INmune Bio Inc (INMB) 2025 Q1 法說會逐字稿

Greetings, and welcome to the INmune Bio first quarter 2025 earnings call. (Operator Instructions) As a reminder, this conference is being recorded. A transcript will follow within 24 hours of this conference call.

大家好，歡迎參加 INmune Bio 2025 年第一季財報電話會議。（操作員指示）提醒一下，本次會議正在錄音。本次電話會議結束後 24 小時內將提供會議記錄。

At this time, it is my pleasure to introduce Mr. David Moss, CFO of INmune Bio. David?

現在，我很高興介紹INmune Bio的財務長David Moss先生。戴維？

Thank you, Jesse, and good afternoon, everyone. We thank you for joining us for the call for INmune Bio's first quarter 2025 financial results. With me on the call today are Dr. RJ Tesi, CEO of INmune Bio; and Dr. Mark Lowdell, Chief Scientific Officer of INmune Bio, who will provide an update on our CORDStrom and INKmune programs.

謝謝你，傑西，大家下午好。感謝您參加 INmune Bio 2025 年第一季財務業績電話會議。今天與我一起參加電話會議的還有 INmune Bio 執行長 RJ Tesi 博士和 INmune Bio 首席科學官 Mark Lowdell 博士，他們將介紹我們的 CORDStrom 和 INKmune 計劃的最新進展。

Also on the call is Dr. CJ Barnum, Head of Neuroscience, who will be here to answer questions. Before we begin, I remind everyone that except for statements of historical facts, the statements made by management and responses to questions on this conference call are forward-looking statements under the Safe Harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements involve risks and uncertainties that can cause actual results to differ materially from those such as forward-looking statements. Please see the forward-looking statements disclaimer on the company's earnings press release, as well as risk factors in the company's SEC filings, including our most recent quarterly filings with the SEC. There's no assurance of any specific outcome.

神經科學主任 CJ Barnum 博士也將參加電話會議並回答問題。在我們開始之前，我提醒大家，除了歷史事實陳述外，管理層在電話會議上所作的陳述和對問題的回答都是根據 1995 年《私人證券訴訟改革法》安全港條款做出的前瞻性陳述。這些聲明涉及風險和不確定性，可能導致實際結果與前瞻性聲明的結果有重大差異。請參閱公司收益新聞稿中的前瞻性聲明免責聲明，以及本公司向美國證券交易委員會提交的文件中的風險因素，包括我們最近向美國證券交易委員會提交的季度文件。沒有任何具體成果的保證。

Undue reliance should not be placed on forward-looking statements, which speak only as of the date they are made as the facts and circumstances underlying these forward-looking statements may change. Except as required by law, INmune Bio disclaims any obligation to update these forward-looking statements to reflect future information, events, or circumstances.

不應過度依賴前瞻性陳述，這些陳述僅代表其作出之日的觀點，因為這些前瞻性陳述所依據的事實和情況可能會改變。除法律要求外，INmune Bio 不承擔更新這些前瞻性聲明以反映未來資訊、事件或情況的義務。

With that behind us, now it's my pleasure to turn the call over to RJ Tesi. RJ?

談完這些之後，現在我很高興將發言權交給 RJ Tesi。RJ？

Thank you, David. For our first quarter 2025 call, I will review key takeaways and provide an update on our platform programs. Following my comments on recent developments, Dr. Mark Lowdell, INmune Bio's CSO and inventor of both CORDStrom and INKmune, will provide an update on those programs. David Moss, INmune Bio's CFO, will then conclude with a review of first-quarter financial results and update future catalysts. Then we will be happy to take your questions.

謝謝你，大衛。在 2025 年第一季的電話會議上，我將回顧關鍵要點並提供有關我們平台計劃的最新資訊。在我對最新進展發表評論之後，INmune Bio 的 CSO 兼 CORDStrom 和 INKmune 的發明者 Mark Lowdell 博士將提供有關這些計劃的最新資訊。INmune Bio 財務長 David Moss 將總結第一季的財務表現並更新未來的催化劑。我們將很樂意回答您的問題。

I'm sure everyone on this call knows we will soon be reporting top-line results from MINDFuL. That is our Phase II trial in patients with early Alzheimer's disease. The results are expected mid to late June. That is in, give or take, 50 days, we will know the answer to the question, what happens in Alzheimer's disease when you properly target neuroinflammation.

我相信參加這次電話會議的每個人都知道我們很快就會報告 MINDFuL 的最終結果。這是我們對早期阿茲海默症患者進行的第二階段試驗。預計結果將於六月中旬至下旬公佈。也就是說，大約 50 天后，我們就會知道這個問題的答案：當你正確針對神經發炎時，阿茲海默症會發生什麼事。

Our last investor update call was just a short six weeks ago, but there have been important, and I believe, positive changes in the AD market -- Alzheimer's disease marketplace during that short period of time. We believe these changes will be beneficial to XPro's market opportunity in early Alzheimer's disease.

我們上一次與投資者召開最新電話會議是在短短六週前，但我相信，在那短短的時間內，AD 市場——阿茲海默症市場發生了重要的積極變化。我們相信這些變化將有利於XPro在早期阿茲海默症領域的市場機會。

Last month at AD/PD, which is the largest Alzheimer's disease meeting in Europe, INmune Bio reported the biomarker profile of patients enrolled in the MINDFuL study. The data confirmed that we have been underestimating the market opportunity for XPro in patients with early AD. Historically, we stated that up to half of the early Alzheimer's patients will qualify for XPro based on the biomarkers we used as our enrollment criteria.

上個月，在歐洲最大的阿茲海默症會議 AD/PD 上，INmune Bio 報告了參與 MINDFuL 研究的患者的生物標記概況。數據證實，我們一直低估了XPro在早期阿茲海默症患者中的市場機會。我們曾表示，根據我們用作入組標準的生物標誌物，多達一半的早期阿茲海默症患者將符合XPro的治療資格。

Based on the data INmune Bio and other companies presented at AD/PD, we now believe more than two-thirds of early Alzheimer's disease patients will be eligible for XPro based on ApoE4 status alone. I remind you that ApoE4 positivity was one of the four enrichment or enrollment criteria we used in the MINDFuL study. The ApoE4 status of patients in MINDFuL is almost identical to what is reported in recent major trials in Alzheimer's. Patients with at least one ApoE4 allele make up more than two thirds of the patients in these trials. This means the market opportunity for XPro in early AD has increased to nearly 70% of early AD patients, not the 40% we have previously been talking about.

根據 INmune Bio 和其他公司在 AD/PD 上展示的數據，我們現在認為，僅基於 ApoE4 狀態，超過三分之二的早期阿茲海默症患者將有資格接受 XPro 治療。我提醒您，ApoE4 陽性是我們在 MINDFuL 研究中使用的四個豐富或登記標準之一。MINDFuL 中患者的 ApoE4 狀態與最近阿茲海默症主要試驗中報告的狀態幾乎相同。在這些試驗中，攜帶至少一個 ApoE4 等位基因的患者佔患者的三分之二以上。這意味著 XPro 在早期 AD 中的市場機會已增加到早期 AD 患者的近 70%，而不是我們之前談論的 40%。

On the call six weeks ago, I also mentioned the safety profile of XPro in the MINDFuL trial. Nothing has changed. There are no reports of area. No patients have had unscheduled MRIs due to CNS symptoms or headache, et cetera, and there have been no deaths. So far, XPro is safe and well tolerated in this patient population that has an average age of 73 years old, and many of them have a long list of comorbidities.

在六週前的電話會議上，我還提到了 XPro 在 MINDFuL 試驗中的安全性。一切都沒有改變。沒有關區域的報告。沒有患者因為中樞神經症狀或頭痛等原因而接受未安排的 MRI 檢查，也沒有出現死亡病例。到目前為止，XPro 對於平均年齡為 73 歲的患者群體來說是安全的，並且耐受性良好，其中許多人患有多種合併症。

The excellent safety profile of XPro in these patients provides a unique ApoE4-related market opportunity for XPro. Let me explain. Both the EU and the UK have approved lecanemab, the Eisai-Biogen drug, for patients with early Alzheimer's disease who have none or one copy of the ApoE4 gene. The market authorizations specifically exclude patients who carry two ApoE4 alleles.

XPro 在這些患者中表現出的出色安全性為 XPro 提供了獨特的 ApoE4 相關市場機會。讓我解釋一下。歐盟和英國都已批准衛材-百健（Eisai-Biogen）公司的藥物 lecanemab，用於治療沒有或只有一個 ApoE4 基因拷貝的早期阿茲海默症患者。市場授權明確排除攜帶兩個 ApoE4 等位基因的患者。

In the early AD trials that report ApoE4 status, ApoE4 homozygotes, that's the patients that have two ApoE4 alleles, are 15% of the patients. That means because of labeling restrictions on lecanemab in the UK and EU, early Alzheimer's patients who are ApoE4 homozygotes will not be eligible for therapy with the anti-amyloid drugs. This group now is an important unmet need ideally suited for XPro therapy. In the US, recent surveys of practice patterns indicate that this patient population is not treated in many centers due to the risk variance.

在報告 ApoE4 狀態的早期 AD 試驗中，ApoE4 純合子（即具有兩個 ApoE4 等位基因的患者）佔患者的 15%。這意味著，由於英國和歐盟對 lecanemab 的標籤限制，ApoE4 純合子的早期阿茲海默症患者將沒有資格接受抗澱粉樣蛋白藥物治療。該群體目前存在重要的未滿足需求，非常適合 XPro 療法。在美國，最近的實踐模式調查表明，由於風險差異，許多中心並未對此類患者群體進行治療。

So even in the US, where the labeling is different than you see in the UK and Europe, we believe that after approval, XPro will be the best and only treatment option available for this subgroup, important subgroup of early Alzheimer's patients. We should have an exclusive biologically based market.

因此，即使在美國，其標籤與英國和歐洲不同，我們相信，獲得批准後，XPro 將成為這一亞群（早期阿茲海默症患者的重要亞群）可用的最佳且唯一的治療選擇。我們應該擁有一個專屬的生物市場。

Finally, the biomarker landscape of Alzheimer's disease is really evolving quite quickly, and it's evolving in a way that highly -- that really benefits our focus in these patients. Now once the diagnosis of Alzheimer's is made, pTau217 in blood has become the biomarker of most important interest by clinical teams treating these early Alzheimer's patients. pTau217 levels define the severity of Alzheimer's. pTau217 levels in the blood have prognostic value and correlate with stage of disease. In the near future, we predict that changes in pTau217 blood levels will be used as a pharmacodynamic blood marker of therapeutic response in these patients. As a reminder, XPro significantly decreased pTau217 during the three-month Phase I study, and this biomarker is included in the package of biomarkers that we are studying as in the MINDFuL Phase II program.

最後，阿茲海默症的生物標記模式確實正在迅速發展，而且其發展方式非常有利於我們關注這些患者。現在，一旦確診為阿茲海默症，血液中的 pTau217 就成為治療早期阿茲海默症患者的臨床團隊最感興趣的生物標記。 pTau217 水平決定了阿茲海默症的嚴重程度。血液中的 pTau217 水平具有預後價值，並與疾病的分期相關。在不久的將來，我們預測 pTau217 血液水平的變化將被用作這些患者治療反應的藥效學血液標記。提醒一下，XPro 在為期三個月的第一階段研究中顯著降低了 pTau217，並且該生物標誌物包含在我們正在研究的 MINDFuL 第二階段計劃中的生物標誌物包中。

Having a great drug is a necessary element for a successful clinical program, but it is only part of the story. Since the last patient was enrolled in November, we have been highlighting the hard work needed to report the top line data in June. These are the busiest of times. For example, after the last patient has their final safety visit but before the database is locked, a complex series of critical data management and quality assurance tasks are undertaken to ensure the data are complete, accurate, and ready for analysis. The process begins with data cleaning, where we review case report forms and entries into the electronic data capture system and to resolve discrepancies, fill in missing data and look for outliers. This is a patient-by-patient process. It is labor-intensive.

擁有好藥是臨床計畫成功的必要因素，但這只是故事的一部分。自 11 月最後一位患者入組以來，我們一直在強調報告 6 月頂線數據所需的艱苦工作。這是最忙碌的時刻。例如，在最後一位患者進行最後一次安全存取後但在資料庫鎖定之前，需要執行一系列複雜的關鍵資料管理和品質保證任務，以確保資料完整、準確且可供分析。這個過程從資料清理開始，我們審查病例報告表和電子資料收集系統中的條目，以解決差異、填寫缺失資料並尋找異常值。這是一個針對每個患者的過程。它是勞力密集型的。

Queries are issued to trial sites to clarify inconsistencies and there are source data verification to confirm that the recorded data matches the source documents, for instance, the medical records. This phase also involves ensuring compliance with regulatory standards such as GCP by maintaining [loss] trails and documenting all changes.

向試驗地點發出查詢以澄清不一致之處，並進行來源資料驗證以確認記錄的資料與來源文件（例如醫療記錄）相符。此階段還涉及透過維護[損失]軌跡和記錄所有變化來確保遵守 GCP 等監管標準。

After data cleaning are complete, the focus shifts to the final quality checks and preparation for database lock. A comprehensive review of the data is performed to confirm that all queries are resolved, deviation is documented and validation checks satisfied. The statistical plan cross check to ensure that all data points are present and correctly formatted and the data monitoring committees conduct final safety and efficacy reviews. We don't lock the database until we are sure the database is clean and accurate. I will also say that this is all done with blinded data. No one knows who got what during this process

資料清理完成後，重點轉移到最終的品質檢查和資料庫鎖定的準備。對數據進行全面審查，以確認所有疑問都已解決、偏差已記錄且驗證檢查已滿足。統計計劃經過交叉檢查，以確保所有數據點都存在且格式正確，並且數據監測委員會進行最終的安全性和有效性審查。在我們確定資料庫乾淨且準確之前，我們不會鎖定資料庫。我還要說的是，這一切都是透過盲數據完成的。沒有人知道在這個過程中誰得到了什麼

We don't run the statistical programs until the database are locked. And it's only when that statistical package comes out that the real unblinding occurs. We remain on track for this process to be completed in mid- to late June. And I can tell you we can't hardly wait to see the fruits of our labors. This is the moment we've been anticipating for several years. We are confident we will report results that will change the care of patients with early Alzheimer's disease. And this confidence is highlighted by management's substantial share ownership. Like you, we are investors in INmune Bio. Our interests are aligned with yours.

直到資料庫被鎖定時，我們才會執行統計程式。只有當統計數據出來後，真正的揭盲才會發生。我們仍有望在六月中旬至下旬完成這項進程。我可以告訴你們，我們迫不及待想看到我們勞動的成果。這是我們多年來一直期盼的時刻。我們有信心報告的結果將改變早期阿茲海默症患者的照護。管理階層持有的大量股份凸顯了這種信心。和您一樣，我們也是 INmune Bio 的投資者。我們的利益與您的利益一致。

As important as MINDFuL, our Alzheimer's trial, is to the company and our investors. It's not our only program. CORDStrom for the treatment of children with recessive dystrophic epidermolysis bullosa, or RDEB, is expected to file a BLA in 2026. The INKmune program continues to move forward in men with metastatic castrate-resistant prostate cancer. The light is shining more brightly on CORDStrom these days. It is an orphan disease program.

我們的阿茲海默症試驗 MINDFuL 對公司和我們的投資者同樣重要。這不是我們唯一的項目。CORDStrom 用於治療隱性營養不良型大皰性表皮鬆解症（RDEB）的兒童，預計將於 2026 年提交 BLA。INKmune 計畫在轉移性去勢抵抗性前列腺癌患者中持續進行。最近，CORDStrom 的光芒更加明亮了。這是一項孤兒病計畫。

And after recent comments from the FDA, we are increasingly excited by its prospects. The FDA has stated their intention to move rare disease treatments through the approval process faster with more input from patients and caregivers. In RDEB, CORDStrom provides a systemic therapy for a systemic genetic disease that has the support of patients and caregivers. But enough from me.

在聽取了 FDA 最近的評論後，我們對其前景越來越感到興奮。FDA 表示，他們打算透過更多聽取患者和照護者的意見來加速罕見疾病治療的審批程序。在 RDEB 中，CORDStrom 為系統性遺傳疾病提供系統性治療，並得到患者和照護者的支持。但我說的已經夠多了。

I will turn this over -- the microphone over to Mark Lowdell, the CSO and inventor of both CORDStrom and INKmune, to give you more in-depth update on those programs. Mark?

我將把麥克風交給 CORDStrom 和 INKmune 的首席策略長兼發明者 Mark Lowdell，以便為大家提供有關這些程序的更深入的更新資訊。標記？

Thanks, RJ, and good afternoon, everyone, and thank you for joining us. So with regard to the latest developments with INKmune, the CaRe PC trial in prostate cancer completed the Phase I dose escalation cohorts in December, which allowed us to open the Phase II extensions of both the high dose and intermediate dose cohorts in parallel, and we reported that in our last call.

謝謝，RJ，大家下午好，謝謝你們加入我們。因此，關於 INKmune 的最新進展，前列腺癌的 CaRe PC 試驗於 12 月完成了 I 期劑量遞增隊列，這使我們能夠同時啟動高劑量和中劑量隊列的 II 期擴展，我們在上次電話會議上報告了這一點。

We saw no adverse events in any of the patients treated during Phase I, which thus met the primary end point of the entire trial. And similarly, none of the patients treated in Phase II to date has shown any adverse event, and INKmune remains extremely well tolerated in this challenging and elderly group of patients who've had lots of previous treatment and a lot of comorbidities. At the end of March, we reported that INKmune infusions had led to increased NK cell potency in the patients treated at the lowest dose and the blood samples from patients at the intermediate and high-dose cohorts of Phase I and Phase II are being received in the INmune Bio labs in London and are being prepared for testing.

我們在第一階段接受治療的患者中沒有發現任何不良事件，因此達到了整個試驗的主要終點。同樣，迄今為止，接受 II 期治療的患者均未出現任何不良事件，並且 INKmune 在這一具有挑戰性的老年患者群體中仍然具有極好的耐受性，這些患者之前接受過大量治療，並且患有許多合併症。3 月底，我們報告稱，INKmune 輸注已導致接受最低劑量治療的患者的 NK 細胞效力增強，並且 I 期和 II 期中劑量和高劑量組的患者的血液樣本正在倫敦的 INmune Bio 實驗室接收並準備進行測試。

We'll share those results as soon as they're available. But in parallel, we're receiving the independent reports of the PSMA PET screening of the Phase I patients who've completed follow-up. This is a very precise assay of the sizes of individual tumor lesions in each patient before treatment and at three months after completion of INKmune treatment. This very complex data set is being reviewed by the lead clinician for CaRe PC, but we can already see from subjects in the lowest dose cohort that some lesions have resolved completely following INKmune treatment. We eagerly await the data from the intermediate and high-dose patients, which we'll share as soon as they become available.

一旦結果出來，我們會立即分享。但同時，我們正在收到已完成追蹤的 I 期患者的 PSMA PET 篩檢的獨立報告。這是對每個患者在治療前和完成 INKmune 治療三個月後單一腫瘤病變大小的非常精確的分析。CaRe PC 的首席臨床醫生正在審查這個非常複雜的數據集，但我們已經可以從最低劑量組的受試者中看到，一些病變在接受 INKmune 治療後已經完全消退。我們熱切期待中劑量和高劑量患者的數據，一旦獲得這些數據，我們將立即分享。

The CaRe PC trial continues to recruit Phase II subjects and remains on track to complete enrollment this year. The INmune Bio team has manufactured all doses of INKmune for the completion of the trial, and we've successfully transitioned the US drug supply logistics from our previous contractor into a US company called Cryoport. So we're set up for the successful conclusion of the trial.

CaRe PC 試驗繼續招募第二階段受試者，並預計在今年完成招募。INmune Bio 團隊已經生產了完成試驗所需的所有劑量的 INKmune，並且我們已成功將美國藥品供應物流從先前的承包商轉移到一家名為 Cryoport 的美國公司。因此，我們已為成功完成審判做好了準備。

In line with our ambitions for INKmune in prostate cancer and other solid tumors, we're transitioning our manufacturing into a UK government incubator facility where we can manufacture future drug batches for clinical trial and if successful, commercial manufacture and global supply.

為了實現 INKmune 在前列腺癌和其他實體腫瘤領域的目標，我們正在將生產轉移到英國政府孵化器設施，在那裡我們可以生產未來用於臨床試驗的藥物批次，如果成功，還可以進行商業化生產和全球供應。

But while CaRe PC is ongoing, in February, as RJ just said, we announced the development of our CORDStrom asset towards a BLA filing next year, 2026, for the treatment of the extremely debilitating disease, recessive dystrophic epidermolysis bullosa or RDEB. Epidermolysis bullosa, which RDEB is the most severe form, is an inherited disease, which manifests in the first two years of life. The outer layer of the skin, the epidermis, doesn't anchor properly to the underlying dermis, and children suffer horrific skin blisters over their whole body. Worse still is that EB is a systemic disease, as RJ said, so children suffer with lesions behind their eyes, down their esophagus, and throughout their gastrointestinal tract. There's no systemic treatment for RDEB.

但是，雖然 CaRe PC 仍在進行中，但正如 RJ 剛才所說，我們在二月份宣布開發 CORDStrom 資產，以便在明年（2026 年）提交 BLA 申請，用於治療一種極其衰弱的疾病——隱性營養不良型大皰性表皮鬆解症或 RDEB。大皰性表皮鬆解症（RDEB 是最嚴重的形式）是一種遺傳性疾病，在生命的頭兩年內出現症狀。皮膚的外層，即表皮，無法正確地固定在下面的真皮上，導致兒童全身出現可怕的皮膚水泡。更糟的是，正如 RJ 所說，EB 是一種全身性疾病，因此兒童的眼睛後方、食道和整個胃腸道都會受到病變的困擾。目前尚無針對 RDEB 的系統治療方法。

CORDStrom is an off-the-shelf allogeneic mesenchymal stromal cell drug from pooled donors, which INmune Bio has owned since 2019. It was developed by me and some colleagues with academic funding in the UK and is a platform that can be used to treat multiple clinical indications by specific selection of the individual donor products used to formulate the final pooled drug. We've been selling one formulation of CORDStrom into the UK multicenter trial led by pediatricians from Great Ormond Street Children's Hospital in London to treat children with RDEB.

CORDStrom 是一種來自集中捐贈者的現成同種異體間質基質細胞藥物，自 2019 年起由 INmune Bio 擁有。它是由我和一些同事在英國獲得學術資助而開發的，是一個可以透過特定選擇用於配製最終混合藥物的個人捐贈產品來治療多種臨床適應症的平台。我們一直在向英國多中心試驗出售一種 CORDStrom 配方，該試驗由倫敦大奧蒙德街兒童醫院的兒科醫生領導，用於治療患有 RDEB 的兒童。

This was a double-blind, placebo-controlled crossover trial, which treated all of the eligible children in England with RDEB. The results, when they were unblinded, led to the clinicians and the UK National Institute of Health Research asking INmune Bio to develop CORDStrom as a licensed medicine for this disease, and we presented the trial data to the USFDA in December, as RJ said. The FDA data review led to the award of rare pediatric disease and orphan drug status for the treatment of epidermolysis bullosa. And following a Type C meeting with the FDA in February, we have a clear route to submission of a BLA in 2026 and parallel submissions to the UK MHRA and to the European Medicines Agency.

這是一項雙盲、安慰劑對照交叉試驗，對英格蘭所有符合條件的 RDEB 兒童進行了治療。當結果揭曉後，臨床醫生和英國國立衛生研究院要求 INmune Bio 將 CORDStrom 開發為這種疾病的許可藥物，並且我們於 12 月向美國食品藥品監督管理局提交了試驗數據，正如 RJ 所說。FDA 的數據審查授予該藥物用於治療大皰性表皮鬆解症的罕見兒科疾病和孤兒藥地位。在二月與 FDA 舉行 C 類會議之後，我們明確了在 2026 年提交 BLA 的路線，並同時向英國 MHRA 和歐洲藥品管理局提交申請。

On our last call, we said that clinical trials are the poster children of drug development. But moving from trials to market requires much more, not least a full development of the drug manufacturing pathway, which meets regulatory requirements, is cost effective at scale and meets likely global demand. We've taken this onboard with respect to CORDStrom in the same way we have with INKmune and in fact, have developed parallel processes for both drugs, which can share the same manufacturing platforms at all stages of manufacture. This not only simplifies manufacturing facility setup and design, but it allows us to maximize the use of manufacturing space we rent by being able to switch between INKmune production and CORDStrom manufacture, thus controlling our production costs as we move towards commercial supply.

在我們上次的電話會議中，我們說臨床試驗是藥物開發的典型代表。但從試驗到市場，還需要做更多工作，尤其是要全面開發藥品製造途徑，以滿足監管要求、規模效益和滿足可能的全球需求。我們在 CORDStrom 中採用了與 INKmune 相同的方法，事實上，我們已經為這兩種藥物開發了平行流程，可以在製造的所有階段共享相同的製造平台。這不僅簡化了製造設施的設置和設計，而且使我們能夠透過在 INKmune 生產和 CORDStrom 製造之間切換來最大限度地利用我們租用的製造空間，從而在走向商業供應的過程中控制我們的生產成本。

Having used the income from selling CORDStrom into the UK trial in EB to subsidize the supply of INKmune into CaRe PC, the equipment we've been using to manufacture INKmune is now subsidizing the development of CORDStrom, which is a nice, very circular situation. Having completed all manufacture of INKmune for CaRe PC, the UK team is dedicated to providing all of the data needed for the BLA submission next year for CORDStrom and transitioning into the new production space to allow CORDStrom as a commercial product and INKmune as an investigational product for our next clinical trials.

我們利用英國 EB 試驗中銷售 CORDStrom 的收入來補貼向 CaRe PC 供應 INKmune，而我們用來製造 INKmune 的設備現在又用來補貼 CORDStrom 的開發，這是一種很好的、循環的局面。在完成 INKmune 用於 CaRe PC 的所有製造後，英國團隊致力於為 CORDStrom 提供明年提交 BLA 所需的所有數據，並過渡到新的生產空間，以允許 CORDStrom 作為商業產品，INKmune 作為我們下一次臨床試驗的試驗產品。

So that ends my update on the INKmune and CORDStrom platforms. Just to say we're as excited about these as we are about the AD02 trial.

我對 INKmune 和 CORDStrom 平台的更新到此結束。我想說的是，我們對這些感到興奮，就像對 AD02 試驗一樣。

And I'd like to turn the call over to David Moss, our CFO, to discuss financials. David?

我想將電話轉給我們的財務長戴維·莫斯 (David Moss)，討論財務問題。戴維？

Thank you, Mark. As usual, I'll provide a brief overview of our financial results and upcoming milestones before we head to our Q&A session. Net loss attributable to common stockholders for the quarter ended March 31, 2025, was approximately $9.7 million compared with approximately $11 million for the comparable period in '24. Research and development expenses totaled approximately $7.6 million for the quarter ended March 2025 compared with approximately $8.7 million for the comparable period in 2024. General and administrative expenses were approximately $2.3 million for the quarter ended March 31, 2025, compared with approximately $2.3 million for the comparable period in 2024.

謝謝你，馬克。像往常一樣，在進入問答環節之前，我將簡要概述我們的財務表現和即將到來的里程碑。截至 2025 年 3 月 31 日的季度，普通股股東淨虧損約為 970 萬美元，而 2024 年同期約為 1,100 萬美元。截至 2025 年 3 月的季度，研發費用總計約為 760 萬美元，而 2024 年同期約為 870 萬美元。截至 2025 年 3 月 31 日的季度，一般及行政費用約為 230 萬美元，而 2024 年同期約為 230 萬美元。

At March 31, 2025, the company had cash and cash equivalents of approximately $19.3 million. Based on our current operating plan, we believe our cash is sufficient to fund our operations through Q3 of 2025. As of May 8, 2025, the company had approximately 23.2 million shares of common stock outstanding. Subsequent to the end of the quarter, the company raised gross proceeds of approximately $2.1 million from the sale of common stock on the ATM.

截至 2025 年 3 月 31 日，該公司擁有現金和現金等價物約為 1,930 萬美元。根據我們目前的營運計劃，我們相信我們的現金足以支持我們到 2025 年第三季的營運。截至 2025 年 5 月 8 日，該公司已發行約 2,320 萬股普通股。本季結束後，該公司透過出售 ATM 普通股籌集了約 210 萬美元的總收益。

Now I'd like to focus on some key upcoming milestones. As everybody is well aware, we expect to have top-line cognitive data from our Phase II trial in Alzheimer's disease in the second half of June. As RJ stated, everyone at the company is looking forward to this event. We anticipate an end of Phase II meeting with the FDA in the fourth quarter of 2026 to agree on the design of a Phase III trial. We expect to complete enrollment in the Phase II portion of the INKmune trial this year, as Mark mentioned earlier, with periodic updates on immunologic and therapeutic responses to INKmune as data becomes available.

現在我想重點談談一些即將到來的重要里程碑。眾所周知，我們預計將在 6 月下半月獲得阿茲海默症 II 期試驗的頂級認知數據。正如 RJ 所說，公司裡的每個人都對這次活動充滿期待。我們預計將於 2026 年第四季結束與 FDA 的第二階段會議，以就第三階段試驗的設計達成協議。正如馬克之前提到的，我們預計今年將完成 INKmune 試驗第二階段的招募，並在獲得數據後定期更新 INKmune 的免疫學和治療反應。

We anticipate filing a BLA for CORDStrom in RDEB in the first half of 2026. Finally, we will initiate a Phase II trial of XPro in patients with treatment-resistant depression once NIH funding is made available.

我們預計將於 2026 年上半年在 RDEB 為 CORDStrom 提交 BLA。最後，一旦獲得 NIH 資金支持，我們將啟動針對難治性憂鬱症患者的 XPro II 期試驗。

At this point, Jesse, I'd like to now poll for questions and open it up to Q&A. Jesse?

傑西，現在我想進行民意調查並開放問答環節。傑西？

(Operator Instructions) Gary Nachman, Raymond James.

（操作員指示）Gary Nachman、Raymond James。

This is Denis Reznik on for Gary Nachman. Thank you for taking our questions, and congrats on all the progress. So just assuming a positive readout in June, can you just continue walking us through the exact next steps for the program? You mentioned you can meet with the FDA at the end of this year. But what's a realistic timeline as to when you can initiate the Phase III?

這是加里·納赫曼 (Gary Nachman) 的丹尼斯·雷茲尼克 (Denis Reznik)。感謝您回答我們的問題，並祝賀您取得的所有進展。那麼，僅假設 6 月份的結果為正數，您能否繼續向我們介紹該計劃的具體後續步驟？您提到您可以在今年年底與 FDA 會面。但是何時才能啟動第三階段的實際時間表是怎麼樣的呢？

And then could you provide maybe some metrics as to what's reasonable as to how many sites you can get online and how many patients could be enrolled each month? And then I've got a couple of follow-ups.

然後，您能否提供一些指標來表明有多少網站可以上網以及每月可以招募多少名患者是合理的？然後我還有一些後續問題。

Yeah. Well, this is RJ. So thank you, Gary. But I think expecting us to really design the trial before we have the results and before we talk to the FDA, we would like to defer that opportunity. Our goal, as David mentioned in his milestones, is to as quickly as possible get to the FDA for an end of Phase II meeting.

是的。嗯，這是 RJ。所以謝謝你，加里。但我認為，在我們獲得結果之前並與 FDA 交談之前，我們希望推遲這個機會。正如 David 在他的里程碑中提到的那樣，我們的目標是盡快到達 FDA 並結束第二階段會議。

It is classified as a Type B meeting by the FDA process. So there's a 70-day clock. So hopefully, as we said, it will be completed in the fourth quarter. And then we will move as quickly as possible to getting sites open and those first patients enrolled. We are lucky that we've got a lot of sites ready to go in Europe, and we know there's considerable enthusiasm for this program in the US.

根據 FDA 流程，它被歸類為 B 類會議。因此有一個 70 天的時鐘。因此，正如我們所說，希望它能在第四季度完成。然後，我們將盡快開放站點並招募首批患者。我們很幸運，我們在歐洲已經準備好了許多站點，我們知道美國對這個計畫有相當大的熱情。

So we anticipate being able to get the sites. But I don't want to put a date on it. We don't want to put a size on it. And because we can't put a size on it, we don't want to really predict what the capital needs will be at this point. But suffice it to say that we believe that it will look very much like the Phase II trial, although we expect it will be larger. And there may be a fewer set of biomarkers, but we won't know until we talk to the FDA.

因此我們期望能夠獲得這些網站。但我不想為此設定一個日期。我們不想給它設定一個尺寸。因為我們無法確定其規模，所以我們不想真正預測此時的資本需求是多少。但可以說，我們相信它看起來會非常類似第二階段試驗，儘管我們預期它的規模會更大。生物標誌物的數量可能會少一些，但我們只有在與 FDA 溝通後才會知道。

Totally understand that, and that's helpful color. Just a couple of quick follow-ups. Just because there's been a lot of turnover uncertainty at the FDA recently, can you comment on if the people who will be reviewing your program are all still there, everyone who's previously signed off on the EMAC is all still there? And then with the poster presentation you guys had at AD/PD last month, maybe you can you just talk a little bit more about the receptivity you received at the conference and then overall, the level of interest the company has been receiving recently as we approach June readouts?

完全理解這一點，這是一種很有幫助的顏色。只需進行一些快速的跟進。由於最近 FDA 的人員流動存在很大不確定性，您能否評論一下，負責審查您專案的人員是否都還在，之前簽署 EMAC 協議的所有人是否都還在？然後，根據您上個月在 AD/PD 上進行的海報展示，也許您可以再多談一下您在會議上收到的反饋，以及總體而言，隨著我們接近 6 月份的讀數，公司最近收到的關注程度如何？

Yeah. I'll let CJ answer the AD/PD call. But we have obviously, like any biotech, have been watching what's going on at the FDA quite closely. Our intelligence, which is pretty good, we believe, suggests that when it comes to drug development, the FDA has remained on track. The delays appear to not be developing that everyone feared.

是的。我會讓 CJ 接聽 AD/PD 電話。但顯然，像任何生物技術公司一樣，我們一直在密切關注 FDA 的動態。我們相信，我們的情報相當可靠，顯示 FDA 在藥物研發方面仍保持著正軌。大家擔心的延誤情況似乎並沒有發生。

We can't predict the future, but everything we hear so far is okay. I remind you that the FDA has not yet completely signed off on EMACC. Our goal is -- and CJ can add more color to this. Our goal is to present them EMACC data side by side with the CDR data. And one of the major questions we'll be asking them at the end of Phase II meeting is can we use EMACC as the primary end point for the registration trial.

我們無法預測未來，但目前我們聽到的一切都還好。我提醒您，FDA 尚未完全批准 EMACC。我們的目標是——CJ 可以為此增添更多色彩。我們的目標是向他們並列 EMACC 資料和 CDR 資料。在第二階段會議結束時，我們要問他們的一個主要問題是，我們是否可以將 EMACC 作為註冊試驗的主要終點。

Remember, we've been saying this forever, that we believe that it is a far superior metric of cognitive function in these patients. But it's the FDA's sandbox, and if they say, no, we'll move -- we're not happy, but we will move ahead with CDR. And in fact, the trial that we are currently doing will allow us to perfectly design the study to get the kind of result we need.

請記住，我們一直在說這一點，我們相信這是衡量這些患者認知功能的更優越的指標。但這是 FDA 的沙盒，如果他們說不，我們就會採取行動——我們不高興，但我們將繼續推進 CDR。事實上，我們目前正在進行的試驗將使我們能夠完美地設計研究以獲得我們需要的結果。

CJ, you want to add any color to that and also comment on his AD/PD question?

CJ，你想對此進行補充並對他的 AD/PD 問題進行評論嗎？

Yeah. Yeah, sure. Just to sort of give you -- clarify a little bit about what we've communicated with the FDA, is we've given them the plans and what we intend to do with EMACC. And what they've done is they've responded to that in a way that really says that outlines their guidelines on how you validate a measure, really think about it more like a checklist, which is what we've done in this trial. So they're not going to comment on that until they see the data. That's just a standard response. But we think we've done everything. We've hit all those bullets and those checklists, and I think we're in a good position.

是的。是的，當然。只是為了向您澄清一下我們與 FDA 溝通的內容，我們已經向他們提供了計劃以及我們打算對 EMACC 做什麼。他們所做的就是對此做出回應，概述了關於如何驗證測量的指導方針，實際上將其視為一個清單，這也是我們在這次試驗中所做的。因此，在看到數據之前，他們不會對此發表評論。這只是一個標準答案。但我們認為我們已經做了一切。我們已經完成了所有目標和清單，我認為我們處於有利地位。

AD/PD was pretty interesting. I think there's two things that, I think, really came out of it as it relates to feedback we got from the poster. One is, I think, people are excited for an anti-amyloid or a therapy that's not targeting anti-amyloid. And I would say, even more so, we got a lot of traffic and interest in EMACC. And one of the comments that we -- especially from neuropsychs that work with big companies or worked in the space and measuring cognition are really excited that there is a company that's driving the appropriate measures moving forward.

AD/PD 非常有趣。我認為，有兩件事與我們從海報上得到的回饋有關。一方面，我認為人們對抗澱粉樣蛋白或不針對抗澱粉樣蛋白的療法感到興奮。我想說的是，更重要的是，我們在 EMACC 上獲得了大量的流量和興趣。我們聽到的一條評論——尤其是與大公司合作或在該領域工作並測量認知的神經心理學家的評論——真的很高興有一家公司正在推動適當的措施向前發展。

So there's a lot of excitement and interest around the outcome there.

因此，人們對其結果感到非常興奮和感興趣。

Great. Thanks so much. We're looking forward to the readout.

偉大的。非常感謝。我們期待結果的公佈。

George Farmer, Scotiabank.

加拿大豐業銀行的喬治法默。

Thanks for taking my questions. A couple for me on XPro. So with the trial coming up and the data coming up, how do you think investors will react to a scenario whereby you hit on EMACC, but CDR is a bit equivocal? And then, RJ, you were talking about the potential benefit of XPro in patients who would be ineligible for anti-amyloid therapy, including APOE homozygotes. Do you have a feel for how many APOE homozygotes are in your trial?

感謝您回答我的問題。我在 XPro 上有幾個。那麼，隨著試驗的臨近和數據的出現，您認為投資者會如何應對您擊中 EMACC 但 CDR 有點模棱兩可的情況？然後，RJ，您談到了 XPro 對不適合接受抗澱粉樣蛋白治療的患者（包括 APOE 純合子）的潛在益處。您是否知道試驗中有多少 APOE 純合子？

Yeah. So CJ, why don't you address the specific question about hitting on EMACC and what CDR will look like because it will be more correlative than not?

是的。那麼 CJ，為什麼不回答有關 EMACC 的具體問題以及 CDR 會是什麼樣子，因為它會更具相關性？

So yeah. I'm not sure where you're going with that, RJ. But let me just -- to answer the question about how investors feel, I actually would ask you that question. From a scientific standpoint, what I can tell you is that the EMACC is the tool that's actually capturing cognitive changes that occur in early AD. It's identifying those patients. It's the ability to measure cognitive decline. It's the right tool. The CDR is a more blunt instrument.

是的。我不確定你要說什麼，RJ。但是，為了回答投資者的感受，我實際上會問你這個問題。從科學的角度來看，我可以告訴你的是，EMACC 是一種能夠捕捉早期 AD 認知變化的工具，它可以識別這些患者。它是衡量認知能力下降的能力。這是正確的工具。CDR 是一種更為遲鈍的工具。

I think RJ -- I think what RJ is saying is one of the things that we're seeing is it correlates very well. So we're seeing correlations between EMACC and CDR, which is a good sign, but the CDR is inherently more noisy because it's a blunt tool.

我認為 RJ——我認為 RJ 所說的是我們看到的事情之一，它具有很好的相關性。因此，我們看到了 EMACC 和 CDR 之間的相關性，這是一個好兆頭，但 CDR 本質上更吵雜，因為它是一種鈍器。

So I would say, well, I can't answer how the investors would think, and I'm hopeful that we've talked with enough of you so that you understand how we think in the scientific validity and rationale for using the EMACC. For us, the EMACC is really the primary driver. And we'll understand what -- if the EMACC, for example, is fuzzy, we'll have a clear understanding. The most likely scenario is just it's a power issue due to noise, but we won't know until we see the data.

所以我想說，好吧，我無法回答投資者會怎麼想，我希望我們已經與你們進行了足夠多的交流，以便你們了解我們如何看待使用 EMACC 的科學有效性和基本原理。對我們來說，EMACC 確實是主要的驅動力。我們會明白——例如，如果 EMACC 模糊不清，我們就會有清晰的理解。最有可能的情況是，這只是由於噪音導致的電源問題，但我們只有在看到數據後才會知道。

Yeah. And just to add a little bit more color, we believe that professionals in the field, i.e., the academics and potential biopharma partners, will be able to understand if there is -- they don't both -- aren't both perfect. And as CJ said, it's not going to be that CDR is going in a different direction. It's just going to be a power and a size of study issue, which is easily solved in the Phase III trial.

是的。為了增加一點色彩，我們相信該領域的專業人士，即學者和潛在的生物製藥合作夥伴，將能夠理解是否存在 - 它們並非都是完美的。正如 CJ 所說，CDR 不會走向不同的方向。這只是一個研究的力量和規模問題，在第三階段試驗中很容易解決。

Yeah, so let's talk about the ApoE4 homozygotes. In both the lecanemab and the donanemab trial, they were 15% of the patients. In our trial, it is 9%. They're smaller trials. I don't see 15% to 9% that different. I expect it will be in the 15% range.

是的，那麼我們來談談 ApoE4 純合子。在 lecanemab 和 donanemab 試驗中，他們佔患者的 15%。在我們的試驗中，這一比例為 9%。這些都是規模較小的試驗。我認為 15% 和 9% 之間並沒有什麼區別。我預計它會在 15% 左右。

Interesting, aducanumab did not break out the homozygotes in number. At least, I don't remember it. So I think it's a pretty -- and when you go into the literature and you look at clinical data, that's about where it stands. So that's a pretty -- in Europe, it's a big population. And as you know, in Europe and the UK, they don't do off-label prescribing. So that group is ready and waiting for XPro, in my opinion.

有趣的是，aducanumab 並沒有在數量上打破純合子。至少，我不記得了。所以我認為這很漂亮——當你查閱文獻並查看臨床數據時，你會發現它的情況就是這樣。在歐洲，這是一個相當大的人口群體。眾所周知，在歐洲和英國，他們不會進行非說明書處方。所以，我認為該團隊已經做好準備並等待 XPro。

All right. Great. And then one more for me. The impact of XPro on Phospho Tau 217 looks pretty compelling. Went back and looked at your presentation. How do you think that magnitude compares to other Alzheimer's disease treatment approaches that have been published?

好的。偉大的。然後我再加一個。XPro 對 Phospho Tau 217 的影響看起來非常引人注目。回去看了你的簡報。您認為這種治療方法與已發表的其他阿茲海默症治療方法相比如何？

CJ, I'm going to leave that one to you.

CJ，我將這個留給你。

I think that's tough to answer. I think the only thing that I can say is you don't rarely -- I can't think of another study where you saw changes in the CSF that early quite robustly. I think this is one of those things that holds a lot of promise, but we'll see what it looks like when we have the full data set.

我認為這個問題很難回答。我想我唯一能說的是，你很少——我想不出另一項研究能如此早地看到腦脊髓液的變化。我認為這是一件很有希望的事情，但只有當我們擁有完整的資料集時，我們才能看到它是什麼樣子。

Yeah. We're the only company with data in a drug that treats neuroinflammation. The anti-amyloid drugs do decrease tau. I mean, I think when you -- when neurons stop dying, you get some decrease in tau. But we like pTau217. The clinical teams like pTau217, and I suspect most of the regulatory agencies are going to be very receptive to that biomarker as part of our data package.

是的。我們是唯一一家擁有治療神經發炎藥物數據的公司。抗澱粉樣蛋白藥物確實會降低 tau 蛋白。我的意思是，我認為當神經元停止死亡時，tau 就會減少。但我們喜歡 pTau217。臨床團隊喜歡 pTau217，我猜大多數監管機構都會非常樂意接受該生物標記作為我們資料包的一部分。

Okay. Thanks very much.

好的。非常感謝。

Tom Shrader, BTIG.

BTIG 的湯姆·施拉德 (Tom Shrader)。

Thanks for taking the questions. You said something that I hadn't really thought of, and I'm wondering if it's correct -- if I'm hearing. But are ApoE4 patients inherently inflammatory? And then the other question, I kind of want to reask George's question with a little bit of a -- how much does CDR have to decline? How big does the reduction in decline of CDR have to be to hit in a trial of 300 patients that's only run for six months? Is it reasonable? Or is it a pipe dream? Do you have a sense of what the reduction in decline would have to be for a trial of this size to hit? Thanks.

感謝您回答這些問題。你說了一些我之前沒有想到的事情，我不知道這是否正確——如果我沒聽錯的話。但是 ApoE4 患者天生就容易發炎嗎？然後是另一個問題，我想重新問喬治的問題——CDR 必須下降多少？在僅進行六個月的 300 名患者試驗中，CDR 下降幅度需要減少多少才能達到預期？合理嗎？還是這只是一場白日夢？您是否知道，要達到如此規模的試驗效果，下降幅度需要減少多少？謝謝。

Yeah, I can take that. I can take the first one or the second one. So Tom, what's interesting is when we powered the study, which was powered on CDR, by the way, the assumptions that we had in terms of our expectation for decline over six months, and the effect size of that decline was somewhat conservative based on the ADNI group that was used to power the study. What's interesting is a few years later when lecanemab and donanemab came out, what we saw was the assumptions that we use were exactly what happened.

是的，我可以接受。我可以選第一個或第二個。湯姆，有趣的是，當我們為這項研究提供支持時，順便說一下，這項研究是基於 CDR 進行的，我們對六個月內下降的預期以及下降的影響大小的假設是基於用於支持這項研究的 ADNI 組得出的，這些假設有些保守。有趣的是，幾年後，當 lecanemab 和 donanemab 問世時，我們看到的正是我們所使用的假設所發生的事情。

So within a six-month period, both aducanumab or lecanemab and donanemab were statistically significant at six months. And their decline was about the exact same number that we used to decline for six months. And I think that's important. And the effect size of that decline was almost exactly what we used to calculate our power. So I think that gives us a lot of confidence that our assumptions were right on. I think the other thing that I want to point out is this is for a cohort of patients that weren't enriched for inflammation. And of course, we expect that we're going to get a little bit faster decline with inflammation. So I think that gives us even a little more confidence.

因此，在六個月的時間內，aducanumab 或 lecanemab 和 donanemab 均具有統計意義。他們的下降幅度與我們過去六個月的下降幅度大致相同。我認為這很重要。而這個下降的效果量幾乎與我們用來計算力量的大小完全相同。所以我認為這給了我們很大的信心，相信我們的假設是正確的。我想指出的另一件事是，這是針對一群沒有發炎的患者。當然，我們預期發炎會導致病情惡化得更快一些。所以我認為這給了我們更多的信心。

Can I follow-up --

我可以跟進一下嗎——

Yeah, and (inaudible) would be considered an inflammation gene. It is considered that with your ApoE4/4, you are hot, so to speak. And as CJ just said, if you look at ApoE4 either heterozygotes or homozygotes, they actually get -- their age of onset is earlier, with the heteros being later than the homozygotes. Their progression of the disease, i.e., going from MCI to early mild AD is faster. And actually, when you look at the APOE homozygotes, ApoE4 homozygotes, they actually have a higher mortality rate.

是的，並且（聽不清楚）會被視為一種發炎基因。可以認為，有了 ApoE4/4，您就很熱，可以這麼說。正如 CJ 剛才所說，如果你觀察 ApoE4 無論是雜合子還是純合子，它們的發病年齡實際上更早，雜合子比純合子晚。他們的病情進展，從 MCI 到早期輕度 AD 的進展速度更快。實際上，當你觀察 APOE 純合子、ApoE4 純合子時，你會發現他們的死亡率實際上更高。

They have about a five- to seven-year worse survival compared to the other groups. So ApoE4 is a bad gene to have. And any of you that have done 23andMe and they ask that question, do you want to know what your Alzheimer's risk is? The main thing they're going to tell you was whether or not you were ApoE4 positive. And I don't know how many said yes, but I never wanted to know, quite frankly.

與其他群體相比，他們的生存期大約要差五到七年。所以 ApoE4 是一種有害基因。你們當中有誰做過 23andMe 測試並且他們問過這個問題，你想知道你患阿茲海默症的風險是多少嗎？他們要告訴你的主要事情是你是否是 ApoE4 陽性。我不知道有多少人回答了“是”，但坦白說，我從來都不想知道。

If I can follow up with CJ, how much of the other two trials hitting -- they're bigger trials, right? They're something like -- I don't know, there's a few fold more patients. Is that the reason they separated in six months? Or do you still think you're powered correctly to see it if you have a similar type effect?

如果我可以跟進 CJ，那麼另外兩個試驗的效果如何——它們是更大的試驗，對嗎？他們就像──我不知道，病人數量多了幾倍。這就是他們六個月內分手的原因嗎？或者，如果您有類似類型的效果，您是否仍然認為您有正確的能力來看到它？

Well, I think -- so I mean, there's some variables we don't know. I think we're close. I don't think we're going to be off too much. So it's hard to answer that question, but I would say that the data that we see, despite the fact that they've got more patients, we're looking at standard deviation. The standard deviation was around one for those trials, which is very similar to what we used in our projections. And the decline was quite similar. So I think we're going to be okay. I think we're going to be close.

嗯，我認為——我的意思是，有一些變數我們不知道。我認為我們已經很接近了。我認為我們不會偏離太多。所以這個問題很難回答，但我想說的是，我們看到的數據，儘管他們有更多的病人，但我們正在看標準差。這些試驗的標準差約為 1，與我們在預測中使用的非常相似。且下降趨勢也相當相似。所以我認為我們會沒事的。我想我們很快就會接近目標了。

And Tom, remember, we bragged about the quality control that we've had in this trial, the procedure of the type of patient we enrolled. I think that is really an unheralded advantage. When you're out enrolling 1,600 patients, and I think it was like in 42 countries or something as those big trials. The quality control -- you just get a messier patient population. It's not because people aren't trying.

湯姆，記住，我們吹噓過我們在這次試驗中的品質控制，以及我們招募的患者類型的程序。我認為這確實是一個未被預料到的優勢。當你招募 1,600 名患者時，我認為這就像在 42 個國家或地區進行的大型試驗一樣。品質控制－你只會得到更混亂的患者群體。這並不是因為人們沒有努力。

It's just it's a big animal pain. And I can tell you, we've spent a huge amount of time and resources on our quality control, and our trial was much smaller. It was, what, 208 patients in eight or nine countries. So the quality will be reflected in the output, I believe.

這確實給動物帶來了巨大的痛苦。我可以告訴你，我們在品質控制上花費了大量的時間和資源，而我們的試驗規模卻小得多。總共有來自八、九個國家的 208 名患者。所以我相信品質將反映在產出上。

All right. Great. Thanks for all the color.

好的。偉大的。感謝所有的色彩。

James Molloy, Alliance Global Partners.

聯盟全球合作夥伴的詹姆斯‧莫洛伊 (James Molloy)。

This is Laura Suriel on for Jim Molloy. Thank you for taking the questions. So for CORDStrom, are you still on track to initiate the 12-month open-label trial mid this year? And I also see that US patients are expected to be enrolled later on. So what's the enrollment aim here? And how many sites do you expect to have open in the US?

我是 Laura Suriel，為 Jim Molloy 主持節目。感謝您回答這些問題。那麼對於 CORDStrom 來說，您是否仍計劃在今年年中啟動為期 12 個月的開放標籤試驗？我還看到預計稍後會有美國患者入組。那這裡的招生目標是什麼呢？您預計在美國開設多少個站點？

Mark?

標記？

Yeah. Great. Thanks very much for that question. What we're doing at the moment is following the guidance from the FDA about how to make the UK-manufactured cords usable in the US, and we're starting the manufacturing program later this week, actually, of a new batch of products using US-approved cord donors. So that allows us, once we have those data, to draft the IND.

是的。偉大的。非常感謝您提出這個問題。我們目前正在做的是遵循 FDA 的指導，使英國製造的臍帶能夠在美國使用，實際上，我們將在本週晚些時候啟動使用美國批准的臍帶捐贈者生產新一批產品的生產計劃。這樣，一旦我們獲得這些數據，我們就可以起草 IND。

We're expecting to submit an IND later this year. We've been delayed by various factors, including the funding situation. But we're still on track to -- or still planning an IND in the US for CORDStrom. But the BLA is not dependent upon the US IND or, indeed, the open label in the UK. We're going to submit for the BLA with the data we already have from the double-blind, placebo-controlled crossover trial. And then we will run a follow-on trial in the US, opening next year in parallel with trials in the UK in which we will further explore the dosing of the drug and the periodicity of dosing. Does that answer your question?

我們預計將於今年稍後提交 IND。我們因各種因素而被推遲，包括資金狀況。但我們仍在按計劃進行 — — 或仍在計劃在美國為 CORDStrom 進行 IND。但 BLA 並不依賴美國 IND 或英國的開放標籤。我們將使用從雙盲、安慰劑對照交叉試驗中獲得的現有數據來提交 BLA。然後，我們將在美國進行後續試驗，該試驗將於明年與英國的試驗同時進行，我們將進一步探索藥物的劑量和給藥週期。這回答了你的問題嗎？

Yeah, it does. Thank you. And just as a follow-up, with the BLA filing coming up, how would you describe like the overall regulatory space for RDEB treatment? Like Abeona, for instance, just got approval for their gene therapy for RDEB. So do you consider this a positive for where you're at with CORDStrom?

是的，確實如此。謝謝。作為後續問題，隨著 BLA 申請的臨近，您如何描述 RDEB 治療的整體監管空間？例如，Abeona 剛剛獲得 RDEB 基因療法的批准。那麼，您是否認為這對您在 CORDStrom 的處境來說是一件好事？

So there are two things about that. That's a very good question. So the synthetic skin gene-modified product, which has just been licensed, it's a great product. The challenge is that it's applicable to open wounds on the skin, and that's its only indication. And it requires a surgical intervention to apply it.

關於這一點，有兩件事。這是一個非常好的問題。因此，剛獲得許可的合成皮膚基因改造產品是一款很棒的產品。挑戰在於它僅適用於皮膚上的開放性傷口，而這是它的唯一適應症。並且需要透過手術幹預才能應用它。

One of the known side effects is itch. And obviously, one of our principal clinical outcomes from the first trial has been reduction in itch. So even in the presence of the gene-modified product, we have a window to treat the same patients with CORDStrom.

已知的副作用之一是搔癢。顯然，第一次試驗的主要臨床結果之一就是搔癢的減少。因此，即使有基因改造產品，我們仍然有機會用 CORDStrom 治療相同的患者。

But more importantly, CORDStrom is a systemic therapy. And as I said in the presentation just a few minutes ago, RDEB and indeed, all EB, is actually a systemic disease. And these kids, and indeed when they get through to adulthood, they suffer the condition throughout their esophageal tract and more widespread than just the dermis. And so we see a role for CORDStrom in treating those patients with systemic disease, as well as skin lesions. Indeed, a trial of a similar MSC product that's not going through to license in South Korea showed improvement in skin scores after treatment as well when the children were followed up long enough.

但更重要的是，CORDStrom 是一種系統性療法。正如我幾分鐘前在演講中所說的那樣，RDEB 以及所有 EB 實際上是一種全身性疾病。這些孩子，甚至當他們成年時，他們的整個食道都會受到這種疾病的困擾，而且這種疾病不僅限於真皮。因此，我們看到了 CORDStrom 在治療全身性疾病以及皮膚病變的患者方面發揮的作用。事實上，在韓國尚未獲得許可的類似 MSC 產品的試驗表明，如果對兒童進行足夠長時間的跟踪，治療後皮膚評分也有所改善。

So we do see a really good opportunity for CORDStrom even in the presence of the current licensed alternatives.

因此，即使存在當前許可的替代方案，我們確實看到了 CORDStrom 的一個非常好的機會。

Understood. Thank you for taking the questions.

明白了。感謝您回答這些問題。

And now that will conclude the Q&A portion. I will turn the program back over to Dr. Tesi for any additional or closing remarks.

問答部分現在就結束。我將把節目交還給 Tesi 博士，請他發表任何補充或結束語。

Yes. Thank you all for attending today's call. And I just make a moment of closing comments because we are excited. We are approaching the most significant milestone in the company's history. When we started INmune Bio and got XPro in 2017 and became a company focused on Alzheimer's disease, no one viewed it as an immunologic disease or few did.

是的。感謝大家參加今天的電話會議。由於我們都很興奮，所以我想做一點總結發言。我們即將迎來公司史上最重要的里程碑。當我們在 2017 年創立 INmune Bio 並收購 XPro 並成為一家專注於阿茲海默症的公司時，沒有人將其視為一種免疫性疾病，或者很少有人這麼認為。

At that time, the focus was exclusively on amyloid and tau. Since then, with two anti-amyloid drugs on the market, we have seen a sea change in the way Alzheimer's disease is viewed. Alzheimer's is considered by many a CNS disease driven by immune dysfunction. Neuroinflammation is no longer viewed as a side effect of Alzheimer's disease pathology, but a significant driver of that pathology. INmune Bio has positioned itself as a leader in targeting the immune dysfunction that drives destructive neuroinflammation.

當時，人們的關注點完全集中在澱粉樣蛋白和 tau 蛋白上。從那時起，隨著兩種抗澱粉樣蛋白藥物的上市，我們看到了人們對阿茲海默症的看法發生了巨大的變化。許多人認為阿茲海默症是一種由免疫功能障礙引起的中樞神經系統疾病。神經發炎不再被視為阿茲海默症病理的副作用，而是此病理的重要驅動因素。INmune Bio 已將自己定位為針對導致破壞性神經發炎的免疫功能障礙的領導者。

We are very careful and precise with our terminology here. Stopping neuroinflammation by immunosuppression, glial suppression is very different than stopping destructive neuroinflammation by repolarizing glial cells to support the CNS cellular unit and improving remodeling and repair. The goal of effective therapy for the Alzheimer's disease is to reestablish normal glial homeostasis. The idea that the brains of the elderly with dementia can undergo remodeling and repair is novel. We believe data from the MINDFuL trial will change the direction of scientific research and discovery in the neurology and CNS drug development arena.

我們在這裡使用術語時非常謹慎和精確。透過免疫抑制、神經膠質抑制來阻止神經發炎與透過重新極化神經膠質細胞來支持中樞神經系統細胞單元並改善重塑和修復來阻止破壞性神經發炎有很大不同。阿茲海默症有效治療的目標是重建正常的神經膠質穩態。老年癡呆症患者的大腦可以進行重塑和修復的想法是新穎的。我們相信 MINDFuL 試驗的數據將改變神經病學和中樞神經系統藥物開發領域的科學研究和發現的方向。

I've said before that this is the dawn of the golden age of CNS development. We believe it is, and we look forward to leading the charge. So with that, I thank you, and I'm sure we will be talking to you all soon when the data are released. So thank you very much.

我以前說過，這是中樞神經發展黃金時代的曙光。我們相信這一點，並且我們期待引領這項變革。因此，我感謝你們，我相信當數據發佈時我們很快就會與你們交談。非常感謝。

Thank you. Ladies and gentlemen, this does conclude today's program. Thank you for your participation. You may disconnect at any time.

謝謝。女士們、先生們，今天的節目到此結束。感謝您的參與。您可以隨時斷開連線。