前接受商業治療的個別患者的收入確認。

So I don't think at this point, we're ready to provide any more details. But certainly, we do want -- we do understand the market's interest in helping them forecast. So as we head into later this year and 2025, we'll try to provide a bit more metrics, which really can help provide some benefit to you all as we think about how the product will grow over time.

所以我認為目前我們還沒有準備好提供更多細節。但當然，我們確實希望——我們確實了解市場對幫助他們進行預測的興趣。因此，當我們進入今年晚些時候和 2025 年時，我們將嘗試提供更多的指標，當我們考慮產品將如何隨著時間的推移而增長時，這確實可以幫助為大家帶來一些好處。

Ladies and gentlemen, it appears we have no further questions this morning. Dr. Ragan, I'd like to turn things back to you, ma'am, for any closing comments.

女士們先生們，看來我們今天早上沒有其他問題了。拉根博士，女士，我想請您提出結束語。

Well, thank you so much, operator. Thank you, everyone, for joining the call today, and we hope you have an excellent rest of your day. Take care.

嗯，非常感謝你，接線生。感謝大家今天加入電話會議，我們希望您有個愉快的一天。小心。

Thank you, Dr. Ragan. Ladies and gentlemen, again, that does conclude today's X4 Pharmaceuticals' Second Quarter Earnings Call. Again, thanks so much for joining us, everyone. And again, we wish you all a great day. Goodbye.

謝謝你，拉根醫生。女士們先生們，今天的 X4 Pharmaceuticals 第二季財報電話會議到此結束。再次非常感謝大家加入我們。再次祝福大家有個愉快的一天。再見。