Omeros Corp (OMER) 2024 Q2 法說會逐字稿

Good afternoon, and welcome to today's earnings call for Omeros Corporation. (Operator Instructions) Please be advised that this call is being recorded at the company's request, and a replay will be available on the company's website for one week from today.

下午好，歡迎參加今天的 Omeros Corporation 財報電話會議。（操作員說明）請注意，本次通話是應公司要求進行錄音的，從今天起一週內將在公司網站上提供重播。

I'll now turn the call over to Jennifer Williams, Investor Relations for Omeros.

我現在將把電話轉給 Omeros 投資者關係部的 Jennifer Williams。

Good afternoon, and thank you for joining the call today. I'd like to remind you that some of the statements that will be made on the call today will be forward-looking. These statements are based on management's beliefs and expectations as of today only and are subject to change.

下午好，感謝您今天加入通話。我想提醒大家，今天的電話會議上發表的一些聲明將具有前瞻性。這些陳述僅基於管理層截至今天的信念和期望，並且可能會發生變化。

All forward-looking statements involve risks and uncertainties that could cause the company's actual results to differ materially. Please refer to the special note regarding forward-looking statements in the company's quarterly report on Form 10-Q, which was filed today with the SEC, and the Risk Factors section of the company's most recent annual report on Form 10-K for a discussion of these risks and uncertainties.

所有前瞻性陳述均涉及風險和不確定性，可能導致本公司的實際結果出現重大差異。請參閱今天向 SEC 提交的公司 10-Q 表格季度報告中有關前瞻性陳述的特別說明，以及公司最新 10-K 表格年度報告中的風險因素部分進行討論這些風險和不確定性。

Now I would like to turn the call over to Dr. Greg Demopulos, Chairman and CEO of Omeros.

現在我想將電話轉給 Omeros 董事長兼執行長 Greg Demopulos 博士。

Thank you, Jennifer. And good afternoon, everyone. I'm joined on today's call by David Borges, our Chief Accounting Officer; Nadia Dac, our Chief Commercial Officer; Andreas Grauer, our Chief Medical Officer; Cathy Melfi, our Chief Regulatory Officer; and Steve Whitaker, our Clinical Vice President.

謝謝你，詹妮弗。大家下午好。我們的首席會計官 David Borges 也參加了今天的電話會議。 Nadia Dac，我們的首席商務官； Andreas Grauer，我們的首席醫療官； Cathy Melfi，我們的首席監理官；以及我們的臨床副總裁 Steve Whitaker。

I'll start today with an overview and discussion of our 2024 second-quarter financial results, followed by an update on our ongoing development program. David will then provide a more detailed financial summary before we open the call to questions.

今天我將首先概述和討論我們 2024 年第二季的財務業績，然後介紹我們正在進行的開發計劃的最新情況。然後，在我們開始提問之前，大衛將提供更詳細的財務摘要。

Now let's look at our financial results for the second quarter. Our net loss for the second quarter of 2024 was $56 million or $0.97 per share compared to a net loss of $37.2 million or $0.63 per share in the first quarter of 2024.

現在讓我們來看看第二季的財務表現。我們 2024 年第二季的淨虧損為 5,600 萬美元，即每股 0.97 美元，而 2024 年第一季的淨虧損為 3,720 萬美元，即每股 0.63 美元。

In the second quarter, we incurred $17.6 million of R&D expense related to the manufacture of narsoplimab drug substance lots that commenced in October 2023, $21.2 million in discounted term loan related debt repurchase, and $1.9 million of term loan related transaction costs. These significant cash outlays representing a total of $40.7 million are not expected to be repeated in the foreseeable future.

第二季度，我們產生了 1,760 萬美元與 2023 年 10 月開始生產 narsoplimab 原料藥批次相關的研發費用、2,120 萬美元的貼現定期貸款相關債務回購費用以及 190 萬美元的定期貸款相關交易成本。這些總計 4,070 萬美元的巨額現金支出預計在可預見的未來不會重複。

As of June 30, 2024, we had $158.9 million of cash and investments on hand. In addition, as explained in earlier calls, our recent sale of OMIDRIA royalties to DRI Healthcare carries with it two sales contingent milestones payable by DRI to Omeros, each up to $27.5 million with payment dates in January 2026 and January 2028.

截至 2024 年 6 月 30 日，我們手頭上有 1.589 億美元的現金和投資。此外，正如先前電話會議中所解釋的，我們最近向DRI Healthcare 出售OMIDRIA 特許權使用費附帶了DRI 應付給Omeros 的兩個銷售或有里程碑，每個里程碑高達2750 萬美元，付款日期為2026 年1月和2028 年1 月。

In the second quarter, we substantially strengthened our balance sheet through our debt repurchase and exchange transaction. At March 31, 2024, we had $216 million in notional value outstanding on our 2026 notes, all maturing in February 2026.

第二季度，我們透過債務回購和換匯交易大幅強化了資產負債表。截至 2024 年 3 月 31 日，我們 2026 年票據的名目價值為 2.16 億美元，全部於 2026 年 2 月到期。

In June, we entered into an agreement with Athyrium Capital Management and Highbridge Capital Management, under which we repurchased and retired 2026 notes held by Athyrium and by Highbridge, representing a collective notional value of $118.1 million for $21.2 million in cash and $67.1 million in a new secured term loan with maturity extended out to June of 2028.

6 月，我們與Athyrium Capital Management 和Highbridge Capital Management 簽訂了一項協議，根據該協議，我們回購並註銷了Athyrium 和Highbridge 持有的2026 年票據，總計名義價值為1.181 億美元，其中包括2,120 萬美元現金和6,710 萬美元債券。

The total of $88.3 million in cash paid and new term loan represents a discount of approximately 25% or $29.8 million off of par. This transaction brings our remaining 2026 notes down by 55% from $216 million to a much more manageable $98 million with the option at Omeros' discretion to reduce that amount by another $15 million in notional value with additional lenders.

支付的現金和新定期貸款總額為 8,830 萬美元，折價約 25%，即比面值低 2,980 萬美元。這項交易使我們剩餘的 2026 年票據減少了 55%，從 2.16 億美元減少到更易於管理的 9,800 萬美元，而 Omeros 可以選擇與其他貸款人一起將名義價值再減少 1,500 萬美元。

We view the outcome of the transaction as highly favorable to Omeros for the following reasons. Through both the restructuring and repurchase of $118.1 million of our outstanding 2026 notes, we extinguished nearly $30 million of debt through financial engineering alone. The transaction was completed at approximately 75% of par. So rather than being priced at the typical 10% to 15% premium above the prevailing market, our transaction was priced slightly below the market. We've further de-risked the balance sheet with the majority of our debt now extinguished or having its maturity profile extended from February 2026 to June 2028.

我們認為交易結果對 Omeros 非常有利，原因如下。透過重組和回購 1.181 億美元的 2026 年未償還票據，我們僅透過金融工程就消除了近 3,000 萬美元的債務。該交易以面額約 75% 的價格完成。因此，我們的交易定價略低於市場，而不是比當前市場高出 10% 至 15% 的溢價。我們進一步降低了資產負債表的風險，我們的大部分債務現已消除，或將其到期期限從 2026 年 2 月延長至 2028 年 6 月。

Again, unusual for this type of transaction, the deal is non-dilutive for Omeros with no common stock or warrants included. The interest rate on the new term loan is highly competitive, resulting in a lower cash outflow than our prior principal and interest payments on the retired 2026 notes. Omeros also retains the ability to spend up to $25 million to repurchase additional outstanding 2026 notes.

同樣，對於此類交易來說不同尋常的是，該交易對 Omeros 來說是非稀釋性的，不包含普通股或認股權證。新定期貸款的利率極具競爭力，導致現金流出低於我們先前已退休 2026 年票據的本金和利息支付。Omeros 也保留花費最多 2,500 萬美元回購額外未償還 2026 年票據的能力。

In addition, as I mentioned a bit earlier, Omeros has the option to allow other lenders to join the transaction to a total of an additional $15 million of notional value, provided that they are given terms no better than those received by Athyrium and Highbridge.

此外，正如我之前提到的，Omeros 可以選擇允許其他貸方加入交易，名義價值總計為 1500 萬美元，前提是它們的條款不優於 Athyrium 和 Highbridge 收到的條款。

The term loan agreement also includes an additional commitment by the lenders to fund a $25 million delayed draw facility at our request prior to July 3, 2025, contingent on FDA approval of narsoplimab in TA-TMA. This additional commitment provides Omeros with a ready source of capital to fuel narsoplimab's early commercialization while preserving our cash to fund program development, to reduce the remaining balance of our outstanding 2026 notes, and/or to reduce the outstanding balance on our term loan.

定期貸款協議還包括貸款人的額外承諾，根據我們的要求，在 2025 年 7 月 3 日之前為 2500 萬美元的延遲提款融資提供資金，具體取決於 FDA 批准 TA-TMA 中的 narsoplimab。這項額外承諾為Omeros 提供了現成的資金來源，以推動narsoplimab 的早期商業化，同時保留我們的現金來資助項目開發，減少我們未償付的2026 年票據的剩餘餘額，和/或減少我們定期貸款的未償餘額。

As part of this transaction, we also retain all future value of the capped calls associated with all the 2026 notes, including those retired in the debt repurchase and exchange. Collectively, these capped calls represent substantial potential value.

作為本次交易的一部分，我們也保留與所有 2026 年票據相關的上限贖回權的所有未來價值，包括在債務回購和交換中退役的票據。總的來說，這些上限看漲期權代表著巨大的潛在價值。

In summary, our recent debt repurchase and exchange non-dilutively reduces by more than half the outstanding principal amount of our 2026 maturing date at a substantial discount to par value, replacing a portion of that outstanding debt with a term loan maturing out in June of 2028, and also provides funding flexibility to manage the remaining lesser balance of our 2026 notes, to fund the early commercialization of narsoplimab and to continue advancing through upcoming shareholder value driving catalysts within our development programs.

總而言之，我們最近的債務回購和交換以非稀釋性方式將2026 年到期日的未償本金減少了一半以上，其票面價值大幅折扣，並用2026 年6 月到期的定期貸款取代了部分未償債務。

Building on our earlier retirement of $9.1 million notional value of our 2026 notes and our recent open market purchases and return to our treasury of 8% of Omeros' outstanding common stock, this transaction with Athyrium and Highbridge represents another substantial step in managing our capital structure to remove financial overhang.

在此基礎上，我們提前收回了名義價值910 萬美元的2026 年票據，以及最近在公開市場購買了8% 的Omeros 已發行普通股並將其返還至我們的庫房，此次與Athyrium 和Highbridge 的交易標誌著我們在管理資本結構方面又邁出了重要一步以消除財務積壓。

With that, let's move on to an update on our development programs. I'll begin with narsoplimab, our MASP-2 inhibitor, targeting the lectin pathway of complements.

接下來，讓我們繼續更新我們的開發計劃。我將從 narsoplimab 開始，它是我們的 MASP-2 抑制劑，針對補體的凝集素途徑。

As I described in our last earnings call and at our annual shareholder meeting in June, we continue to engage with FDA regarding the proposed resubmission of our biologics license application or BLA for narsoplimab in hematopoietic stem cell transplant associated thrombotic microangiopathy or TA-TMA. Another upcoming meeting has been scheduled with FDA. In the meantime, we're preparing the BLA for resubmission.

正如我在上次財報電話會議和6 月的年度股東大會上所述，我們將繼續與FDA 就擬重新提交narsoplimab 治療造血幹細胞移植相關血栓性微血管病或TA-TMA 的生物製劑許可申請或BLA 進行接觸。已安排與 FDA 舉行另一次會議。同時，我們正在準備 BLA 以供重新提交。

We're further encouraged by FDA's recent establishment of the Rare Disease Innovation Hub, which will serve as a single point of connection and engagement within the FDA to promote and support the development of therapeutics for rare diseases.

FDA 最近建立的罕見疾病創新中心進一步鼓舞了我們，該中心將作為 FDA 內部的單一聯繫和參與點，以促進和支持罕見疾病療法的開發。

The hub is designed to focus especially on rare diseases for which the natural history is variable and not fully understood, recognizing that development of therapies for these conditions is often particularly challenging.

該中心旨在特別關注自然史可變且尚未完全了解的罕見疾病，並認識到針對這些疾病的治療方法的開發通常特別具有挑戰性。

Regarding our plans for resubmission and its anticipated timing, we will provide a further update when more definitive information from our FDA interactions becomes available.

關於我們的重新提交計劃及其預期時間，當我們與 FDA 的互動獲得更明確的資訊時，我們將提供進一步的更新。

Also, in addition to the publications already available, two more manuscripts authored by international transplant experts are in preparation. One directed to the results of a survival comparison between our pivotal TA-TMA trial and a rigorous external control. And the second, detailing the survival data in our expanded access program.

此外，除了已有的出版物外，由國際移植專家撰寫的另外兩份手稿正在準備中。其中一項針對我們的關鍵 TA-TMA 試驗與嚴格的外部對照之間的生存比較結果。第二，詳細說明我們擴展存取計劃中的生存數據。

Physicians continue to increasingly request narsoplimab under expanded access for their patients with TA-TMA. Given that there is no approved treatment for this life-threatening condition, we continue to do what we can to help these patients.

醫生們繼續越來越多地要求 narsoplimab 為 TA-TMA 患者提供更多的使用機會。鑑於這種危及生命的疾病尚無批准的治療方法，我們將繼續盡我們所能幫助這些患者。

In parallel with our efforts to obtain FDA approval for narsoplimab, we continue rapidly advancing the clinical development program for our MASP-3 inhibitor, targeting the alternative pathway of complement.

在我們努力獲得 FDA 批准 narsoplimab 的同時，我們繼續快速推進 MASP-3 抑制劑的臨床開發計劃，以補體替代途徑為目標。

OMS906, our lead MASP-3 antibody, now has a recently adopted non-proprietary name, zaltenibart. We have multiple Phase 2 studies of zaltenibart ongoing in two rare disease indications, paroxysmal nocturnal hemoglobinuria, or PNH, a life-threatening hematologic disorder, and complement 3 glomerulopathy, or C3G, a debilitating and potentially life-threatening kidney disease.

OMS906 是我們的領先 MASP-3 抗體，最近採用了一個非專有名稱 zaltenibart。我們正在進行針對兩種罕見疾病適應症的多項扎替尼巴特2 期研究：陣發性睡眠性血紅蛋白尿症（PNH）（一種危及生命的血液系統疾病）和補體3 腎小球病（ C3G）（一種使人衰弱且可能危及生命的腎臟疾病）。

We'll look first at our ongoing Phase 2 PNH clinical trial. The annual European Hematology Association Congress in June, Dr. Morag Griffin, an internationally recognized PNH expert from St. James University Hospital in the UK, presented the results of the adjunctive treatment stage of our switchover study in PNH patients with a suboptimal response to treatment with the C5 inhibitor, ravulizumab.

我們首先來看看正在進行的 PNH 2 期臨床試驗。在六月的歐洲血液學協會年度大會上，來自英國聖詹姆斯大學醫院的國際知名 PNH 專家 Morag Griffin 博士介紹了我們針對治療反應不佳的 PNH 患者進行的輔助治療階段的轉換研究結果與C5 抑製劑ravulizumab 一起使用。

The study evaluated two zaltenibart dose levels. As previously discussed, patients in this study began adjunctive zaltenibart treatment while receiving ravulizumab, and then those patients adequately responding to combination treatment were switched to zaltenibart monotherapy.

該研究評估了兩種扎替尼巴特劑量水平。如前所述，本研究中的患者在接受拉維珠單抗的同時開始輔助扎替尼巴特治療，然後那些對聯合治療有充分反應的患者轉為扎替尼巴特單藥治療。

During the adjunctive therapy period, the statistically significant mean hemoglobin improvement from baseline was 3.27 grams per deciliter, and 10 of 12 patients advanced to monotherapy. Absolute reticulocyte count also demonstrated statistically significant improvement, and consistent with our clinical experience with zaltenibart, the drug was safe and well tolerated.

在輔助治療期間，平均血紅素相對於基線的改善具有統計意義，為 3.27 克/分升，12 名患者中有 10 名進展為單一治療。絕對網織紅血球計數也顯示出統計學上顯著的改善，並且與我們使用紮替尼巴特的臨床經驗一致，該藥物是安全且耐受性良好的。

The last patient visit in this switchover study is scheduled for October, and patients completing the study are entering our long-term extension study. An abstract providing results of the zaltenibart monotherapy stage has been submitted to the American Society of Hematology for presentation at their annual meeting in December.

這項轉換研究的最後一次患者訪視定於 10 月進行，完成研究的患者將進入我們的長期擴展研究。一份提供扎替尼巴特單藥治療階段結果的摘要已提交給美國血液學會，以便在 12 月的年會上發表。

The efficacy and safety profiles of zaltenibart as monotherapy remain strong, including demonstration of sustained and clinically meaningful improvements in hemoglobin levels and absolute reticulocyte counts, as well as prevention of extravascular and intravascular hemolysis. We look forward to sharing these data with you later this year.

扎替尼巴特作為單一療法的療效和安全性仍然強勁，包括證明血紅蛋白水平和絕對網織紅血球計數持續且具有臨床意義的改善，以及預防血管外和血管內溶血。我們期待在今年稍後與您分享這些數據。

Our second ongoing Phase 2 PNH study continues to progress well. This one is in patients who are naive to complement inhibitor treatment, meaning that they have not received previous complement therapy.

我們正在進行的第二項 PNH 2 期研究繼續進展順利。這種情況是針對未接受過補體抑制劑治療的患者，這意味著他們之前沒有接受過補體治療。

After presentation of the early results of subcutaneous zaltenibart treatment at the 2023 American Society of Hematology meeting last December, we amended the study protocol to identify the plasma concentrations and the level of MASP-3 inhibition required to inhibit breakthrough hemolysis.

去年 12 月在 2023 年美國血液學會會議上展示皮下紮替尼巴特治療的早期結果後，我們修改了研究方案，以確定抑制突破性溶血所需的血漿濃度和 MASP-3 抑制水平。

These data, together with data derived from our switchover PNH study and from our Phase 1 studies in healthy subjects, provide the basis for selecting the zaltenibart dose for our upcoming Phase 3 clinical trials. We now have the needed data to finalize our Phase 3 dose selection and analyses will be completed soon.

這些數據，加上我們的 PNH 轉換研究和健康受試者 1 期研究的數據，為我們即將進行的 3 期臨床試驗選擇扎替尼巴特劑量提供了基礎。我們現在擁有完成第三階段劑量選擇所需的數據，分析很快就會完成。

Looking ahead to our PNH Phase 3 program, we remain on track to initiate both our naive patient and switchover Phase 3 trials later this year. All required activities have either been completed or are progressing as planned. We have completed three international and US medical advisory boards to gather input on the current and future needs of the PNH community and to understand how best to position zaltenibart for success against competitors.

展望我們的 PNH 3 期項目，我們仍有望在今年稍後啟動首次患者試驗和轉換 3 期試驗。所有必需的活動均已完成或正在按計劃進行。我們已經成立了三個國際和美國醫療諮詢委員會，以收集有關 PNH 社區當前和未來需求的意見，並了解如何最好地定位扎特尼巴特，從而在競爭中取得成功。

Physician advisors and focus group patients have uniformly responded positively to the unique properties of both zaltenibart and its MASP-3 target as well as to our clinical data and they expect that zaltenibart, when approved, will see significant uptake.

醫生顧問和焦點小組患者對扎替尼巴特及其 MASP-3 標靶的獨特特性以及我們的臨床數據一致做出積極反應，他們預計扎替尼巴特一旦獲得批准，將會得到顯著採用。

These PNH experts are working closely with us to ensure that our study protocols are designed to demonstrate zaltenibart's potential and to help us prepare for efficient study execution.

這些 PNH 專家正在與我們密切合作，以確保我們的研究方案旨在展示扎替尼巴特的潛力，並幫助我們為高效的研究執行做好準備。

Our clinical operations team has already begun site identification and has been developing strong relationships with investigators, which should serve to speed enrollment. We anticipate having broad and geographically diverse site participation that will support both post-approval adoption and utilization.

我們的臨床操作團隊已經開始進行地點識別，並與研究人員建立了牢固的關係，這將有助於加快入組速度。我們預計會有廣泛且地域多樣的站點參與，這將支持批准後的採用和利用。

All external vendors have already been identified and awarded. And on the manufacturing front, all zaltenibart needed for our Phase 3 program has already been manufactured and earmarked for the clinical trials.

所有外部供應商均已確定並授予。在製造業方面，我們第三階段計劃所需的所有紮替尼巴特都已製造完畢並指定用於臨床試驗。

With respect to our zaltenibart regulatory efforts, we have prepared our briefing materials to confirm FDA and European regulatory alignment with our clinical plan and Phase 3 trial designs.

關於我們的 zaltenibart 監管工作，我們準備了簡報資料，以確認 FDA 和歐洲監管與我們的臨床計劃和 3 期試驗設計的一致性。

Following our meeting with FDA earlier this year, we incorporated all FDA feedback and we look forward to our upcoming pre-Phase 3 meetings with both US and European regulators which have already been scheduled or requested.

在今年稍早與 FDA 舉行會議之後，我們納入了 FDA 的所有回饋，我們期待即將與美國和歐洲監管機構舉行的第三階段前會議，這些會議已經安排或要求進行。

Turning now to zaltenibart in C3 glomerulopathy or C3G, our Phase 2 study is enrolling. We look forward to sharing data from this study later this year or early next. A Phase 3 program in C3G is planned to initiate in the first quarter of 2025.

現在轉向扎替尼巴特治療 C3 腎小球病或 C3G，我們的 2 期研究正在招募。我們期待在今年稍後或明年初分享這項研究的數據。C3G 的第三階段計劃計劃於 2025 年第一季啟動。

Finally, while our current primary objective is to initiate our zaltenibart PNH Phase 3 trials quickly and to drive them to successful completion, we remain acutely focused on the wide applicability of MASP-3 and alternative pathway inhibition.

最後，雖然我們目前的主要目標是快速啟動 zaltenibart PNH 3 期試驗並推動其成功完成，但我們仍然高度關注 MASP-3 和替代途徑抑制的廣泛適用性。

As previously discussed, competitors with other agents targeting the alternative pathway have substantially de-risked for zaltenibart an array of indications beyond PNH. These include IgA nephropathy, C3 glomerulopathy, and AIDS-related macular degeneration. All of these and others, zaltenibart could provide significant patient benefits over currently available treatments, specifically better safety, more consistent efficacy, and superior dosing convenience and compliance.

如前所述，與其他針對替代途徑的藥物的競爭對手已經大大降低了扎替尼巴特 PNH 以外的一系列適應症的風險。這些包括 IgA 腎病、C3 腎小球病和愛滋病相關黃斑部病變。所有這些和其他方面，與目前可用的治療方法相比，扎替尼巴特可以為患者帶來顯著的益處，特別是更好的安全性、更一致的療效以及優越的給藥便利性和依從性。

I'll now turn back to our lectin pathway franchise and update you on OMS1029, our next generation long-acting inhibitor of MASP-2. We've now successfully completed both the single and multiple ascending dose Phase 1 studies of OMS1029. The results support once-quartered redosing administered either subcutaneously or intravenously. OMS1029 remains well tolerated with no safety concerns identified.

現在我將回到我們的凝集素途徑專營權並向您介紹 OMS1029 的最新情況，OMS1029 是我們的下一代長效 MASP-2 抑制劑。我們現已成功完成 OMS1029 的單次和多次劑量遞增的 1 期研究。結果支持每季度一次皮下或靜脈內重新給藥。OMS1029 仍然具有良好的耐受性，未發現任何安全性問題。

As previously disclosed, we're evaluating several chronic large value indications for potential development of OMS1029, one of these being neovascular age-related macular degeneration, also known as wet AMD.

如同先前所揭露的，我們正在評估 OMS1029 潛在開發的幾個慢性大價值適應症，其中之一是新生血管性老年黃斑部病變，也稱為濕性 AMD。

In a pre-clinical murine model of wet AMD, MASP-2 inhibition showed good efficacy. We are now running a primate study comparing OMS1029 to Eylea, which by recent report holds the major revenue share in AMD. We look forward to receiving those data later this quarter.

在濕性 AMD 的臨床前小鼠模型中，MASP-2 抑制顯示出良好的療效。我們現在正在進行一項靈長類動物研究，將 OMS1029 與 Eylea 進行比較，根據最近的報告，Eylea 佔據了 AMD 的主要收入份額。我們期待在本季稍後收到這些數據。

As discussed on our last call, all currently approved treatments for wet AMD, be it Eylea, Lucentis, they all require frequent intravitreal injections, meaning injections directly into the posterior chamber of the eye. By contrast, because MASP-2 is produced only in the liver, MASP-2 inhibition throughout the body is achieved through systemic administration by any route of administration, whether that be intravenously, subcutaneously, or orally.

正如我們上次電話會議中所討論的，目前所有批准的濕性 AMD 治療方法，無論是 Eylea 還是 Lucentis，都需要頻繁的玻璃體內注射，這意味著直接注射到眼後房。相較之下，因為MASP-2僅在肝臟中產生，所以透過任何給藥途徑（無論是靜脈內、皮下或口服）全身給藥，可以實現全身的MASP-2抑制。

Wet AMD is a very large market, and our work to date indicates that an effective therapeutic that does not require patients to receive painful injections in their eyes would be regarded by both patients and their physicians as a highly attractive alternative.

濕性 AMD 是一個非常大的市場，迄今為止我們的工作表明，一種不需要患者接受痛苦的眼睛注射的有效治療方法將被患者及其醫生視為一種極具吸引力的替代方案。

As detailed in earlier earnings calls and during our annual shareholder meeting last June, we continue our work with narsoplimab and MASP-2 inhibition in both severe acute and long COVID, or PASC, as well as an acute respiratory distress, or ARDS, including H1N1 and the recently concerning H5N1.

正如先前的財報電話會議和去年6 月的年度股東大會上詳細介紹的那樣，我們將繼續開展narsoplimab 和MASP-2 抑制藥物治療嚴重急性和長期COVID（PASC）以及急性呼吸窘迫（ARDS）（包括H1N1 ）的工作。

In addition to the wide therapeutic potential of MASP-2 inhibition, we're also developing a diagnostic approach for these broad indications. Adoption and utilization of a diagnostic for MASP-2 and lectin pathway hyperactivation would not only help physicians and their patients in early diagnosis, but would also drive adoption and utilization of narsoplimab and other members of our MASP-2 inhibitor therapeutic franchise across these diseases. As they become available, we'll share further important updates on our efforts on this front.

除了 MASP-2 抑制的廣泛治療潛力之外，我們還在開發這些廣泛適應症的診斷方法。採用和利用MASP-2 和凝集素路徑過度活化的診斷方法不僅可以幫助醫生及其患者進行早期診斷，還可以推動narsoplimab 和我們的MASP-2 抑制劑治療系列的其他成員在這些疾病中的採用和利用。當它們可用時，我們將分享我們在這方面的努力的進一步重要更新。

In addition to our MASP-2 and MASP-3 antibodies, narsoplimab, zaltenibart, and OMS1029, our growing complement franchise includes small molecule orally available MASP-2 and MASP-3 inhibitor programs. Both these programs are progressing rapidly with lessons learned from our MASP-2 small molecule program being applied to and levered for advancing development of our MASP-3 orally administered agents.

除了我們的 MASP-2 和 MASP-3 抗體、narsoplimab、zaltenibart 和 OMS1029 之外，我們不斷增加的補充產品系列還包括小分子口服 MASP-2 和 MASP-3 抑制劑專案。這兩個計畫都進展迅速，我們從 MASP-2 小分子計畫中學到的經驗教訓被應用於並用於推進我們的 MASP-3 口服製劑的開發。

Let's turn now to OMS527, our PD-7 inhibitor program aimed at treating addictions, compulsions, and movement disorders. As previously disclosed, the National Institute on Drug Abuse, or NIDA, requested that we shift our focus for OMS527 from nicotine addiction to the unmet and urgent need for a treatment for cocaine use disorder.

現在讓我們來看看 OMS527，我們的 PD-7 抑制劑計畫旨在治療成癮、強迫症和運動障礙。正如先前披露的，國家藥物濫用研究所 (NIDA) 要求我們將 OMS527 的重點從尼古丁成癮轉移到對可卡因使用障礙治療的未滿足和迫切需求。

Our program is funded by NIDA through a $6.7 million grant. We anticipate receiving results later this year from a preclinical toxicology study of primates exposed concurrently to cocaine and OMS527.

我們的計畫由 NIDA 撥款 670 萬美元資助。我們預計今年稍後將收到同時暴露於可卡因和 OMS527 的靈長類動物進行的臨床前毒理學研究的結果。

Assuming positive results in the toxicology study, together with NIDA, we plan to initiate next year a randomized double-blind in-patient clinical trial evaluating OMS527 treatment in individuals with cocaine use disorder.

假設毒理學研究取得正面結果，我們計劃明年與 NIDA 一起啟動一項隨機雙盲住院臨床試驗，評估 OMS527 對可卡因使用障礙患者的治療。

As also discussed in earlier calls, we're exploring the potential use of OMS527 in movement disorders, specifically levodopa-induced dyskinesias, or LID. This is a major problem in patients with Parkinson's disease and is caused by levodopa, the most common therapeutic used in Parkinson's disease. There is no good treatment for LID, and as a result, LID certainly represents a large and essentially untapped commercial market for an effective and safe therapeutic.

正如先前電話會議中所討論的，我們正在探索 OMS527 在運動障礙中的潛在用途，特別是左旋多巴引起的運動障礙 (LID)。這是帕金森氏症患者的一個主要問題，是由左旋多巴引起的，左旋多巴是帕金森氏症最常用的治療方法。LID 尚無良好的治療方法，因此，LID 無疑代表了一個巨大且基本上尚未開發的有效且安全的治療方法的商業市場。

We'll wrap up our program discussion with our family of biologic and cellular immuno-oncology platforms, namely our signaling-driven immunomodulators, our antigen-driven immunomodulators that function not only as therapeutics but also as cancer vaccines, our oncotoxins, and our adoptive T-cell therapy that requires no engineering and could supersede CAR-T, able to treat both liquid and solid tumors and amenable to multiple repetitive dosing.

我們將透過我們的生物和細胞免疫腫瘤學平台系列來結束我們的計劃討論，即我們的信號驅動的免疫調節劑、我們的抗原驅動的免疫調節劑（不僅可以作為治療劑，還可以作為癌症疫苗）、我們的癌毒素和我們的收養劑。

All these platforms are advancing quickly. Around each, we continue generating really a steady stream of compelling and exciting in vivo data, expanding and strengthening their respective intellectual property positions.

所有這些平台都在快速發展。圍繞著每個領域，我們持續產生源源不絕的引人注目且令人興奮的體內數據，擴大和加強各自的智慧財產權地位。

Each of our IO platforms represent novel approaches to cancer treatment designed to, one, target both cell surface and intracellular cancer targets for broad cancer applicability; two, increase CD4, CD8, and memory T-cell levels to attack cancer cells effectively and sustainably; three, create immune memory against future relapse; and four, eliminate the need for costly and time-intensive cellular modification or engineering.

我們的每個 IO 平台都代表了癌症治療的新穎方法，其設計目的是：第一，針對細胞表面和細胞內癌症靶點，以實現廣泛的癌症適用性；二、提高CD4、CD8和記憶T細胞水平，有效且可持續地攻擊癌細胞；三、建立免疫記憶，防止未來復發；第四，消除昂貴且耗時的細胞改造或工程的需要。

Given the growing volume and breadth of encouraging data, we are working diligently to include them in our patent applications. Once all initial patent applications have been filed, we will share data through one or more public avenues.

鑑於令人鼓舞的數據數量和廣度不斷增長，我們正在努力將它們納入我們的專利申請中。一旦提交了所有初始專利申請，我們將透過一個或多個公共途徑共享資料。

I'll now turn the call over to David Borges, our Chief Accounting Officer, to go through a more detailed discussion of our financial results. David?

我現在將把電話轉給我們的首席會計官大衛博爾赫斯，以對我們的財務業績進行更詳細的討論。大衛？

Thanks, Greg. Our net loss for the second quarter of 2024 was $56 million or $0.97 per share compared to a net loss of $37.2 million or $0.63 per share in the first quarter of 2024.

謝謝，格雷格。我們 2024 年第二季的淨虧損為 5,600 萬美元，即每股 0.97 美元，而 2024 年第一季的淨虧損為 3,720 萬美元，即每股 0.63 美元。

In Q2, we incurred $17.6 million of R&D expense related to the manufacture of narsoplimab drug substance lots that commenced in October 2023 and were delivered in the second quarter of this year. Recall that our accounting policy is to expense all manufacturing costs related to drug candidates until regulatory approval is reasonably assured in either the US or the European Union.

第二季度，我們產生了 1760 萬美元與 narsoplimab 原料藥批次的生產相關的研發費用，這些批次於 2023 年 10 月開始，並於今年第二季度交付。回想一下，我們的會計政策是費用化與候選藥物相關的所有製造成本，直到美國或歐盟的監管部門批准合理保證為止。

We also incurred $21.2 million in discounted term loan related debt repurchase and $1.9 million for term loan related transaction costs in the second quarter. Costs and expenditures of this significance, the narsoplimab manufacturing amounts, the discounted term loan related debt repurchase and the term loan related transaction cost representing a total of $40.7 million are not expected in a foreseeable future.

第二季度，我們也產生了 2,120 萬美元的折現定期貸款相關債務回購費用和 190 萬美元的定期貸款相關交易成本。如此重要的成本和支出、narsoplimab 的生產量、折現定期貸款相關債務回購和定期貸款相關交易成本總計 4,070 萬美元，預計在可預見的未來不會出現。

As of June 30, 2024, we had $158.9 million of cash and investments on hand, a decrease of $71.4 million from March 31, 2024. Two second-quarter transactions significantly affected our Q2 ending cash balance. One, we paid $10.7 million in the second quarter and $6.9 million in the first quarter related to six lots of narsoplimab drug substance which were commenced in October 2023 and released in the current quarter. And two, we paid $21.2 million to Highbridge and Athyrium in connection with the discounted debt repurchase in June 2024.

截至2024年6月30日，我們手頭現金及投資為1.589億美元，較2024年3月31日減少7,140萬美元。第二季的兩筆交易嚴重影響了我們第二季末的現金餘額。首先，我們在第二季支付了 1,070 萬美元，在第一季度支付了 690 萬美元，涉及 6 批 narsoplimab 原料藥，這些原料藥於 2023 年 10 月開始使用，並在本季度發布。第二，我們就 2024 年 6 月的折現債務回購向 Highbridge 和 Athyrium 支付了 2,120 萬美元。

Costs and expenses from continuing operations for the second quarter were $59.2 million, which was an increase of $20.1 million from the first quarter of this year. The increase was driven by narsoplimab drug substance manufacturing, zaltenibart clinical research and the discounted debt repurchase transaction cost, all partially offset by lower expenditures on the IGAN clinical trial as we closed down that trial.

第二季持續營運成本和費用為5,920萬美元，比今年第一季增加了2,010萬美元。這一增長是由 narsoplimab 原料藥製造、zaltenibart 臨床研究和折扣債務回購交易成本推動的，所有這些都被我們關閉 IGAN 臨床試驗的支出減少所部分抵消。

Interest expense for the second quarter was $9.2 million, which is $1 million higher than the first quarter of this year due to increased interest expense associated with the DRI transaction that was finalized in February 2024. The primary drivers of interest expense are the 2026 notes and the DRI OMIDRIA royalty obligation.

第二季的利息支出為 920 萬美元，比今年第一季增加了 100 萬美元，原因是 2024 年 2 月完成的 DRI 交易相關的利息支出增加。利息支出的主要驅動因素是 2026 年票據和 DRI OMIDRIA 特許權使用費義務。

Interest and other income for the second quarter was $3.2 million, which was slightly lower compared to interest and other income from the first quarter of this year due to lower cash balances to invest.

第二季的利息和其他收入為 320 萬美元，由於投資現金餘額減少，與今年第一季的利息和其他收入相比略有下降。

Income from discontinued operations in the second quarter of this year was $9.1 million and includes two primary components. One, $4.3 million of interest earned on the OMIDRIA contract royalty asset; two, $4.3 million of remeasurement adjustments to the OMIDRIA contract royalty asset.

今年第二季終止經營業務的收入為 910 萬美元，包括兩個主要部分。一、OMIDRIA 合約特許權使用費資產所賺取的 430 萬美元利息；二是對 OMIDRIA 合約特許權使用費資產進行 430 萬美元的重新計量調整。

As we have previously made clear, royalties earned are recorded as a reduction in the OMIDRIA contract royalty asset on our balance sheet and not in our income statement. OMIDRIA royalties for the second quarter were $10.9 million on OMIDRIA net sales of $36.4 million. This is compared to royalties of $9.4 million on first-quarter net sales of $31.2 million, a $5.2 million increase in net sales of the first quarter of 2024. The increase in net sales from the first quarter is consistent with historical OMIDRIA net sales trends.

正如我們先前明確指出的，所賺取的特許權使用費在我們的資產負債表上記錄為 OMIDRIA 合約特許權使用費資產的減少，而不是在我們的損益表中。OMIDRIA 第二季特許權使用費為 1,090 萬美元，OMIDRIA 淨銷售額為 3,640 萬美元。相比之下，第一季淨銷售額為 3,120 萬美元，特許權使用費為 940 萬美元，2024 年第一季淨銷售額增加了 520 萬美元。第一季淨銷售額的成長與歷史 OMIDRIA 淨銷售額趨勢一致。

As we discussed in last quarter's earnings call, in February 2024, we entered into an amended agreement with DRI, by which they acquired the right to receive all US OMIDRIA royalties payable by Rayner through December 31, 2031. We continue to hold all royalty rights to ex-US sales of OMIDRIA and, after December 31, 2031, all US royalty payments also accrued to Omeros. In addition, we have the opportunity to earn two sales contingent milestones, each up to $27.5 million with payment dates in January 2026 and January 2028.

正如我們在2024 年2 月的上季財報電話會議中討論的那樣，我們與DRI 簽訂了修訂後的協議，根據該協議，他們獲得了接收Rayner 在2031 年12 月31 日之前應付的所有美國OMIDRIA特許權使用費的權利。我們繼續持有 OMIDRIA 在美國以外銷售的所有特許權使用費，並且在 2031 年 12 月 31 日之後，所有美國特許權使用費也將計入 Omeros。此外，我們還有機會獲得兩個銷售或有里程碑，每個里程碑高達 2750 萬美元，付款日期為 2026 年 1 月和 2028 年 1 月。

As Greg mentioned, in June, we substantially strengthened our balance sheet to our debt repurchase and exchange transaction, whereby we repurchased and retired a portion of our 2026 notes for cash and a new term loan. The total principal amount of the 2026 notes exchanged was $118.1 million in exchange for total consideration of $88.3 million, consisting of a cash payment of $21.2 million and a new secured loan of $67.1 million, extending maturity on that portion of our debt out to June of 2028.

正如 Greg 所提到的，6 月份，我們大幅加強了債務回購和交換交易的資產負債表，其中我們回購並註銷了部分 2026 年票據，以換取現金和新的定期貸款。2026 年票據交換的本金總額為1.181 億美元，總代價為8,830 萬美元，其中包括2,120 萬美元的現金支付和6,710 萬美元的新擔保貸款，將我們這部分債務的到期日延長至2020 年6 月。

In addition, we have a $25 million delayed term loan available to be drawn on or prior to June 3, 2025, contingent on regulatory approval of narsoplimab in TA-TMA. The term loan is secured by substantially all our assets and carries an interest rate based on the current adjusted secured overnight financing rate, or SOPR, plus 875 basis points. The combined rate at June 30, 2024, was 14.2%.

此外，我們還有 2500 萬美元的延遲定期貸款可在 2025 年 6 月 3 日或之前提取，具體取決於 TA-TMA 中 narsoplimab 的監管批准。此定期貸款由我們幾乎所有資產提供擔保，利率基於當前調整後的擔保隔夜融資利率（SOPR）加上 875 個基點。截至 2024 年 6 月 30 日的綜合率為 14.2%。

The term loan agreement includes a covenant requiring us to maintain unrestricted cash and cash equivalents of at least $25 million. The $88.3 million of new term loan and total cash spent to repurchase debt represent a discount of approximately 25% or $29.8 million off of par.

定期貸款協議包括一項契約，要求我們保留至少 2500 萬美元的不受限制的現金和現金等價物。8,830 萬美元的新定期貸款和用於回購債務的現金總額折價約 25%，即較面額低 2,980 萬美元。

This transaction brings our remaining total of outstanding 2026 notes down by 55% from $216 million to a much more manageable $98 million. The $29.8 million discount from par has been recorded as a premium, i.e. an increase to the term loan being recognized as a gain on early extinguishment of debt.

這項交易使我們剩餘的 2026 年未償票據總額減少了 55%，從 2.16 億美元減少到更易於管理的 9,800 萬美元。面額的 2,980 萬美元折扣已記錄為溢價，即定期貸款的增加被確認為提前清償債務的收益。

The $29.8 million premium will be amortized against interest expense over the term of the loan, which results in an effective interest rate of 1.6% for financial statement purposes. The amount of interest expense expected to be recognized in the third quarter related to this new term loan is approximately $400,000.

2,980 萬美元的溢價將在貸款期間根據利息費用進行攤銷，因此就財務報表而言，實際利率為 1.6%。預計第三季確認的與這筆新定期貸款相關的利息費用約為 40 萬美元。

Now let's look at our expected third-quarter results. We expect overall operating costs from continuing operations in the third quarter to decrease by approximately $20 million. The decrease is primarily due to the significant expenses reported in the second quarter related to the receipt of narsoplimab drug substance, the cash paid to repurchase our 2026 notes, and transaction costs incurred in connection with the discounted debt repurchase.

現在讓我們來看看我們預期的第三季業績。我們預計第三季持續營運的整體營運成本將減少約 2,000 萬美元。減少的主要原因是第二季報告的與接收 narsoplimab 原料藥相關的大量費用、回購 2026 年票據支付的現金以及與貼現債務回購相關的交易成本。

Interest income for the third quarter should be nearly $2.5 million and interest expense should be approximately $8.2 million, a decrease of $1 million from the second quarter due to a decrease in our outstanding debt and the lower financial statement interest rate on the term loan.

第三季的利息收入應接近 250 萬美元，利息支出應約為 820 萬美元，比第二季減少 100 萬美元，原因是我們的未償債務減少以及定期貸款的財務報表利率較低。

And finally, income from discontinued operations should be in the $7 million to $8 million range.

最後，來自已終止經營業務的收入應在 700 萬至 800 萬美元範圍內。

With that, I'll turn the call back over to Greg.

這樣，我會將電話轉回給格雷格。

Thanks, David. Operator, let's please open the call to questions.

謝謝，大衛。接線員，讓我們開始提問。

(Operator Instructions) Steve Brozak, WBB.

（操作員說明）Steve Brozak，WBB。

I actually have two. The first one is, on the quarter in terms of the expenses, can you give us any modeling or any idea as to how this expense that we modeled in basically would factor into the future? And I've got to follow up after that, please.

我其實有兩個。第一個是，就本季的費用而言，您能否給我們任何模型或任何關於我們建模的費用基本上將如何影響未來的想法？之後我必須跟進，請。

I hope we were pretty clear, Steve, that those expenses that were the large expenses and charges and costs incurred in the second quarter were those that we don't expect to be repeated. There was a large manufacturing cost. There was a repurchase of the term loan related debt, $21.2 million, where again that was at 75% of par. So all of that got retired. And then there were the term loan related transaction costs. I'm not sure that answered your question, but if not, let me know and we can...

史蒂夫，我希望我們非常清楚，第二季發生的巨額開支、費用和成本是我們預計不會重複的費用。製造成本很高。回購了 2,120 萬美元的定期貸款相關債務，回購率為面額的 75%。所以所有這些都退休了。然後是與貸款相關的交易成本。我不確定這是否回答了您的問題，但如果沒有，請告訴我，我們可以...

Yeah, no. That covered it exactly. I just wanted to understand how to see about modeling into the future. And these were just obviously one-time expenses, which actually brings me on the manufacturing side. For the purposes of future, when you see approval, what else do you need now that you've got this manufacturing expense done? What else do you need to be able to go out there? And when you will get approval, what else would you need to literally go into the markets? And you can be as detailed as you want on that because I'm kind of curious to see how to model that in the future, please.

是的，不。這完全涵蓋了它。我只是想了解如何看待未來的建模。這些顯然只是一次性費用，這實際上讓我想到了製造方面。出於未來的目的，當您獲得批准時，既然您已經完成了製造費用，您還需要什麼？您還需要什麼才能出去？當您獲得批准後，您還需要什麼才能真正進入市場？您可以根據需要詳細說明這一點，因為我很好奇將來如何對其進行建模。

Well, there's one rather large component that we would need, which would be FDA approval. And we're working hard to secure that. When you look at the data, we think the data are very clear. And frankly, as I said, we have a meeting scheduled with FDA. Those were part of the prepared comments. And we believe we're in very good shape with respect to the data and the case for its approval. So we just need to play this out a bit. But that's really what we need in the US. We're also pursuing, as you know, EMA approval for narsoplimab.

嗯，我們需要一個相當大的組件，那就是 FDA 的批准。我們正在努力確保這一點。當你看數據時，我們認為數據非常清楚。坦白說，正如我所說，我們已安排與 FDA 舉行會議。這些是準備好的評論的一部分。我們相信，我們在數據和批准案例方面處於非常良好的狀態。所以我們只需要稍微發揮一下。但這確實是我們在美國所需要的。如您所知，我們也正在尋求 EMA 對 narsoplimab 的批准。

Let me stop and get Cathy to weigh in. And then I'll also ask Nadia and Andreas to comment if they have anything else to add from the commercial or clinical perspective. But Cathy, do you want to take that?

讓我停下來讓凱西參與一下。然後我也會請 Nadia 和 Andreas 從商業或臨床角度看他們是否還有其他需要補充的內容。但是凱茜，你想接受這個嗎？

I think also part of the question was when we get approval, what next? And we are well positioned and we're anticipating and we're preparing for success. And we're set up in terms of our packaging operations and that sort of thing from the regulatory perspective. So we are definitely planning for success in terms of what we have lined up, as you said, Steve, when we get approval. But I'll let commercial and clinical teams weigh in as well.

我認為問題的一部分是當我們獲得批准時，下一步是什麼？我們已做好準備，我們正在期待並為成功做好準備。從監管角度來看，我們在包裝業務和此類事情方面已經做好了準備。因此，正如你所說，史蒂夫，當我們獲得批准時，我們肯定會根據我們所安排的內容來計劃成功。但我也會讓商業和臨床團隊參與其中。

I'll jump in. And we are planning for success and have been. And so we have everything laid out down to the last month and gated behind key decisions all the way down to brand name as well and making sure all of that is ready to go. So that's kind of the good news of the delay is that we could plan for this. There are multiple things that we still need and everything is being planned for and progressed.

我會跳進去。我們正在為成功做計劃，而且已經做到了。因此，我們把上個月的一切都安排好了，並在關鍵決策的背後一直到品牌名稱，並確保所有這些都準備就緒。因此，延遲的好消息是我們可以為此做好計劃。我們仍然需要很多東西，一切都在計劃和進展中。

Well, from a clinical point of view, I think what we need or after approval is patients and physicians ready to use the product. And if our expanded access program is any indication, then they're ready there. They want narsoplimab. They're using it in their patients, especially in patients that fail currently available treatments. So we're encouraged that we can bring something to market that is needed and will help patients.

嗯，從臨床的角度來看，我認為我們需要或批准後是患者和醫生準備好使用該產品。如果我們的擴展訪問計劃有任何跡象的話，那麼他們已經準備好了。他們想要 narsoplimab。他們將其用於患者，尤其是目前可用治療失敗的患者。因此，我們感到鼓舞的是，我們可以將需要的東西推向市場，並幫助患者。

And also, the international scientific community seems to look differently at transplant associated TMA in the last years. They've issued a harmonization of the diagnostic criteria. They've identified clear high risk and treatment criteria.

而且，近年來國際科學界似乎對移植相關的 TMA 有著不同的看法。他們發布了統一的診斷標準。他們已經確定了明確的高風險和治療標準。

So to Greg's point, the only thing we need is approval.

因此，就格雷格而言，我們唯一需要的就是批准。

As I think everyone has relayed in their comments, we believe we are well prepared and frankly well deserving of having narsoplimab approved. Patients clearly need it. Patients clearly are using it. Physicians using it, as Andreas said, through the expanded access program. And remember, the only thing they have available now are all off-label therapies.

正如我認為每個人都在評論中轉述的那樣，我們相信我們已經做好了充分的準備，坦白說，我們非常值得 narsoplimab 獲得批准。患者顯然需要它。患者顯然正在使用它。正如安德烈亞斯所說，醫生透過擴大訪問計劃來使用它。請記住，他們現在唯一可用的是標籤外療法。

And as I think we pointed out in a previous call in the expanded access program where we treat both adult and pediatric patients, of the 136 patients when we did the data cut on the expanded access program, 53 of those had failed or stopped treatments with one or more other agents. And so, you really have to think about that as when narsoplimab was used in these patients, it was really catching a falling knife. And yet, despite that, about half of those patients did well and resolved their TMA. So that is a somewhat controlled experiment, an internal control, that you see the effect of narsoplimab.

正如我認為我們在之前的擴大准入計劃電話會議中指出的那樣，我們治療成人和兒童患者，當我們對擴大准入計劃進行數據削減時，在136 名患者中，其中53 名患者失敗或停止了治療一種或多種其他試劑。所以，你真的必須考慮一下，當 narsoplimab 用於這些患者時，它實際上是在接住一把掉落的刀。然而，儘管如此，大約一半的患者表現良好並解決了 TMA。所以這是一個有點受控的實驗，一個內部對照，你可以看到 narsoplimab 的效果。

So we are obviously pushing hard here, and we need to get to resolution quickly.

因此，我們顯然正在努力推動這一點，我們需要盡快找到解決方案。

Thank you for the iteration on all the steps that have been covered, that's something that frankly I'm thrilled that you've covered those bases, and I look forward to obviously the good news in the future.

感謝您對已涵蓋的所有步驟進行迭代，坦白說，我很高興您已經涵蓋了這些基礎，並且我顯然期待將來的好消息。

Olivia Brayer, Cantor.

奧利維亞·布雷爾，坎托。

Greg, can you tell us when that additional meeting with the FDA is expected to happen and whether there's any new information or data that they've asked for ahead of that meeting? And then I have a follow-up on the pipeline on 906.

Greg，您能否告訴我們預計何時與 FDA 舉行額外會議，以及他們在會議之前是否要求任何新資訊或數據？然後我對 906 的管道進行了跟進。

We won't specify the date of the meeting, and the information is -- as you might expect, information on our programs and responses to FDA questions. I think that's a fair representation, but again, let me see if Cathy wants to add anything specifically to that.

我們不會具體說明會議的日期，正如您所期望的那樣，這些資訊是有關我們的計劃和對 FDA 問題的答复的資訊。我認為這是一個公平的表述，但再次讓我看看凱西是否想對此添加任何具體內容。

Yeah, really nothing to add. I think you covered it, Greg.

是的，確實沒什麼好補充的。我想你已經涵蓋了，格雷格。

On 906, I just wanted to clarify that you guys do have the monotherapy data in-house. And if so, can you give us a sense for just level of confidence around that program and anything you can tell us at this point on the Phase 3 trial designs, both for PNH but also C3G?

關於 906，我只是想澄清一下，你們確實擁有內部單一療法數據。如果是這樣，您能否讓我們了解對該計劃的信心程度，以及您目前可以告訴我們的有關 PNH 和 C3G 第 3 階段試驗設計的任何資訊？

Let me hand that over to Steve Whitaker.

讓我把這個交給史蒂夫‧惠特克。

First, I'll talk about the monotherapy data. Unfortunately, we can't give you any specific details on that data because, as Greg said, that's been submitted to an ASH abstract and it's embargoed until ASH publishes that, which will come later in the year.

首先，我將談談單一療法的數據。不幸的是，我們無法向您提供有關該數據的任何具體細節，因為正如 Greg 所說，這些數據已提交給 ASH 摘要，並且在 ASH 發布（將於今年晚些時候發布）之前處於禁運狀態。

The data is strong. We're feeling positive about everything we're seeing, both in that adjunctive switch study as well as the data we've already presented in the naïve study. And Greg also mentioned that we've amended that protocol to develop data to help with dose selection.

數據很強。我們對所看到的一切都感到積極，無論是在輔助開關研究中還是在我們已經在幼稚研究中提供的數據。格雷格還提到，我們已經修改了該協議以開發數據來幫助劑量選擇。

Your second question was related to how the Phase 3 is moving forward.

你的第二個問題涉及第三階段的進展。

Well, I think one was also how are we viewing the program generally?

嗯，我想其中之一就是我們一般如何觀看該節目？

Very excited about the program. The data are coming in strongly. Good differentiators with competitors out there, both in the target as well as the drug and having tremendous response from both key opinion leaders as well as potential clinical trial sites who we're talking to now. The response has been uniformly positive in the advisory boards that we've conducted to date.

對這個計劃非常興奮。數據正在強勁湧現。與競爭對手的良好差異化優勢，無論是在標靶還是藥物方面，都得到了關鍵意見領袖以及我們現在正在交談的潛在臨床試驗場所的巨大反響。到目前為止，我們所進行的諮詢委員會的反應都是正面的。

Just a little anecdotal example, at European Hematology Association, we took clinical operations people with us to get ready for the Phase 3 trials. Not only do they have a lot of meetings set up with investigative sites who are interested, but people we had in contact with were coming up to our booth and asking to talk to them about potentially getting their patients into the clinical trials.

舉一個小例子，在歐洲血液學協會，我們帶著臨床操作人員一起為三期試驗做好準備。他們不僅與有興趣的研究機構舉行了許多會議，而且我們接觸過的人也來到我們的展位，要求與他們討論是否有可能讓他們的患者參與臨床試驗。

So there's a lot of excitement out there around the program, which is keeping us very excited.

因此，圍繞該計劃有很多令人興奮的事情，這讓我們非常興奮。

Andreas, collectively, your views of the Phase 3 program and upcoming -- do you anticipate any change in the response we're seeing in patients between the Phase 2 program and what we could see in the Phase 3?

Andreas，總的來說，您對第 3 階段計劃和即將到來的計劃的看法 - 您預計我們在第 2 階段計劃和第 3 階段計劃之間看到的患者反應會有什麼變化嗎？

I think even in the Phase 2, although the Phase 2 studies are naturally small, we've a fairly representative set of patients. And so we expect to see very similar results in the Phase 3 program.

我認為即使在第二階段，雖然第二階段的研究自然規模很小，但我們有一組相當有代表性的患者。因此，我們預計在第三階段計劃中會看到非常相似的結果。

I agree. Everything is looking good.

我同意。一切看起來都不錯。

Serge Belanger, Needham & Company.

謝爾蓋貝蘭格 (Serge Belanger)，李約瑟公司。

This is John on for Serge today. My first question regarding zaltenibart and the C3G program. You're currently still enrolling patients for the Phase 2 study. Do you have any timelines set to complete that study ahead of the potential launch in the first quarter of next year and whether or not there could be any potential overlap between Phase 2 and Phase 3 during that time?

這是今天為 Serge 發言的約翰。我的第一個問題是關於 zaltenibart 和 C3G 計劃。您仍在招募患者參加第 2 期研究。您是否設定了在明年第一季可能啟動之前完成研究的時間表，以及在此期間第二階段和第三階段之間是否可能存在任何潛在重疊？

My second question was regarding OMS1029. You're expecting data in the third quarter of this year. Pending good data, how quickly do you think you'd be able to get this into the clinic following the study?

我的第二個問題是關於 OMS1029 的。您預計今年第三季的數據。在獲得良好的數據之前，您認為研究後多久才能投入臨床？

I'll hand this off to clinical in a moment, but as I mentioned, we think that we will have data from the C3G Phase 2 study later this year or early next. And also, as I noted, we're planning to initiate the Phase 3 program early in 2025. Steve, again, do you want to take this on C3G and the question about potential overlap of a Phase 2 and Phase 3?

我稍後會將其交給臨床，但正如我所提到的，我們認為我們將在今年稍後或明年初獲得 C3G 2 期研究的數據。而且，正如我所指出的，我們計劃在 2025 年初啟動第三階段計劃。Steve，您想再討論一下 C3G 以及有關第 2 階段和第 3 階段潛在重疊的問題嗎？

I would actually anticipate having an overlap between Phase 2 and Phase 3. The Phase 2 study has your improvement. And if you recall, I believe it was seven patients that Novartis presented at an ASH meeting or an ERA meeting. So you can get a good sense of efficacy with small numbers of patients in an open-label study. As soon as we gather the data to see a strong efficacy signal, we would start initiating Phase 3 activities immediately before we run the study out completely.

我實際上預計第二階段和第三階段會有重疊。第二階段研究讓你有所進步。如果您還記得的話，我相信諾華在 ASH 會議或 ERA 會議上介紹了七名患者。因此，您可以在開放標籤研究中透過少量患者獲得良好的療效。一旦我們收集數據並看到強烈的功效訊號，我們就會在研究完全結束之前立即開始啟動第三階段活動。

Your second question about 1029 was how quickly following data we think we could be in the clinic, and I'm assuming meaning in the clinic in a Phase 2 study. Andreas, do you have any thoughts on that?

你關於 1029 的第二個問題是，我們認為我們可以在臨床中追蹤數據的速度有多快，我假設在臨床 2 期研究中有意義。安德烈亞斯，你對此有什麼想法嗎？

If the data are as clear as we would hope, then I think we would get right to finalizing a clinical trial design. We would get the necessary input from the opinion leaders in the field to make sure that the trial design makes sense and will deliver a clear answer. So I would expect us to move into the clinic sometime early in 2025.

如果數據像我們希望的那樣清晰，那麼我認為我們就可以最終確定臨床試驗設計。我們將從該領域的意見領袖那裡獲得必要的意見，以確保試驗設計有意義並提供明確的答案。因此，我預計我們將在 2025 年初的某個時候進入診所。

Again, the objective of the primate study is to run a comparator. So we're looking at the effects of 1029 versus Eylea in these animals. So we think that that model is representative or pretty well predictive of what one would see in the human. And that way I think we'll get a pretty good sense coming out of these primate studies. And as Andreas said, we're hoping to see the data that we expect.

同樣，靈長類動物研究的目的是運行比較器。因此，我們正在研究 1029 與 Eylea 對這些動物的影響。因此，我們認為該模型能夠代表或很好地預測人們在人類身上看到的情況。這樣我想我們就能從這些靈長類動物研究中得到很好的認識。正如安德烈亞斯所說，我們希望看到我們期望的數據。

This does conclude our question-and-answer session. I would now like to turn it back to Dr. Demopulos for final remarks.

我們的問答環節到此結束。現在我想請德普洛斯博士做最後的演講。

Thank you, operator. And again, thank you all for joining this afternoon.

謝謝你，接線生。再次感謝大家今天下午的參與。

I think we've laid out clear paths around how we are advancing our program. We are really quite excited about the data we're generating. I know there again remains a question about narsoplimab in everyone's mind and timing. We're confident in the effect of narsoplimab in these patients, the need for narsoplimab in these patients. And we expect we're going to get there. As Cathy made clear, can't give you the when right now, but as soon as we have a better sense of the when, we will. But all of these programs are moving forward, and I think when you look at what's coming next in zaltenibart, it certainly looks to be exciting.

我認為我們已經為如何推進我們的計劃制定了明確的道路。我們對我們產生的數據感到非常興奮。我知道每個人的心中仍然存在著關於 narsoplimab 的問題和時機。我們對 narsoplimab 對這些患者的效果以及這些患者對 narsoplimab 的需求充滿信心。我們希望我們能夠實現這一目標。正如凱西明確表示的，現在無法告訴您具體時間，但一旦我們對具體時間有更好的了解，我們就會告訴您。但所有這些項目都在向前推進，我認為當你看到扎爾特尼巴特接下來發生的事情時，它看起來肯定令人興奮。

So with that, I will thank you all for your continued support, and have a good rest of the day.

因此，我將感謝大家一直以來的支持，並祝您今天休息愉快。

This concludes the program. You may now disconnect.

程式到此結束。您現在可以斷開連線。