X4 Pharmaceuticals Inc (XFOR) 2025 Q1 法說會逐字稿

Greetings and welcome to the X4 Pharmaceuticals first quarter 2025 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 call is being recorded. It is now my pleasure to introduce your host, Dan Ferry from LifeSci Advisors. Please begin.

問候並歡迎參加 X4 Pharmaceuticals 2025 年第一季財務和經營業績電話會議。此時，所有參與者都處於只聽模式。正式演講結束後將進行問答環節。提醒一下，本次電話會議正在錄音。現在我很高興介紹您的主持人，來自 LifeSci Advisors 的 Dan Ferry。請開始。

Thank you operator good morning everyone. Presenting on today's call will be X4 Chief Executive Officer, Doctor Paula Ragan, and Chief Financial Officer, Adam Mostafa. Following prepared remarks by each, we will open the call to your questions and we'll be joined by Chief Commercial Officer, Mark Baldry and Chief Medical Officer, Doctor Christophe Arbet-Engels.

謝謝接線員，大家早安。出席今天電話會議的有 X4 執行長 Paula Ragan 博士和財務長 Adam Mostafa。在每位嘉賓都準備好發言之後，我們將開始回答大家的問題，首席商務官馬克·鮑德里 (Mark Baldry) 和首席醫療官克里斯托夫·阿爾貝特·恩格斯 (Christophe Arbet-Engels) 醫生也將加入我們的討論。

As a reminder on today's call, the company will be making forward-looking statements regarding regulatory and product development plans. These statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted. A description of these risks can be found in X4's most recent filings with the SEC, including last year's For 10-K and this past quarters form 10-Q. Which is expected to be filed after market close today.

在今天的電話會議上提醒大家，公司將就監管和產品開發計畫做出前瞻性聲明。這些聲明受風險和不確定性的影響，可能導致實際結果與預測結果不同。這些風險的描述可以在 X4 向美國證券交易委員會提交的最新文件中找到，包括去年的 10-K 表和過去幾個季度的 10-Q 表。預計今天收盤後提交。

Please note that the X4 investor deck was updated this morning on the company website to include slides detailing some of the data analysis mentioned in this morning's press release and on this call today. I'd now like to turn the call over to ex-Force's President and CEO Doctor Paula Reagan. Paula?

請注意，X4 投資者簡報已於今天早上在公司網站上更新，其中包括詳細介紹今天早上新聞稿和今天電話會議中提到的一些數據分析的幻燈片。現在我想將電話轉給前 Force 總裁兼執行長 Paula Reagan 博士。保拉？

Thank you, Dan, and thanks to all of you for joining us this morning.

謝謝你，丹，也謝謝大家今天早上加入我們。

The first quarter of 2025 was an extremely productive and value building period for X4, with clinical trial advancement in chronic neutropenia, continued progress in the commercialization of the remedy for WHIM syndrome in the US, and our achievement of several significant milestones towards expanding the global potential of Mavericks for patients.

2025 年第一季對於 X4 來說是極其高效和價值積累的時期，慢性中性粒細胞減少症的臨床試驗取得了進展，WHIM 綜合徵藥物在美國的商業化方面繼續取得進展，我們在擴大 Mavericks 對患者的全球潛力方面取得了幾個重要的里程碑。

Let's begin with Maverick support in chronic neutropenia, or CN. As having successfully developed Maverick 4 branded Xolremdi for patients with WHIM syndrome in the US, we are now also developing Maverick 4 for the larger indication of chronic neutropenia. There are currently about 50,000 people diagnosed with some form of CN in the US based on retrospective ICB 10 code analysis.

讓我們從 Maverick 對慢性中性粒細胞減少症 (CN) 的支持開始。由於我們已在美國成功開發出用於治療 WHIM 綜合徵患者的 Maverick 4 品牌 Xolremdi，因此我們現在也在開髮用於治療更大適應症——慢性中性粒細胞減少症的 Maverick 4。根據回顧性 ICB 10 代碼分析，目前美國約有 5 萬人被診斷出患有某種形式的 CN。

Of those currently diagnosed with primary CN, we estimate that approximately 15,000 individuals, or about 30 to 40%, have remaining health challenges and continue to experience low absolute neutrophil counts, or A&Cs, and recurrent infections despite available standard of care.

在目前被診斷為原發性中性粒細胞減少症 (CN) 的患者中，我們估計約有 15,000 人（約佔 30% 至 40%）仍面臨健康問題，儘管有標準護理，但仍會繼續經歷低絕對中性粒細胞計數（A&C）和反覆感染。

We define this as the high unmet needs patient population, and it is with this population in mind that we are launching our ongoing forward trial, a global pivotal phase 3 clinical trial evaluating the safety and efficacy of once daily oral mavericks before in people with certain chronic neutropenic conditions, including primary autoimmune, idiopathic, and congenital neutropenia. And who are experiencing recurrent and or serious infections.

我們將其定義為高未滿足需求的患者群體，正是考慮到這一群體，我們啟動了正在進行的前瞻性試驗，這是一項全球關鍵的 3 期臨床試驗，評估每日一次口服 mavericks 對患有某些慢性中性粒細胞減少症（包括原發性自體免疫、特發性、先天性中性粒細胞減少症）的人的安全性和有效性。以及正在經歷反覆或嚴重感染的人。

As we reported on our last call, we're now screening and enrolling participants in over 20 countries with more than 90% of our targeted global trial sites being activated. Additionally, we've now finalized the trial design based on the feedback we also discussed previously from both the FDA and EMA to focus on those with the highest unmet needs, a population that matches up well with Mavericks for its targeted commercial CN patient population.

正如我們在上次電話會議上報告的那樣，我們目前正在 20 多個國家/地區篩選和招募參與者，我們目標全球試驗地點的 90% 以上已經啟動。此外，我們現在已經根據先前討論過的 FDA 和 EMA 的回饋完成了試驗設計，重點關注那些未滿足需求最高的患者，這一人群與 Mavericks 針對的商業 CN 患者群體非常匹配。

The trials enrolling with those with moderate severe CN or A&C below 1,000 cells per microliter and experiencing two or more infections over the past 12 months. We also finalize the ANC response endpoint. The definition of ANC response is now uniform across all participants and is defined as an increase in A&C greater than 500 cells per microliter versus baseline ANC and occurring at 50% or more of the time points evaluated in the trial.

試驗招募了患有中度嚴重 CN 或 A&C（低於每微升 1,000 個細胞）並且在過去 12 個月內經歷過兩次或兩次以上感染的患者。我們也最終確定了 ANC 響應端點。現在，所有參與者對 ANC 反應的定義都是統一的，即與基線 ANC 相比，A&C 的增加量大於每微升 500 個細胞，並且發生在試驗中評估的 50% 或更多時間點。

The trial seeks to demonstrate statistically significant increases in ANC response and corresponding decreases in annualized infection rates between those on Mavericks floor versus placebo. To date, the demographics of the enrolled population are balanced and representative of the target commercial CN patient populations, and baseline ANCs and historical infection rates are consistent with this high unmet need population.

該試驗旨在證明 Mavericks 場地上的 ANC 反應與安慰劑組相比有統計上顯著的增加，且年感染率相應下降。迄今為止，入組人群的人口統計數據是平衡的，並且代表了目標商業 CN 患者人群，並且基線 ANC 和歷史感染率與這一高未滿足需求人群一致。

We recently completed some additional data analyses that further increased our confidence in the success of the forward trial. Individual patient data from both the Mavericks for Phase 34IM trial and the completed phase 2 CN trial have now been analyzed applying this just mentioned forward trial and response criteria. The full details of these analyses can be found in the updated investor deck that is on the front page of the investor section of our website.

我們最近完成了一些額外的數據分析，進一步增強了我們對前向試驗成功的信心。現在已經應用剛才提到的前瞻性試驗和反應標準對來自 Mavericks 34 期 IM 試驗和已完成的 2 期 CN 試驗的個人患者數據進行了分析。這些分析的完整詳情可在我們網站投資者部分首頁的更新後的投資者資料中找到。

In summary, we created what we're calling heat maps, which detail individual ANC responses across all trial participants at all of the assessed time points. Specifically, the 4 whim phase 3 heat map provides a benchmark for ANC responses that translated into a 60% reduction in annualized infection rates when comparing Mavericks for treatment to placebo.

總之，我們創建了所謂的熱圖，其中詳細記錄了所有評估時間點的所有試驗參與者的個人 ANC 反應。具體來說，4 whim 第 3 階段熱圖為 ANC 反應提供了基準，當將 Mavericks 與安慰劑進行比較時，年感染率降低了 60%。

The CM Phase 2 heat map demonstrates the impact of Mavericks for undurable increases in AMC across those with idiopathic, cyclic, and congenital chronic neutropenia. ANC outcomes in the CN phase 2 trial looks similar to ANC responses seen in the Maverick's forearm of the 4 whim trial.

CM 第 2 期熱圖展示了 Mavericks 對特發性、週期性和先天性慢性嗜中性白血球減少症患者的 AMC 持續增加的影響。CN 第 2 階段試驗中的 ANC 結果與 4 次 whim 試驗中 Maverick 前臂中看到的 ANC 反應相似。

When taken together, these heat map analyses provide evidence supporting the potential success of the CN phase 3 trial, where we believe that the expected ANC responses resulting from maverick support treatment will correspond to a significant decrease in annualized infection rates.

綜合起來，這些熱圖分析提供了支持 CN 3 期試驗潛在成功的證據，我們相信，特立獨行支持治療產生的預期 ANC 反應將對應於年感染率的顯著下降。

We also continue to believe that the forward trial is rigorously designed and power to demonstrate the impact of Mavericks for NCM. The trial is powered at greater than 95% to assess the ANC response endpoint, and the 150 participant sample size independently supports robust powering at greater than 90% for the infection rate results.

我們也繼續相信，前瞻性試驗是經過嚴格設計的，能夠證明 Mavericks 對 NCM 的影響。該試驗的功效超過 95% 以評估 ANC 反應終點，並且 150 名參與者的樣本量獨立支持感染率結果的強大功效超過 90%。

As of today, we continue to anticipate full enrollment in the trial in the 3rd or 4th quarter of 2025, which would enable disclosure of top line data in the second half of 2026. Lastly, on the front, we have good news from the US Patent Office. We received a notice of allowance on our application which claims include the use of Mavericks for in treating severe chronic idiopathic and autoimmune neutropenia in patients without a CHCR4 genetic variants.

截至今天，我們仍預期該試驗將在 2025 年第三季或第四季全面完成招募，這將使我們能夠在 2026 年下半年揭露頂線數據。最後，我們從美國專利局傳來了好消息。我們的申請收到了批准通知，其中聲稱包括使用 Mavericks 治療沒有 CHCR4 基因變異的患者的嚴重慢性特發性和自體免疫性中性粒細胞減少症。

The patent is expected to expire in the US in March of 2041. Similar patent applications are pending in Europe, China, Japan, and Canada. To conclude, we remain confident that we'll be able to deliver on our upcoming milestones in CN that will have a long-termed protection in the indication, and that the value proposition for Mavericks 4 and CN could represent a $1million to $2 billion dollar opportunity in the US alone. With that, let's turn now to our progress with Mavericks 4 and WHIM syndrome.

該專利預計將於2041年3月在美國到期。歐洲、中國、日本和加拿大也正在申請類似的專利。總而言之，我們仍然相信，我們將能夠實現我們在 CN 中即將取得的里程碑，這將對指示產生長期保護，並且 Mavericks 4 和 CN 的價值主張僅在美國就可能代表 100 萬至 20 億美元的機會。有了這些，現在讓我們來談談 Mavericks 4 和 WHIM 症候群的進展。

At the end of March of this year, cumulative sales of the Remy reached 3.5 million since our mid-May launch last year.

自去年5月中旬上市以來，截至今年3月底，Remy累計銷量已達350萬輛。

This quarter's sales were slightly lower than those reported in the fourth quarter because of the fluctuations in the timing of inventory resupply, which causes some lumpiness to sales. This is typical with markets anchored in small patient populations and early in launch. We do expect us to even out over time with the increasing demand that we're already seeing.

本季銷售額較第四季略有下降，原因是庫存補給時間波動，導致銷售額出現一定波動。對於患者群體較少且處於產品發布初期的市場來說，這種情況很常見。我們確實希望隨著時間的推移，隨著我們已經看到的不斷增長的需求，我們能夠實現平衡。

We are currently in the thick of conference season and continue to have fruitful engagements with all of our targeted top tier immunologists and hematologists, increasing the visibility of WHIM syndrome, and we're seeing success in our educational efforts that support HCPs in finding WHIM patients, with new patients now representing approximately 40% of our current oredi treated population at the end of the first quarter.

我們目前正處於會議季的高峰期，並繼續與所有目標頂級免疫學家和血液學家進行富有成效的交流，提高了 WHIM 綜合徵的知名度，並且我們在支持 HCP 尋找 WHIM 患者的教育工作中取得了成功，截至第一季度末，新患者約占我們目前接受 oredi 治療人群的 40%。

In addition, we're just about to kick off our WHIM patient ambassador program and hope these efforts will continue to build demand for Xolremdi in the US. As we discussed on our last call, we made significant progress in our efforts to expand the potential global reach of Mavericks for and Whim during the 1st quarter. In January, we announced that our submitted MAA was accepted by European regulatory authorities for review.

此外，我們即將啟動 WHIM 患者大使計劃，並希望這些努力將繼續增加美國對 Xolremdi 的需求。正如我們在上次電話會議上所討論的那樣，我們在第一季為擴大 Mavericks 和 Whim 的潛在全球影響力所做的努力取得了重大進展。今年一月，我們宣布我們提交的MAA已被歐洲監管機構接受審查。

With a typical 12 to 15 month review process, we anticipate potential approval from the EMA as early as the first quarter of 2026. We also announced the completion of two international partnerships in the 1st quarter. The first with Norgine, a leading European specialty pharmaceutical company, to commercialize Mavericks for in Europe, Australia, and New Zealand.

審查流程通常為 12 至 15 個月，我們預計最早可能在 2026 年第一季獲得 EMA 批准。我們也宣佈在第一季完成了兩項國際合作。首次與歐洲領先的專業製藥公司 Norgine 合作，在歐洲、澳洲和紐西蘭推廣 Mavericks。

Norgine will be launching Maverick support for the win indication in the EU should we receive approval there next year, and we are working closely with them to ready for that possibility. The second partnership is with Taba Rare, another specialty pharmaceutical company to commercialize the remedy in the Middle East and North Africa, or MIA region, following any approvals there. The MIA region does have a compassionate use program that allows physicians to prescribe drugs approved in other countries to local patients with no other treatment options. We'll keep you updated on our progress there.

如果我們明年在歐盟獲得批准，Norgine 將推出對勝利指示的 Maverick 支持，我們正在與他們密切合作，為這種可能性做好準備。第二個合作夥伴是與另一家專業製藥公司 Taba Rare 合作，在獲得批准後，在中東和北非（MIA）地區將藥物商業化。MIA 地區確實有一個同情用藥計劃，允許醫生為沒有其他治療選擇的當地患者開出其他國家批准的藥物。我們將隨時向您通報我們的進度。

Lastly, we're continuing to advance the understanding of WHIM syndrome as well as the impact of Mavericks for on the disease. We had two abstracts accepted for publication at the annual meeting of the Clinical Immunology Society, or CIS, which starts today.

最後，我們將繼續加深對 WHIM 症候群以及 Mavericks 對該疾病的影響的理解。我們有兩篇摘要被接受在今天開始的臨床免疫學學會（CIS）年會上發表。

From the 4 phase 3 open label extension, or OLE, we'll be presenting 2 year data that demonstrate a marked clinical improvement in warts severity as assessed by a standard measurement, the clinical global impression of severity across 70 defined war areas. We're also presenting results from the first ever survey looking into infection burden in WHIM patients. 20 WHIM patients provided responses. None were on Xolremdi at the time.

從第 4 階段 3 期開放標籤擴展（OLE）開始，我們將提供 2 年的數據，這些數據表明疣嚴重程度有了顯著的臨床改善，這透過標準測量來評估，即 70 個指定戰區的嚴重程度的臨床整體印象。我們也展示了首次針對 WHIM 患者感染負擔的調查結果。20 名 WHIM 患者給出了答案。當時沒有人服用 Xolremdi。

The survey revealed that 60% of those under 18 years and 73% of those 18 or over reported experiencing at least one infection in the previous 3 months, with 25% requiring overnight hospitalizations due to infection. The study concludes that quote the frequency and severity of infections requiring medical care and hospitalizations underscores the urgency to proactively treat patients with WHIM syndrome.

調查顯示，60% 的 18 歲以下人群和 73% 的 18 歲以上人群報告在過去 3 個月內至少感染過一次病毒，其中 25% 的人因感染而需要住院過夜。研究的結論是，需要醫療護理和住院治療的感染的頻率和嚴重程度強調了積極治療 WHIM 綜合徵患者的緊迫性。

Needless to say, we're very proud to be able to have developed this first approved therapy for WHIM in the US and look forward to commercial updates and continued global advancement milestones in the coming quarters. I'll now turn it over to Adam to run through our financials. Adam.

毋庸置疑，我們非常自豪能夠在美國開發出首個獲批的 WHIM 療法，並期待在未來幾季實現商業更新和持續的全球進步里程碑。現在我將把時間交給亞當來處理我們的財務狀況。亞當。

Thanks, Paula. As we disclosed in the press release this morning, we ended the first quarter of 2025 with just under $90 million in cash and cash equivalents. We continue to believe that we have sufficient funds to support company operations into the first half of 2026. We reported netzo remedy revenues of just under $1 million for the first quarter of 2025. As Paul mentioned, this brings our cumulative total sales since our May 2024 launch to about $3.5 million.

謝謝，保拉。正如我們今天早上在新聞稿中披露的那樣，截至 2025 年第一季度，我們的現金和現金等價物略低於 9000 萬美元。我們仍然相信，我們有足夠的資金來支持公司在2026年上半年的營運。我們報告稱，2025 年第一季度，netzo 補救措施的收入略低於 100 萬美元。正如保羅所提到的，這使我們自 2024 年 5 月推出以來的累計總銷售額達到約 350 萬美元。

Our R&D expenditures totaled $18.5 million for the first quarter, and our SGNA expenses were $15 million for the first quarter. And finally, we had a small amount of net income in the first quarter due to the recognition of $28 million in license and other revenue from our partnership with Norgine and a gain of $10.8 million on our outstanding Class C warrants, which are measured at fair value each quarter.

我們第一季的研發支出總計 1,850 萬美元，第一季的 SGNA 支出為 1,500 萬美元。最後，我們在第一季度獲得了少量淨收入，這是因為我們與 Norgine 的合作獲得了 2800 萬美元的許可和其他收入，並且我們的未償還 C 類認股權證獲得了 1080 萬美元的收益（這些認股權證每季度以公允價值計量）。

We also note that our 1 for 30 reverse stock split became effective on Monday following shareholder and board approval, and we believe this should cure our current deficiency with the NASDAQ listing rules. Lastly, we have not completed the majority of the actions we laid out during our announced strategic restructuring in February. We continue to expect that these efforts will decrease our spending by about $30million to $35 million annually.

我們也注意到，我們的 1 比 30 反向股票分割在股東和董事會批准後於週一生效，我們相信這應該可以解決我們目前在納斯達克上市規則的缺陷。最後，我們尚未完成二月份宣布的策略重組中所製定的大部分行動。我們繼續預期這些努力將使我們的支出每年減少約 3,000 萬至 3,500 萬美元。

We'll now open the call up to your questions, operator.

接線員，我們現在開始回答您的問題。

Thank you. (Operator Instructions) Ted Tenthoff with Piper Sandler.

謝謝。（操作員指示）Ted Tenthoff 與 Piper Sandler。

Great, thank you very much. Can you, give us a sense if you have any visibility into the types of patients that are being enrolled in for work? Is it largely in line with, what you expect in that you are enrolled in the few patients are, and then I follow up on, when.

太好了，非常感謝。您能否告訴我們，您是否了解正在接受治療的患者類型？這是否與您所期望的大致相符，因為您招募了少數患者，然後我會跟進何時。

I'm having a little trouble hearing you, but I think what color, the enrollment profile of the patients and then how that connects to the commercial market. So of course we mentioned that we're very easy to the overall profile but I class and mark.

我聽不太清楚你的意思，但我想問的是顏色、患者的入組情況以及它與商業市場有何關聯。因此，我們當然提到，我們很容易進行整體概況，但我會進行分類和標記。

So, and so we have, the study in itself is very has very specific criteria. And, we have the profile of the patients that we are having is really good so far, and we're tracking this. What I can share as anecdotally, having met with some of the PI and meeting with some others, is that a lot of those PIs would love to include many more patients than the patients we have into the study. And they often have, all these patients waiting, they cannot change their treatment just to include them in the study, obviously, but there's a clear demand here and interest in trying to bring as many patients as possible into the studies.

所以，這項研究本身有非常具體的標準。到目前為止，我們對患者的狀況非常好，我們正在追蹤這一點。我可以分享一些軼事，在與一些 PI 和其他一些人會面後，我發現許多 PI 都希望將比我們現有的患者更多的患者納入研究。他們經常讓所有這些等待的患者都參與進來，顯然，他們不能僅僅為了將他們納入研究而改變治療方法，但這裡有一個明確的需求，並且有興趣嘗試讓盡可能多的患者參與研究。

Yeah, great, thank you very much.

是的，太好了，非常感謝。

Sure, that's okay. Maybe Mark just has one more comment there.

當然可以。也許馬克只是再說一句話。

Yeah, Ted, I was just going to say, we're continuing to build our insights into the CN market itself and actually just completed a large survey with about 95 US physicians who treat. Chronic neutropenia and what we're finding is the case loads of patients that these physicians have are much higher than in WHIMs. So in WHIM it's a very fragmented model, but in chronic neutropenia it's much more concentrated, much more defined because there are distinct definitive ICD 10 diagnosis codes. So we can clearly see where these patients are and the unmet need in in this refractory population.

是的，泰德，我只是想說，我們正在繼續深入了解 CN 市場本身，實際上剛剛對大約 95 名美國治療醫生完成了一項大型調查。慢性中性粒細胞減少症，我們發現這些醫生的病例數比 WHIM 的病例高得多。因此，在 WHIM 中，它是一個非常分散的模型，但在慢性中性粒細胞減少症中，它更加集中，更加明確，因為有明確的 ICD 10 診斷代碼。因此，我們可以清楚地看到這些患者在哪裡以及這一難治性人群中未滿足的需求。

So I think just to quickly summarize, you said, very sick patients being enrolled in the study to enrich for success on the infection end point, higher demand, both locally in the trial because they'd love to get their patients in the trial, but of course we can't accept everybody and then market seeing that pull through in terms of the higher caseloads through our market research.

因此，我認為只需快速總結一下，您說的，重症患者參與研究是為了在感染終點方面取得成功，對當地試驗的需求更高，因為他們希望讓他們的患者參與試驗，但當然我們不能接受所有人，然後市場通過我們的市場研究看到更高的病例量。

Great, that's very helpful. Thank you.

太好了，這非常有幫助。謝謝。

Thank you, Ted.

謝謝你，泰德。

Thank you. RK with HC Wainwright.

謝謝。RK 與 HC Wainwright。

Thank you. Good morning, Paula and Adam. Couple of quick questions. In the, And the patient, the amount of patients that you said who are being diagnosed with CN, which is like 50,000 people, and out of that 15,000 are the ones that seem to be having the high unmet need. These numbers, are they just US or they worldwide?

謝謝。早安，Paula 和 Adam。幾個簡單的問題。至於患者，您說的被診斷患有 CN 的患者數量大約有 50,000 人，而在這 15,000 人中，似乎存在大量未滿足的需求。這些數字僅僅是美國的還是全世界的？

They're just the US okay, yes, we did IPD 0 code analysis and the US claims data.

他們只是美國，是的，我們做了 IPD 0 代碼分析和美國索賠數據。

Okay. Okay, perfect. And then, from your, comments to Ted's question, you were stating that only very sick patients are being enrolled into the study. So I'm thinking about the label. When, what sort of the target population would you be looking at in, on the label? Is it the very sick population or is that beyond that? And how do we define that population?

好的。好的，完美。然後，從您對 Ted 的問題的評論來看，您說只有病情非常嚴重的患者才會參加這項研究。所以我在考慮標籤。什麼時候，您會在標籤上關注什麼樣的目標人群？是病情嚴重的人群，還是其他人群？那我們要如何定義這個群體呢？

So because, okay, this is Christoph here, because we do have already also from the CN phase two experience with also other population, even if we're, if the phase 3 study, includes moderate and severe patients, we are going to build a case for the label. For the entirety and population, and we do have already data to support this, including from the win study, etc. So, we are anticipating a broad level, but obviously this will be a matter of discussions with the SD at the time when it comes.

所以因為，好的，這是 Christoph，因為我們確實已經從 CN 第二階段的經驗中獲得了其他人群的經驗，即使我們，如果第三階段的研究包括中度和重度患者，我們也會為標籤建立一個案例。對於整體和人口而言，我們確實已經有數據來支持這一點，包括來自勝利的研究等。因此，我們預計會達到廣泛的水平，但顯然這將是與 SD 討論的問題。

Yeah. But I mean, our case is to kind of crosswalk obviously into the value proposition. We are focusing on treating patients who are basically refractory with severe recurrent infections. That is who needs remaining treatments. And certainly when we go into the payer systems across the world, we want to be focusing on that, basically. High-end met need with little to no options to demonstrate the value proposition of Xolremdi for mavericks for right now is the end Xolremdi and women maintain our price point. So there's a good connection between the severity of the disease, the trial design, and the ultimate value that we think we can bring to patients.

是的。但我的意思是，我們的案例顯然是跨入價值主張的。我們專注於治療那些基本上難以治癒且感染反覆嚴重的患者。這就是需要剩餘治療的人。當然，當我們進入世界各地的付款系統時，我們基本上希望關注這一點。高端滿足需求，幾乎沒有選擇來證明 Xolremdi 對特立獨行者的價值主張，目前是 Xolremdi 和女性維持我們的價格點的終點。因此，疾病的嚴重程度、試驗設計以及我們認為可以為患者帶來的最終價值之間存在著良好的關聯。

Okay. One last question, I'm sorry. The transaction with the, or the agreement with Norgine that you currently have, for, commercialization in Europe and Australia, does that go by indication or does that go by mavericks for period?

好的。最後一個問題，抱歉。您目前與 Norgine 達成的、針對歐洲和澳洲商業化的交易或協議是按照指示進行的，還是按照特定時期進行的？

Yeah, thanks Adam. So it covers WHIM and CN so it's a license to the across both indications. Obviously first would be when commercially followed by CNN.

是的，謝謝亞當。因此它涵蓋了 WHIM 和 CN，所以它是跨越兩種適應症的許可證。顯然，首先出現的是 CNN 的廣告。

Okay, perfect. Thanks. Thanks for taking my questions.

好的，完美。謝謝。感謝您回答我的問題。

Thank you, RK.

謝謝你，RK。

Thank you. Stephen Willey with Stifel.

謝謝。史蒂芬威利 (Stephen Willey) 與 Stifel 合作。

Yeah, good morning, thanks for taking the questions.

是的，早上好，感謝您回答問題。

I guess with the understanding that the commercial history here in web is a bit abbreviated thus far, is there anything that you can say about just patient persistency and compliance that you're seeing?

我想，考慮到迄今為止網路商業歷史的簡短程度，您能否談談您所看到的患者堅持和依從性？

Sure, good morning, Steph, Mark here. Yeah, I mean, I think what we're pleased with is that although we're not giving out any actual numbers, we're pleased to see that compliance and adherence rates are actually higher than you would expect with a daily oral medication. And I think that speaks to, the unmet need here and that these patients and physicians, understand that this disease needs treatment and, Xolremdi is the solution, if it's taken appropriately. So, we're now actually spending a lot of time beginning to educate patients, and the patient community, and, in fact, excited to announce that we just launched a new website yesterday. In fact, I encourage you to open. Your browser of choice and type in winsyndrome.com and you'll see our new patient education website that features WHIM patients telling their stories and also provides, a lot of resources to help them on their journey with WHIM and with Xolremdi.

當然，早上好，史蒂芬，我是馬克。是的，我的意思是，我認為我們感到高興的是，儘管我們沒有給出任何實際數字，但我們很高興地看到依從性和堅持率實際上高於每日口服藥物的預期。我認為這說明了這裡存在未滿足的需求，這些患者和醫生都明白這種疾病需要治療，如果服用得當，Xolremdi 是解決方案。因此，我們現在實際上花了大量時間開始教育患者和患者社區，事實上，我們很高興地宣布，我們昨天剛剛推出了一個新網站。事實上，我鼓勵你開放。您選擇的瀏覽器並輸入 winsyndrome.com，您將看到我們的新患者教育網站，該網站以 WHIM 患者講述他們的故事為特色，還提供了大量的資源來幫助他們使用 WHIM 和 Xolremdi。

Okay. And are most patients getting a 30 day supply, or are some patients getting 3 months' worth of drug via a single script?

好的。大多數患者是否能獲得 30 天的藥量，或者有些患者是否透過一次處方就能獲得 3 個月的藥量？

The majority of patients are on the higher dose, of course, it's weight-based, so, if the patient is of a lighter weight, they get a lighter dose.

大多數患者服用的劑量較高，當然，這取決於體重，因此，如果患者體重較輕，則服用的劑量也較輕。

And I guess the question was, if the prescription unit size that most patients are getting, is it a month's supply or are some patients getting 3 months' worth of drugs with a single prescription.

我想問題是，大多數患者獲得的處方單位大小是一個月的供應量，還是有些患者只需一張處方就能獲得三個月的藥物量。

It's a month supply at this stage.

目前這是一個月的供應量。

Okay. And then understanding that the forward trial is blinded.

好的。然後了解到前向試驗是盲目的。

But can you just remind us what your assumption was around patient drop out and if there's any data that you're able to see on a blinded basis that would suggest that that assumption is holding up in in the clinical trial.

但是，您能否提醒我們，您對患者退出的假設是什麼，以及您是否能夠在盲法基礎上看到任何數據，表明該假設在臨床試驗中成立。

Yeah, so, Steph, what we're targeting as we shared is about 150 patients and roles and all of that has some degree of assumptions of either dropouts, or screening failures, etc. So, we're building that funnel. I think more importantly, what we can see with the enrolled subjects is really about their profile like are we in good shape on the 150 because that sort of sets our clock. The answer there is definitely yes.

是的，Steph，正如我們分享的那樣，我們的目標是約 150 名患者和角色，所有這些都在一定程度上假設了退出或篩檢失敗等。所以，我們正在建立這個漏斗。我認為更重要的是，我們可以從登記的受試者身上看到他們的概況，例如我們在 150 歲時的狀態是否良好，因為這可以設定我們的時鐘。答案肯定是肯定的。

We're seeing the. Right blend of idiopathic, autoimmune and congenital, it's balanced nicely, and then, of course, this is all blinded, but we're seeing the event rates that you would want to see, early in the study to confirm our assumptions. So in terms of powering that the 150 is belts and suspenders for the co-primary endpoint. So I have to give that give you some confidence in terms of the rate, there's so many ins and outs, what we can say is certainly based on everything we're seeing, we're on track for that Q3, Q4 and full enrollment.

我們看到了。特發性、自體免疫性和先天性的正確組合，達到了很好的平衡，當然，這都是盲法的，但我們在研究早期就看到了您希望看到的事件發生率，以證實我們的假設。因此，從動力方面來看，150 是共同主要終點的安全帶和吊帶。因此，我必須讓大家對這個比率有信心，因為有太多的細節，我們可以肯定地說，基於我們所看到的一切，我們正朝著第三季、第四季和全面招生的目標前進。

Okay. Very helpful. Thanks for taking the questions.

好的。非常有幫助。感謝您回答這些問題。

Thank you, Steph

謝謝你，史蒂芬

Thank you. And we show no further questions at this time. I will turn the call back to Paula Reagan for closing remarks.

謝謝。目前我們沒有其他問題。我將把電話轉回給 Paula Reagan 做最後發言。

Well, thank you very much for joining us today. I'm happy to follow up offline with any other questions and wish you all an excellent rest of your day.

嗯，非常感謝您今天加入我們。我很樂意離線跟進任何其他問題，並祝大家有個愉快的一天。

Thank you.

謝謝。

And this does conclude today's program.

今天的節目到此結束。

Thank you for your participation. You may disconnect at any time.

感謝您的參與。您可以隨時斷開連線。