Gyre Therapeutics Inc (GYRE) 2021 Q3 法說會逐字稿

Welcome to the Catalyst Biosciences Corporate Update Conference Call. This call is being recorded. (Operator Instructions) I would now like to turn the call over to Catalyst Biosciences. Please go ahead.

歡迎參加 Catalyst Biosciences 公司最新動態電話會議。本次電話會議正在錄音中。 （接線員指示）現在我想將電話轉給 Catalyst Biosciences。請繼續。

Thank you, operator, and good morning, everyone. Earlier today, we issued a press release announcing our corporate strategy update. You may access that release and the company's website under the Investors tab. With us today are Dr. Nassim Usman, President and Chief Executive Officer; and Dr. Grant Blouse, Chief Scientific Officer.

謝謝接線員，大家早安。今天早些時候，我們發布了一份新聞稿，宣布了公司策略更新。您可以在「投資者」標籤下造訪該新聞稿和公司網站。今天與我們一起出席的嘉賓有：總裁兼執行長納西姆·烏斯曼博士 (Dr. Nassim Usman) 和首席科學官格蘭特·布勞斯博士 (Dr. Grant Blouse)。

Following our prepared remarks, we will open the call for a question-and-answer session. Before we begin, I would like to remind you that various remarks that we make on this call contain forward-looking statements, including statements regarding expected regulatory and clinical actions and development and projected operating expenses. These statements are subject to risks, uncertainties and other factors that could cause actual results to differ materially from those expressed or implied in the statements themselves.

在我們準備好的發言之後，我們將開始問答環節。在開始之前，我想提醒大家，我們在本次電話會議上發表的各項言論包含前瞻性陳述，包括關於預期的註冊和臨床行動、開發以及預計的營運費用的陳述。這些陳述受風險、不確定性和其他因素的影響，可能導致實際結果與陳述本身明示或暗示的結果有重大差異。

All forward-looking statements made on this call are made based on the beliefs of Catalyst as of this date only. Future events or simply the passage of time may cause these beliefs to change. For a more detailed description of the risks that impact these forward-looking statements, please refer to our most recent quarterly report on Form 10-Q and our annual report on Form 10-K. Please be aware that you should not place undue reliance on the forward-looking statements made today.

本次電話會議中所做的所有前瞻性陳述均僅基於 Catalyst 截至當天的信念。未來事件或時間的流逝可能會導致這些信念發生變化。有關影響這些前瞻性陳述的風險的更詳細說明，請參閱我們最新的 10-Q 表季度報告和 10-K 表年度報告。請注意，您不應過度依賴今天所做的前瞻性陳述。

Finally, this conference call is the property of Catalyst Biosciences, and any recording, other duplication or rebroadcast without the expressed written consent of Catalyst is prohibited. With that, I will turn the call over to Nassim Usman, President and Chief Executive Officer.

最後，本次電話會議的版權歸 Catalyst Biosciences 所有，未經 Catalyst 明確書面許可，禁止任何錄音、複製或轉播。因此，我將把電話會議轉交給總裁兼執行長 Nassim Usman。

Thank you, Ana, and thank you, everyone, for joining us this morning. Our call today is to discuss our strategic decision to stop the clinical development of MarzAA and focus solely on our complement programs and protease medicines platform going forward. It is important to note this decision is not based on any concerns about the efficacy of MarzAA or the ultimate success of the trial.

謝謝安娜，也謝謝大家今天早上的到來。我們今天的電話會議是為了討論我們的策略決策：停止 MarzAA 的臨床開發，未來專注於我們的補體計畫和蛋白酶藥物平台。需要注意的是，這項決定並非基於對 MarzAA 療效或試驗最終成功的擔憂。

Rather, it is a strategic decision based on several factors, including a recently updated feasibility assessment in which we determined that it was not in the best interest of our stockholders to continue to finance MarzAA through the completion of the ongoing trials based on anticipated time lines and expense. Enrollment in our MarzAA clinical trial has been very slow. The pandemic created several logistical challenges that were difficult to overcome. Also, we faced increasing competition for study subjects due to the increasing availability of prophylaxis therapy, both approved and in late-stage development globally. Due to these factors, we estimate that the MAA-304 or Crimson-1 study could be completed by late 2023, a time frame, however, and financing hurdle we could not overcome without a partner.

相反，這是一個基於多種因素的策略決策，包括最近更新的可行性評估，我們認為，根據預期的時間和費用，繼續資助 MarzAA 直至完成正在進行的試驗並不符合我們股東的最佳利益。 MarzAA 臨床試驗的入組速度非常緩慢。疫情帶來了一些難以克服的後勤挑戰。此外，由於全球範圍內已獲批准和處於後期開發的預防性治療藥物越來越多，我們在研究受試者方面的競爭也日益激烈。基於這些因素，我們估計 MAA-304 或 Crimson-1 研究可能在 2023 年底完成，然而，如果沒有合作夥伴，我們無法克服這個時間和資金障礙。

As a protease platform company with a diverse pipeline, we face a strategic decision to either continue as a late-stage hemophilia company developing MarzAA or pivot to an earlier-stage protease medicines company developing our complement portfolio. Our protease platform and complement programs are very exciting and address multiple therapeutic areas ranging from ultra-orphan to large markets in areas such as nephrology, inflammation and ophthalmology.

作為一家擁有多元化產品線的蛋白酶平台公司，我們面臨著一項策略決策：是繼續作為一家開發MarzAA的後期血友病公司，還是轉型成為一家開發補體產品組合的早期蛋白酶藥物公司。我們的蛋白酶平台和補體計畫非常令人振奮，涵蓋多個治療領域，從超級孤兒藥到腎臟病、發炎和眼科等領域的大型市場。

We believe that this shift offers the best forward for the long-term success of the company and value creation for our stockholders. We plan to report data obtained in the MAA-304 or Crimson-1 trial to date.

我們相信，此次轉變將為公司的長期成功和股東價值創造帶來最佳前景。我們計劃報告迄今為止在 MAA-304 或 Crimson-1 試驗中獲得的數據。

The data will show MarzAA has successfully treated bleeds subcutaneously and that we have not observed any treatment-related adverse or thrombotic events. We are exploring opportunities to license or sell our MarzAA and DalcA portfolio.

數據將顯示MarzAA已成功治療皮下出血，且我們未觀察到任何治療相關的不良反應或血栓事件。我們正在探索授權或出售MarzAA和DalcA產品組合的機會。

Turning to our balance sheet for a moment. We reported cash of $64.5 million as of September 30, 2021. Halting development of MarzAA while we search for a partner will allow us to reduce our burn by approximately 40% through headcount and CRO cost reductions and invest our resources in our complement therapeutics and protease medicines platform. Complement is a very large and growing commercial opportunity that is only starting to be tapped. We believe that our key advantage is that our protease platform allows us to develop candidates with a differentiated approach to complement regulation, one that can rapidly engage and degrade high-abundancy targets in a way antibodies and small molecule inhibitors cannot.

先來看看我們的資產負債表。截至2021年9月30日，我們報告的現金為6,450萬美元。暫停MarzAA的開發，同時尋找合作夥伴，將使我們能夠透過減少員工數量和合約研究機構（CRO）成本，將資金消耗降低約40%，並將資源投入到我們的補體療法和蛋白酶藥物平台上。補體是一個非常巨大且不斷成長的商業機會，目前才剛開始被挖掘。我們相信，我們的關鍵優勢在於，我們的蛋白酶平台使我們能夠開發具有差異化補體調控方法的候選藥物，這種方法能夠以抗體和小分子抑制劑無法做到的方式快速作用並降解高豐度標靶。

We believe complement will open up opportunities in multiple disease settings ranging from ultra-orphan to large markets. We will move forward with our clinical development candidate, CB 4332, a subcu enhanced complement factor I as swiftly as possible and continue to generate candidates from our platform that we will either license or develop on our own. We believe the complement market holds tremendous potential that investing our resources in these programs is the optimal strategy going forward.

我們相信，補體將在多種疾病領域帶來機遇，從超級孤兒藥到大型市場。我們將盡快推進臨床開發候選藥物CB 4332（一種皮下增強型補體因子I），並繼續利用我們的平台開發候選藥物，這些候選藥物我們將獲得許可或自行開發。我們相信補體市場潛力巨大，將我們的資源投入這些項目是未來的最佳策略。

Now I will turn the call over to Grant Blouse, our Chief Scientific Officer, who will give more color on our complement pipeline, where it stands as of today and what you can expect in the near term. Grant?

現在，我將把電話轉給我們的首席科學官 Grant Blouse，他將詳細介紹我們的補體產品線，包括目前的狀況以及近期的預期。 Grant？

Thank you, Nassim. Our complement portfolio is led by the development candidates, CB 4332 and CB 2782-PEG, both of which originated from our internal discovery platform. CB 4332, a subcu-dosed enhanced complement [back drive] protease is designed to address the rare immunodeficiency where there is no current standard of care and potentially other complement-related disorders.

謝謝納西姆。我們的補體產品組合由候選藥物CB 4332和CB 2782-PEG主導，這兩種藥物均源自於我們內部的發現平台。 CB 4332是一種亞劑量增強型補體[反向驅動]蛋白酶，旨在治療目前尚無標準治療方法的罕見免疫缺陷症以及其他潛在的補體相關疾病。

CB 2782-PEG is our C3 degrader product candidate, which we've licensed to Biogen and is in preclinical development for the treatment of dry AMD. We plan to complete the transfer of Catalyst-supported activities to Biogen for this product candidate.

CB 2782-PEG 是我們的 C3 降解劑候選產品，我們已將其授權給百健 (Biogen)，目前正處於治療乾性 AMD 的臨床前開發階段。我們計劃將 Catalyst 支援的生產活動全部轉移給百健 (Biogen)。

We recently presented preclinical data on CB 4332, indicating its potential as an effective, longer-acting subcu therapy in CFI-deficient patients, that's complement factor I, by replacing the underlying deficient protease. We also enrolled the first 2 complement factor I-deficient subjects in our ConFIdence natural history study designed to assess and follow the clinical outcomes of patients with complement factor I deficiency and, therefore, support the CB 4332 development program. We plan on submitting an IND for 4332 in 2022 and initiating the first-in-human trial with this product candidate.

我們最近公佈了CB 4332的臨床前數據，顯示其有望成為CFI（補體因子I）缺乏患者的有效長效皮下療法，透過取代潛在的蛋白酶缺陷。我們也在ConFIdence自然史研究中招募了首批2例補體因子I缺乏的受試者，該研究旨在評估和追蹤補體因子I缺乏患者的臨床結局，從而支持CB 4332的開發項目。我們計劃於2022年提交4332的IND申請，並啟動該候選產品的首次人體試驗。

In parallel, we are generating additional leads from our platform technologies, providing an opportunity to either out-license or develop internally. We presented preclinical data last month and expect to nominate our next development candidate originating from our protein discovery platform in 2022.

同時，我們正在利用平台技術開發更多潛在產品，為外部授權或內部開發提供機會。我們上個月公佈了臨床前數據，並預計將於2022年提名下一個基於我們蛋白質發現平台的候選藥物。

Thank you, Grant. The progress and opportunities in our complement programs are quite impressive, which validates our decision to make the pivot now. We thank everyone who has been involved in the MarzAA program over the last several years, particularly our study subjects, clinical trial investigators and site staff, employees and investors.

謝謝你，格蘭特。我們補體專案的進展和機會令人印象深刻，這驗證了我們現在做出轉型的決定。我們感謝過去幾年參與 MarzAA 計畫的所有人，特別是我們的研究對象、臨床試驗研究人員和研究中心的工作人員、員工以及投資者。

It is our hope to see MarzAA on the market one day. We look forward to executing as a protease platform and complement company. With that, I will turn the call back over to the operator, and we will take questions. Operator?

我們希望有一天能看到 MarzAA 上市。我們期待成為一家蛋白酶平台和補充劑公司。好了，我現在把電話轉給接線生，我們會回答您的問題。接線生？

(Operator Instructions) Our first question is from Chris Raymond with Piper Sandler.

（操作員指示）我們的第一個問題來自 Piper Sandler 的 Chris Raymond。

This is Ally Bratzel on for Chris. So I guess, first, on CB 4332, I think you indicated you've enrolled the first 2 CFI-deficient patients in the natural history study. Can you just talk to early observations on that like how you got to find those patients? And then outline your goals for enrollment in the natural history trial prior to the IND filing. And also, clarify what clinical data we can expect to see from that program next year.

我是 Ally Bratzel，代替 Chris 發言。首先，關於 CB 4332，我記得您提到過，您已經在自然史研究中招募了首批 2 位 CFI 缺陷患者。您能否談談這方面的早期觀察，例如您是如何找到這些患者的？然後，概述一下您在 IND 申請之前參與自然史試驗的目標。另外，請說明我們明年可以從該計畫中看到哪些臨床數據。

Yes. Thanks. Let me take that in a couple of parts. So -- and just to make sure I get them all, so your first question was how the ConFIdence natural history study works and what the first 2 patients are, what's going on with the ones that we've enrolled. So the way that study works is there's 2 parts. There's ConFIrm, which is screening, and ConFIdence, which then once they've identified a CFI deficiency, they enroll in ConFIdence.

是的，謝謝。我分幾個部分來回答。為了確保我理解所有問題，你的第一個問題是 ConFIdence 自然史研究是如何運作的，前兩名患者是誰，我們招募的患者情況如何。這項研究分為兩個部分。 ConFIrm 是篩檢，而 ConFIdence 是一旦患者發現 CFI 缺陷，就可以加入 ConFIdence。

This allows us to follow them kind of on their standard of care, which there really is none for complement factor I deficiency, but how they're treated by their clinicians, what their progress is, most of these patients have recurrent bacterial infections, some have autoimmune encephalitis. So we're able to follow kind of the prevalence of their disease and how they do now in the absence of 4332. This allows us to then be able to compare back to kind of pre-drug when we start trials where we're interventional and looking at the long-term benefits of having 4332 on board. So that's the idea behind the natural history studies.

這使我們能夠追蹤他們的治療標準。補體因子I缺乏症實際上沒有標準治療，但我們可以追蹤臨床醫生如何治療他們，他們的病情進展如何。這些患者大多患有復發性細菌感染，有些患有自體免疫性腦炎。因此，我們能夠追蹤他們的病情流行情況，以及在沒有4332的情況下他們的病情如何。這使我們能夠在開始介入性試驗時，將病情與用藥前進行比較，並觀察使用4332的長期益處。這就是自然史研究背後的理念。

They also allow us now in our proactive search and outreach with KOLs to identify those patients that we have an opportunity to enroll in the first-in-human studies next year. So all this is going on now while we work towards filing an IND in 2022. And then -- so the first-in-human study, to the second part of your question, is designed to be a standard safety and a PK/PD study.

它們也使我們能夠主動搜尋並與關鍵意見領袖（KOL）進行聯繫，從而識別出明年有機會入組首次人體試驗的患者。目前，我們正在努力爭取在2022年提交IND申請。然後—關於您問題的第二部分，首次人體試驗旨在進行一項標準的安全性和PK/PD研究。

So you'll have a single ascending dose and a multiple ascending dose regimens for the study, the first of which is single ascending dose, and we expect to be able to have data by the end of the year on the initial PK, and we'll be looking for biomarkers as well because given the introduction of CB 4332, which is the CFI replacement therapy, we'll be able to follow complement biomarkers and then look for a rebalance of those biomarkers in the presence of complement factor I. Does that answer all your parts there?

因此，您將在研究中採用單次遞增劑量和多次遞增劑量方案，其中第一種是單次遞增劑量，我們預計能夠在今年年底之前獲得初始 PK 的數據，並且我們也會尋找生物標誌物，因為考慮到 CB 4332（CFI 替代療法）的引入，我們將能夠尋找補體生物標誌物，然後在補體因子 I 存在的情況下在補體因子 I 存在的情況下尋找這些生物標誌物，然後在補體因子 I 存在的情況下尋找這些生物標誌物。這回答了您的所有問題嗎？

Yes, yes. That's helpful. And maybe just a second one. Could you remind -- on that big partnership, can you just remind us of the Catalyst flow for 2782-PEG? And any event -- or what are the next events that would trigger milestone payments? And related to that, just on the cash runway. I think by our math, the cash burn has been a little over $20 million a quarter in 2021. So 40% reduction would take that to around $13 million a quarter. So that gives you 5 quarters of runway. So I guess, is that in line with your thinking? And just -- and any other -- or outlining the Catalyst flow over the next 5 quarters, again, I think, would just be helpful.

是的，是的。這很有幫助。也許還有第二個問題。關於那項重要的合作，您能否提醒我們2782-PEG的Catalyst流程？還有任何事件——或者說，接下來哪些事件會觸發里程碑付款？與此相關的，關於現金流。根據我們的計算，2021年每季的現金消耗略高於2000萬美元。因此，減少40%將使每季的現金消耗達到約1300萬美元。這樣一來，您就有了5個季度的現金流。我想，這符合您的想法嗎？另外，我想，您能概述一下未來5季的Catalyst流程嗎？

Yes. With respect to 2782-PEG, which is partnered with Biogen, Biogen is controlling the news flow around that molecule. So there's not much more I can say except that we are currently supporting them in IND-enabling activities, and we'll have to wait for them to update the street on what their next step is.

是的。關於與百健（Biogen）合作的2782-PEG，百健控制著該分子的新聞流。所以我能說的不多，只能說我們目前正在支持他們進行IND申報，我們必須等待他們宣布下一步。

Our next milestone from that partnership would be around entering the clinic. With respect to your question on the cash runway, yes, we estimate approximately 1 year is what we have at the moment. And I think that answers all of your questions, I believe?

我們此次合作的下一個里程碑將是進入臨床階段。關於您關於現金流的問題，是的，我們估計目前大約可以維持一年。我想這回答了您所有的疑問，對吧？

Yes. And then maybe just one last one, just on the kind of timing of the strategic decision and the decision process just for halting MarzAA. I think as recently as maybe September, the target was still to have that for submission by year-end and pivotal data in '22. So just wondering if there's anything like specific you can point to that changed over the last 2 months, that really drove the announcement of the strategic decision today. Or was it really just entirely driven by the pace of enrollment?

是的。也許還有最後一個問題，關於暫停 MarzAA 專案的策略決策時機和決策過程。我記得大概就在九月，目標仍然是在年底前提交，並在 2022 年提供關鍵數據。所以，您能否指出過去兩個月發生了哪些具體變化，真正推動了今天宣布戰略決策？還是說，這完全是由招生速度決定的？

Well, it's mostly the pace of enrollment in order -- we were very close to being able to do the DSM-V, but it's the long-term that was impacted by the recent assessment because if you do the math, it's the last patient in that counts, and it's about 14 months for that patient to get through their 10 bleeds. And so when we mapped that all out, the data was getting pushed into the latter part of 2023. And so with slow enrollment, that's what made our decision to switch to the new strategy.

嗯，主要是因為招募速度的問題——我們之前非常接近完成DSM-V，但最近的評估影響了長期效果，因為算一下，最後一位患者也算，而這位患者完成10次採血大約需要14個月。所以，當我們把所有數據都畫出來後，數據就被延後到2023年下半年了。由於招募速度緩慢，我們決定採取新策略。

Our next question is from Timur Ivannikov with Raymond James.

我們的下一個問題來自 Raymond James 的 Timur Ivannikov。

Can you guys just talk about Phase I/II MarzAA in rare blood disorders? Does that -- that basically cannot work without a Phase III study? And how far along was that Phase I/II study in terms of enrollments?

能不能談談 MarzAA 在罕見血液疾病領域的 I/II 期臨床試驗？如果沒有 III 期臨床試驗，那是不是根本就無法進行？就招募病人而言，I/II 期臨床試驗進展如何？

So you are correct that, that study could not -- it was not registrational. It was a rather small study looking at PK/PD. We will be sharing the big details of what's come out of that study along with the 304 study. But that study was also facing enrollment challenges. And without the 304 study leading to an approval, it was not viable as a stand-alone. And again, we just did not feel we had the capital to take that all the way to the end.

所以你說得對，那項研究無法──它不具備註冊性。它是一項規模相當小的研究，主要關注藥物動力學/藥效學。我們會與304研究分享該研究的詳細結果。但那項研究也面臨入組方面的挑戰。如果沒有304研究獲得批准，它就無法作為一項獨立研究。再說一次，我們覺得我們沒有足夠的資金來完成這項研究。

Okay. Got it. And then just in terms of the timing for the strategic decision, I think you mentioned there was a recent feasibility analysis. I'm not sure how much time you've had to think about this shutdown program halt. And I was wondering whether you had a chance to reach out to any potential partners already, including perhaps Novo Nordisk.

好的，明白了。關於策略決策的時間安排，我記得您提到最近進行了可行性分析。我不確定您花了多少時間考慮暫停這個停產項目。我想知道您是否已經聯繫過任何潛在的合作夥伴，其中可能包括諾和諾德。

We don't comment on any specific outreach, but we're always engaged in business development.

我們不對任何具體的外展活動發表評論，但我們始終致力於業務發展。

Okay. Okay. Got it. And then just one more question on the -- so in terms of the complement factor I program, can you just remind us how confident you are, it will not be causing immunogenicity since it is a modified complement factor I?

好的。好的。明白了。然後還有一個問題——那麼就補體因子I項目而言，您能否提醒我們一下，您有多有信心，它不會引起免疫原性，因為它是一種改良的補體因子I？

So the 4332 for CFI deficiency -- and thanks for the question, Timur. It's engineered for extended half life, and we've done -- and are in the process of doing a full immunogenicity risk assessment on that molecule. And we don't feel that there's any -- going to be any issues with that molecule and are very confident with its profile for going into the clinic. And what we do with these molecules is we do a full assessment in silico. We look at potential T-cell epitopes. We look at T-cell regitopes. We look at how the drug product does on its own when, kind of, if you will, fed to antigen-presenting cells and whether or not they induce a T-cell response.

所以，4332 是針對 CFI 缺乏症的——感謝 Timur 的提問。它經過特殊設計，半衰期更長，我們已經完成了——並且正在對該分子進行全面的免疫原性風險評估。我們認為該分子不會有任何問題，對其進入臨床階段的前景非常有信心。我們對這些分子所做的是進行全面的電腦模擬評估。我們會研究潛在的 T 細胞表位。我們會研究 T 細胞受體表位。我們會觀察藥物產品在被抗原呈現細胞（或說，抗原呈現細胞）處理時自身的表現，以及它們是否會誘導 T 細胞反應。

So we really do the full gamut of all preclinical risk assessment we can on 4332 as well as all our molecules going forward to ensure that they're not developing an immune response that would be concerning in the clinic.

因此，我們確實對 4332 以及我們未來的所有分子進行了全面的臨床前風險評估，以確保它們不會產生臨床上令人擔憂的免疫反應。

Okay. Got it. And then maybe one final question. And with regarding to the prevalence of CFI deficiency, it appears to be an ultra-orphan type of indication. I think your KOL, a few months back, Dr. Haerynck, she showed several patients in her practice. But I'm just trying to understand to what extent you know the prevalence in the U.S. and outside.

好的，明白了。然後也許還有最後一個問題。關於CFI缺乏症的盛行率，它似乎是一種罕見疾病。我記得您的關鍵意見領袖，幾個月前，Haerynck醫生，在她的診所裡給幾位病人看過這種藥物。但我只是想了解您對美國及其他國家CFI缺乏症的盛行率了解多少。

Yes. We have that ongoing right now. We've got boots on the ground looking and talking to KOLs and outreach to recruit for the trials. And just as a little bit of color, Dr. Haerynck herself has 12 patients with CFI deficiency. And we've built a pretty solid KOL network across the globe of researchers and clinicians that do have cohorts of CFI-deficient patients that are interested in partnering with us to go forward with the trials for these individuals. So we continue that enrollment and, of course, getting KOLs and patients in the U.S. and the North American content is quite important for us as well. So we're definitely doing that.

是的，我們現在正在進行這項工作。我們已派人實地考察並與關鍵意見領袖（KOL）洽談，並積極招募患者參與試驗。順便提一下，Haerynck 博士本人就有 12 位 CFI 缺乏症患者。我們在全球範圍內建立了一個相當穩固的關鍵意見領袖網絡，其中包括研究人員和臨床醫生，他們確實擁有一群 CFI 缺乏症患者，並有興趣與我們合作，為這些患者開展試驗。因此，我們會繼續招募患者，當然，在美國招募關鍵意見領袖和患者，北美的內容對我們來說也非常重要。我們肯定會這樣做。

Our next question is from Sam Slutsky with LifeSci Capital.

下一個問題來自 LifeSci Capital 的 Sam Slutsky。

I guess, first, just after the SAD/MAD data with 4332, just remind me where you go from there in terms of indication selection within CFI deficiency and then just general potential opportunity with the asset.

我想，首先，在 4332 的 SAD/MAD 資料之後，請提醒我您在 CFI 缺陷內的指示選擇方面從那裡走向何方，然後是資產的一般潛在機會。

Right. So after a SAD/MAD, so we would still be looking at moving forward. We're thinking about a Phase 2 would be in CFI-deficient patients. These patients have recurrent bacterial infections with or without the advent of vasculitis. And that's probably about 85% of the population of CFI, full CFI deficiency. And then you have another subgroup that has a prevalence of kind of a nephropathy -- not a nephropathy. We have those as well in partial deficiencies. That's an opportunity for expansion later.

對。所以在SAD/MAD之後，我們仍在考慮推進。我們正在考慮將二期臨床試驗用於CFI缺乏的患者。這些患者反覆出現細菌感染，無論是否伴隨血管炎。這大概佔CFI（完全CFI缺乏）族群的85%。此外，還有另一個亞組，其盛行率類似於腎臟病——而非單純腎臟病。部分CFI缺乏的患者也存在這種情況。這是一個以後擴展的機會。

On the first study after the SAD/MAD would probably be focused on the fully deficient patients and the opportunities for them. You can think of 4332, it's given as CFI as a natural regulator to the complement cascade. We think there might be potential in other complement-mediated diseases where you add in more complement [back drive]. And so we're exploring those opportunities now. And when we've kind of settled on where those opportunities lie, we'll talk more to them.

在SAD/MAD之後的首項研究可能會聚焦在完全缺陷的患者及其發展機會。例如，4332作為補體抑制劑（CFI）給藥，是補體級聯的天然調節劑。我們認為，在其他補體介導的疾病中，如果添加更多補體[反向驅動]，它可能具有治療潛力。因此，我們現在正在探索這些機會。當我們大致確定了這些機會所在後，我們會與他們進行更多溝通。

Got it. Okay. And then just lastly, so regarding the MarzAA studies that were ongoing, anything to comment on immunogenicity in any of this?

明白了。好的。最後，關於正在進行的 MarzAA 研究，您對其中的免疫原性有什麼看法嗎？

No, nothing to comment on today. We're completing all the follow-up studies, and we'll share that along with the efficacy results soon.

不，今天沒什麼好評論的。我們正在完成所有後續研究，很快就會與療效結果分享。

(Operator Instructions) Our next question is from Catherine Novack with Jones Research.

（操作員指示）我們的下一個問題來自瓊斯研究公司的凱瑟琳·諾瓦克。

Just with regard to the partial MarzAA efficacy safety data that you'd have on hand from the trial as it was conducted to date, when do you think you will be able to share that? And what aspects of that data set would be potentially attractive to a buyer or a partner? And how proactive do you expect to be in looking for a partner or buyer for the asset?

關於迄今為止進行的試驗中您掌握的部分 MarzAA 療效安全性數據，您認為什麼時候可以分享這些數據？這些數據的哪些方面對買家或合作夥伴有潛在的吸引力？您預計在為該資產尋找合作夥伴或買家時會有多積極主動？

Well, it will take a couple of months to wind down the trial because there are mandatory safety things we have to do for the patients who are still in the trial. And we have to clean the data, of course, and then we'll be able to share that data publicly. So a few months is our sort of general guidance. With respect to finding a partner, we will do that as expeditiously as possible.

嗯，結束試驗需要幾個月的時間，因為我們必須對仍在試驗中的患者採取強制性的安全措施。當然，我們必須清理數據，然後才能公開分享這些數據。所以幾個月是我們的一般指導時間。至於尋找合作夥伴，我們會盡快完成。

Got it. And then in terms of the complement degrader programs, after CB 4332 is in the clinic, how are you prioritizing the rest of the complement program in development in terms of indications?

明白了。那麼，就補體降解劑計畫而言，CB 4332 進入臨床後，您如何根據適應症優先考慮其餘正在開發的補體項目？

Yes. So good, great question. So I mean, what we're looking for in indications is that we obviously want the most successful with our next molecule that we're going to nominate as a development candidate next year. And so we're looking both at the indications market prevalence in the competitive market when we go forward. But really, we're also trying to identify opportunities where we, in disease areas such as nephrology and inflammation, where we think our differentiated protease approach will really have advantages over the current modalities that are targeting those indications.

是的。這個問題問得好。我的意思是，我們在適應症方面尋求的是，我們顯然希望我們明年將提名為開發候選藥物的下一個分子能夠取得最大的成功。因此，我們未來不僅會關注適應症在競爭激烈的市場中的流行程度，還會關注適應症的市場流行程度。但實際上，我們也在努力尋找機會，在腎臟病和發炎等疾病領域，我們認為我們差異化的蛋白酶方法將比目前針對這些適應症的治療方式更具優勢。

So we're really kind of looking at a whole global aspect of going forward for opportunity, matching the precision medicine of the protease platform approach. And really, the areas that we're focusing on is nephrology and inflammation.

所以，我們其實是從全球視角來尋找機遇，與蛋白酶平台方法的精準醫療相符。我們真正關注的領域是腎臟病學和發炎。

Okay. And then one last question. In terms of the Biogen collaboration, what's left to complete the transfer of CBIO-supported activities for the C3 degrader?

好的。最後一個問題。就 Biogen 合作而言，還需要做些什麼才能完成 CBIO 支持的 C3 降解劑活動的轉移？

I mean, so we don't really talk about specific details of the activities, but what we've spoken to publicly before with the 2782-PEG program is that we've been responsible for certain IND-enabling studies as well as CMC development. So when our part of those packages are complete, then they're handed over for Biogen to run with internally.

我的意思是，我們實際上不會談論具體活動的細節，但我們之前就2782-PEG項目公開表示，我們負責某些IND申報研究以及CMC開發。因此，當我們的部分工作完成後，就會交給百健公司進行內部運作。

We have reached the end of the question-and-answer session, and I will now turn the call over to management for closing remarks.

問答環節已經結束，現在我將把電話交給管理階層進行結束語。

Thank you, everyone, for joining us today. We look forward to keeping you updated on our progress.

感謝各位今天的參與。我們期待隨時向大家通報最新進展。

This concludes today's conference, and you may disconnect your lines at this time. Thank you for your participation.

今天的發表會到此結束，各位可以掛斷電話了。感謝各位的參與。