Cidara Therapeutics Inc (CDTX) 2025 Q3 法說會逐字稿

Good day, and welcome to the Cidara Therapeutics Q3 2025 conference call. (Operator Instructions) Please note, this event is being recorded.

大家好，歡迎參加 Cidara Therapeutics 2025 年第三季電話會議。（操作說明）請注意，本次活動正在錄影。

I would now like to turn the conference over to Brian Ritchie with LifeSci. Please go ahead.

現在我將把會議交給 LifeSci 的 Brian Ritchie。請繼續。

Thank you, operator, and good afternoon, everyone. With me today on the phone from Cidara Therapeutics is Dr. Jeff Stein, President and Chief Executive Officer. Following Dr. Stein's prepared remarks, we will be joined by Mr. Frank Karbe, Chief Financial Officer; Dr. Nicole Davarpanah, Chief Medical Officer; Dr. Les Tari, Chief Scientific Officer; and Mr. Jim Beitel, Chief Business Officer. To participate in a Q&A session.

謝謝接線員，大家下午好。今天和我一起透過電話的是來自 Cidara Therapeutics 的總裁兼執行長 Jeff Stein 博士。在史丹博士發表準備好的演講之後，我們將與首席財務官弗蘭克·卡貝先生、首席醫療官妮可·達瓦帕納博士、首席科學官萊斯·塔里博士以及首席商務官吉姆·貝特爾先生一同發言。參加問答環節。

Earlier this afternoon, Cidara released financial results and a business update for the third quarter ended September 30, 2025. Both the press release is available on the company's website. Please note that certain information discussed on the call today is covered under the safe harbor provision of the Private Securities Litigation Reform Act.

今天下午早些時候，Cidara 發布了截至 2025 年 9 月 30 日的第三季財務業績和業務更新。新聞稿和新聞稿均可在公司網站上查閱。請注意，今天電話會議中討論的某些資訊受《私人證券訴訟改革法案》安全港條款的保護。

Management will be making forward-looking statements subject to risks and uncertainties that could cause actual results to differ materially. These statements are qualified by the cautionary notes in today's press release and the company's SEC filings.

管理階層將做出前瞻性聲明，但這些聲明存在風險和不確定性，可能導致實際結果與預期結果有重大差異。這些聲明需遵守今天新聞稿和公司向美國證券交易委員會提交的文件中的警示性說明。

This call contains time-sensitive information accurate only as of today, November 6, 2025. Cidara undertakes no obligation to revise or update any forward-looking statements.

本次通話包含時效性訊息，僅截至 2025 年 11 月 6 日有效。Cidara不承擔任何修改或更新任何前瞻性聲明的義務。

With that, I'd like to turn the call over to Jeff Stein. Jeff?

接下來，我想把電話交給傑夫‧史坦。傑夫？

Thank you, Brian, and thank you all for joining us. We are pleased to report another very productive quarter at Cidara. Our lead candidate, CD388 has advanced into Phase III development on an accelerated time line in addition to other notable achievements, including the expansion of the patient population for our Phase III trial, receipt of breakthrough therapy designation from the FDA and securing funding from BARDA for this program.

謝謝你，布萊恩，也謝謝各位的參與。我們很高興地報告，Cidara 又迎來了一個碩果累累的季度。除了其他顯著成就外，我們的領先候選藥物 CD388 已加速進入 III 期開發階段，包括擴大 III 期試驗的患者群體、獲得 FDA 的突破性療法認定以及獲得 BARDA 的資助。

As with prior calls, we will focus our remarks on clinical and corporate updates. As a non-revenue-generating company, we will not have a dedicated section to review our quarterly financial results on this call but instead refer you to today's press release and 10-Q filing.

與以往的電話會議一樣，我們將重點討論臨床和公司最新進展。作為一家不產生收入的公司，我們不會在本次電話會議上專門安排時間回顧我們的季度財務業績，而是請您參閱今天的新聞稿和 10-Q 文件。

Cidara's proprietary Cloudbreak platform has been developed as a fundamentally new approach to treat and prevent serious diseases through the development of novel drug-Fc conjugates or DFCs, a new class of therapeutic that combines the precision of small molecules with the durability of antibodies.

Cidara 的專有 Cloudbreak 平台是一種全新的方法，透過開發新型藥物-Fc 偶聯物或 DFC 來治療和預防嚴重疾病。 DFC 是一種新型療法，它將小分子的精確性與抗體的持久性相結合。

Our lead candidate, CD388 is a highly potent, long-acting antiviral designed to deliver universal once-per-season prevention of seasonal and pandemic influenza by directly inhibiting viral proliferation. Its enhanced antiviral potency and durability make it a potentially transformational non-vaccine preventative of influenza that overcomes the limitations of existing vaccines and antivirals.

我們的領先候選藥物 CD388 是一種高效、長效的抗病毒藥物，旨在透過直接抑制病毒增殖，實現對季節性流感和流行性流感的普遍預防，只需一次即可。其增強的抗病毒效力和持久性使其成為一種具有變革意義的非疫苗流感預防藥物，克服了現有疫苗和抗病毒藥物的限制。

A key accomplishment during the third quarter was the start of our Phase III ANCHOR trial six months earlier than originally planned. This trial will evaluate the safety and efficacy of CD388 in populations at high risk for complications from influenza.

第三季的一項重要成就是我們的 III 期 ANCHOR 試驗比原計劃提前了六個月啟動。這項試驗將評估 CD388 在流感併發症高風險族群中的安全性和有效性。

Based on feedback from the FDA in our end of Phase II meeting, we believe that the ANCHOR trial, if successful, may support potential BLA approval of CD388 in the study populations examined in both the Phase IIb NAVIGATE study as well as the Phase III ANCHOR study.

根據 FDA 在我們 II 期臨床試驗結束會議上的回饋，我們認為，如果 ANCHOR 試驗成功，可能會支持 CD388 在 IIb 期 NAVIGATE 研究和 III 期 ANCHOR 研究的研究人群中獲得潛在的生物製品許可批准。

The ANCHOR trial was started in late September following a constructive end of Phase II meeting with the FDA at the end of August. Initiation of the study triggered a $45 million milestone payment to J&J, which was booked in Q3, but will be paid in Q4.

在 8 月底與 FDA 舉行了富有建設性的 II 期臨床試驗結束會議後，ANCHOR 試驗於 9 月下旬啟動。該研究的啟動觸發了強生公司獲得 4500 萬美元的里程碑付款，該款項已計入第三季度，但將在第四季度支付。

Originally, we plan to enroll participants aged 12 years of age and older with moderate to severe comorbidities as well as subjects who are immunocompromised. However, based on FDA feedback, we expanded enrollment to include healthy adults over 65, a large and growing group that is poorly protected by current influenza vaccines due to age-related declines in immune function. This change has two important implications.

最初，我們計劃招募 12 歲及以上、患有中度至重度合併症的參與者以及免疫功能低下的受試者。然而，根據 FDA 的回饋，我們擴大了招募範圍，將 65 歲以上的健康成年人納入其中。這是一個龐大且不斷增長的群體，由於與年齡相關的免疫功能下降，他們無法得到當前流感疫苗的充分保護。這項變化有兩個重要意義。

First, it more than doubles the initial number of patients who would potentially be eligible to receive CD388 from 50 million to well over 100 million people in the US second, it has helped facilitate faster enrollment. The study began in the Northern Hemisphere in late September.

首先，它使美國可能符合 CD388 治療條件的患者人數從 5,000 萬人增加到超過 1 億；其次，它有助於加快患者入組速度。這項研究於9月下旬在北半球啟動。

The primary endpoint is based on laboratory-confirmed influenza, body temperature of 37.2 degrees centigrade or 99 degrees Fahrenheit or greater and new or worsening of either two respiratory symptoms or one respiratory symptom and one new systemic symptom.

主要終點是基於實驗室確診的流感，體溫達到或超過 37.2 攝氏度或 99 華氏度，並且出現兩種新的呼吸道症狀或一種新的呼吸道症狀和一種新的全身症狀，或者出現這兩種呼吸道症狀或一種新的全身症狀，或出現這兩種呼吸道症狀或一種新的全身症狀。

We plan to enroll 6,000 participants in 150 sites, nearly 3 times the number of sites we used in the Phase IIb NAVIGATE study. All sites are now active and the study is over 50% enrolled, on track to achieve target enrollment in the Northern Hemisphere by December.

我們計劃在 150 個地點招募 6,000 名參與者，這幾乎是我們在 IIb 期 NAVIGATE 研究中使用的地點數量的 3 倍。所有試驗點目前均已投入使用，研究已完成 50% 以上的受試者招募，預計在 12 月實現北半球的目標招募人數。

An interim analysis, most likely in late Q1 2026, will assess the trial size, powering assumptions and event rate to determine if it is necessary to enroll participants in the Southern Hemisphere in the spring of 2026. CD388's progression into Phase III is supported by the strength of the compelling data from our Phase IIb NAVIGATE study, which met its primary endpoint, demonstrating statistically significant prevention of efficacy and a benign safety profile in all three doses tested.

中期分析很可能在 2026 年第一季末進行，屆時將評估試驗規模、功效假設和事件發生率，以確定是否有必要在 2026 年春季在南半球招募參與者。CD388 進入 III 期臨床試驗得益於我們 IIb 期 NAVIGATE 研究的有力數據，該研究達到了主要終點，證明在所有三個測試劑量中均具有統計學意義上的療效預防作用和良好的安全性。

Importantly, the NAVIGATE study showed that a single 450-milligram dose of CD388 provided 76.1% protective efficacy that extended through the entire flu season. We shared additional NAVIGATE data at key scientific meetings this fall. The data presented continues to reinforce CD388's differentiated profile as a long-acting broad-spectrum antiviral for influenza prophylaxis.

重要的是，NAVIGATE 研究表明，單次 450 毫克劑量的 CD388 可提供 76.1% 的保護效力，並持續整個流感季節。今年秋季，我們在重要的科學會議上分享了更多 NAVIGATE 數據。所呈現的數據持續強化了 CD388 作為一種長效廣譜抗病毒藥物在流感預防方面的差異化特性。

The pharmacokinetic data for the 450-milligram dose demonstrated sustained serum concentrations well above the targeted therapeutic threshold, supporting protection through the full flu season with a single 450-milligram dose in both influenza A and B strains, paired with a clean safety and tolerability profile, including low rates of injection site reactions, which further differentiates CD388 from vaccines. These results validate our dose selection for Phase III.

450 毫克劑量的藥物動力學數據顯示，持續的血清濃度遠高於目標治療閾值，支持在整個流感季節對甲型和乙型流感病毒株提供單次 450 毫克劑量的保護，同時具有良好的安全性和耐受性，包括注射部位反應發生率低，這進一步將 CD388 與疫苗區分開來。這些結果驗證了我們為 III 期臨床試驗所選擇的劑量。

Taken together, these findings strengthen our conviction that CD388 can offer clinically meaningful protection in populations at high risk for flu complications, independent of host immune status, setting it apart from both vaccines and currently approved antivirals.

綜上所述，這些發現強化了我們的信念，即 CD388 可以為流感併發症高風險族群提供具有臨床意義的保護，而與宿主的免疫狀態無關，這使其區別於疫苗和目前已批准的抗病毒藥物。

In early October, the FDA granted CD388 breakthrough therapy designation, recognizing preliminary clinical evidence of substantial improvement over existing options. The advantages to Cidara will be enhanced access to the FDA, including more frequent guidance, rolling data review and eligibility for priority review, all of which may accelerate development and regulatory timelines.

10月初，FDA授予CD388突破性療法認定，認可了其在臨床上較現有療法有顯著改善的初步證據。Cidara 的優勢在於能夠更便捷地獲得 FDA 的指導，包括更頻繁的指導、滾動資料審查和優先審查資格，所有這些都可能加快研發和監管進程。

CD388 also holds fast track status, and this latest recognition affirms the quality and promise of the clinical data we have generated. Also in October, we received an award valued up to $339 million from the Biomedical Advanced Research and Development Authority, or BARDA, to support expanded manufacturing and clinical development of CD388.

CD388 也獲得了快速通道資格，這項最新認可肯定了我們所產生的臨床數據的品質和前景。同樣在 10 月份，我們從生物醫學高級研究與發展局 (BARDA) 獲得了價值高達 3.39 億美元的獎勵，用於支持 CD388 的擴大生產和臨床開發。

The multiyear agreement is structured to include a base period and additional option periods. The base period valued at $58 million over the first 24 months will fund the onshoring of manufacturing to the United States, expanding our initial commercial supply chain. It will also support several important development activities, including a clinical trial to demonstrate comparability for a higher concentration formulation.

此多年期協議的結構包括基本期和附加選擇期。前 24 個月的基準期價值 5,800 萬美元，將用於資助製造業遷回美國，擴大我們最初的商業供應鏈。它還將支援幾項重要的研發活動，包括一項臨床試驗，以證明更高濃度配方的可比較性。

And alternative product presentations, nonclinical studies to further characterize CD388's activity against pandemic influenza strains and early work on clinical trial protocols for expanded populations. The option periods could provide up to an additional $281 million in funding to support further clinical and nonclinical studies of CD388 in targeted patient groups and broader population settings.

此外，還有替代產品展示、非臨床研究以進一步表徵 CD388 對流行性流感株的活性，以及​​針對擴大人群的臨床試驗方案的早期研究。選擇權期限可提供高達 2.81 億美元的額外資金，以支持在目標患者群體和更廣泛的人群環境中對 CD388 進行進一步的臨床和非臨床研究。

Thanks to our successful financing in June, we remain in a strong financial position. With approximately $476 million in cash at September 30, our Phase III development program is fully funded through completion in all scenarios, including potentially expanding the study to the Southern Hemisphere if needed.

由於六月融資成功，我們仍然保持穩健的財務狀況。截至 9 月 30 日，我們擁有約 4.76 億美元的現金，足以支持我們的 III 期開發計劃在所有情況下完成，包括必要時將研究擴展到南半球。

Before closing, I want to highlight that we plan to host a virtual R&D Day for the investment community on December 15. We'll provide a detailed update on the CD388 program, including enrolment progress, and we'll share insights from recent market research on the commercial opportunity for CD388. Further details will be announced shortly, and we look forward to your participation in this event.

在結束之前，我想強調一下，我們計劃在 12 月 15 日為投資界舉辦一場虛擬研發日。我們將詳細介紹 CD388 專案的最新進展，包括註冊進度，並分享近期市場調查中關於 CD388 商業機會的見解。更多詳情將於近期公佈，我們期待您的參與。

With that, I will turn it back to the operator to take your questions. Operator?

接下來，我將把電話交還給接線員，由他來回答您的問題。操作員？

(Operator Instructions) Anupam Rama, JPMorgan.

（操作說明）Anupam Rama，摩根大通。

Hi, this is Priyanka on for Anupam. At the interim analysis, how will the external statistician decide how many additional patients are needed to enroll? Thanks.

大家好，我是Priyanka，為您報道Anupam。在期中分析中，外部統計學家將如何決定需要招募多少額外的病人？謝謝。

Yeah, great question, Priyanka. Let me turn that question over to Dr. Davarpanah, our Chief Medical Officer. Nicole?

是的，問得好，普里揚卡。讓我把這個問題交給我們的首席醫療官達瓦爾帕納博士來回答。妮可？

Thank you, Jeff. Priyanka, thanks for the question. So as you know, the purpose of the interim analysis is to look at data at an early time point prespecified at the end of the Northern Hemisphere, approximately end of Q1 of next year to look at the events and to tell us essentially if the powering assumptions, the desired power target for the study was met. So there is a kind of a complex algorithm that has been created for this by our statistician.

謝謝你，傑夫。Priyanka，謝謝你的提問。如您所知，中期分析的目的是查看北半球末期（大約明年第一季末）預先設定的早期時間點的數據，以了解事件情況，並告訴我們研究的功效假設和預期功效目標是否已達到。因此，我們的統計學家為此創建了相當複雜的演算法。

Importantly, this is a statistician independent to the study, and they will be able to see this data, but will not share any of it with us, and so Cidara and the study team will not be informed. We will only be told if the powering assumptions have been met and whether we need to -- we are able to keep the sample size that we have with 6,000 or we need to add additional participants.

重要的是，這位統計學家是獨立於這項研究的，他可以看到這些數據，但不會與我們分享任何數據，因此 Cidara 和研究團隊不會被告知。我們只能得知是否滿足了統計功效假設，以及是否需要增加樣本量——我們能否保持現有的 6,000 人樣本量，還是需要增加參與者。

Maxwell Skor, Morgan Stanley.

麥克斯韋‧斯科爾，摩根士丹利。

Great, thank you for taking my question. Just a follow-up on the first question. Should we not expect at the interim analysis to see any sort of efficacy data across the cohorts? And also, I was wondering at the Investor Day, will you begin to introduce efficacy thresholds across cohorts that we can expect in the Phase III trial?

太好了，謝謝你回答我的問題。針對第一個問題，我再補充一點。我們是否應該期待在期中分析中看到各組之間的任何療效數據？另外，我想在投資人日上問一下，你們是否會在 III 期試驗中開始引入各組的療效門檻？

And the last question, is there a chance you could potentially include mild patients on the label if everything is positive in the Phase III trial? Thank you.

最後一個問題，如果 III 期試驗一切順利，是否有可能將輕症患者納入適應症範圍？謝謝。

Good questions, Max. Again, I'll turn that over to Nicole.

問得好，Max。接下來，我會把這個主題交給妮可。

Thanks, Jeff. Max, thank you for the question. So I think the initial question is, will we know any kind of efficacy data from the interim? And the answer is no. So this is essentially only the blinded status -- unblinded excuse me, statistician will see this information and will not share it with us.

謝謝你，傑夫。Max，謝謝你的提問。所以我認為最初的問題是，我們能否從中期數據中獲得任何療效數據？答案是否定的。所以這基本上只是盲測狀態——不盲測的統計員將會看到這些訊息，但不會與我們分享。

But I think one thing that maybe you're alluding to as well is we may end up enrolling the entire trial in the Northern Hemisphere. And at that time, there may be no need to actually even move into the Southern Hemisphere, and we will make a decision at that time if it's appropriate to kind of call that the efficacy analysis. However, we will not be informed by the interim analysis if we decide to do this.

但我認為你可能也暗示了一點，那就是我們最終可能會在北半球招募所有受試者。到那時，可能根本沒有必要真正進入南半球，到那時我們將決定是否適合稱之為功效分析。但是，如果我們決定這樣做，我們將不會收到中期分析的通知。

I appreciate the question as well about the different kind of subgroups in the trial and what we will be able to show. So we won't be able to show kind of any efficacy estimates for our December Investor Day. We will be able to share a little bit more about the population subgroups that we have enrolled.

我也很感謝您提出的關於試驗中不同類型的亞組以及我們將能夠展示什麼的問題。因此，我們將無法在 12 月的投資者日上展示任何功效評估結果。我們將能夠分享更多關於我們已招募的人口亞群體的資訊。

And I think that will be kind of very enlightening for all of us about the real-world high-risk and 65+ populations as well as IC populations that you can enroll in a trial like this. And you have to remind me of your third question.

我認為這對我們所有人來說都將是一次非常有啟發意義的經歷，讓我們了解現實世界中的高風險人群、65 歲以上人群以及可以參與此類試驗的 IC 人群。你得提醒我你的第三個問題。

Sorry, just one last one around mild patients who are immunocompromised and comorbid. Is there a potential for them to be included in the label?

抱歉，最後再補充一點，關於免疫功能低下且合併其他疾病的輕症患者。它們有可能被納入標籤嗎？

Thank you. That's an excellent question. So in our discussions with the FDA, as you know, they have asked us to expand the trial eligibility to anyone who's 65+. So that means 65 is healthy or those with low or mild comorbidities. And as you can imagine, we are going to enroll a lot of those participants with lower mild comorbidities. So I believe that there's an opportunity for that, but it will require further discussion with FDA.

謝謝。這是一個很好的問題。如您所知，在與 FDA 的討論中，他們要求我們將試驗資格擴大到 65 歲及以上的任何人。所以這意味著 65 歲是健康的，或只有少量或輕微的合併症。正如你所想，我們將招募許多患有較輕合併症的參與者。所以我認為這方面有機會，但這需要與FDA進一步討論。

And just to add to Nicole's comments, the high-risk populations are focused on the moderate to severe comorbidities.

補充妮可的評論，高風險族群主要關注中度至重度合併症。

Seamus Fernandez, Guggenheim Securities.

西莫斯·費爾南德斯，古根漢證券。

Great, thanks for the question. So, two from my side. First, we've heard a lot of updates from some of the flu vaccine manufacturers that flu vaccination rates are down. Wondering if you have a sense of the sort of flu vaccination rates in your study. I think previously, you'd anticipated 65% of the adults potentially having flu vaccination and then having CD388 on top of it. Just wondering how you're thinking about the impact of that and if that 65% is still consistent with your expectations?

太好了，謝謝你的提問。我這邊有兩個。首先，我們從一些流感疫苗生產商那裡聽到了很多關於流感疫苗接種率下降的消息。想知道您是否了解貴研究中流感疫苗接種率的大致情況。我認為之前你們預計會有 65% 的成年人接種流感疫苗，然後再加上 CD388。我想知道您如何看待這件事的影響，以及65%這個數字是否仍符合您的預期？

And then just my second question is on manufacturing and how manufacturing scale-up is progressing and what the needs are from a manufacturing scale up, whether it be with the three-dose or sorry, the three-needle regimen as it stands today? And then what it would take to bring us forward to the sort of vial formulation, which is more consistent with how flu vaccines are delivered today. Thanks so much.

我的第二個問題是關於生產製造，生產規模擴大進展如何，以及生產規模擴大需要哪些條件，無論是目前的三劑注射方案還是三針注射方案？然後，我們需要做些什麼才能使我們發展到小瓶裝的配方，這種配方與目前流感疫苗的給藥方式更加一致。非常感謝。

Great, thanks, Seamus. Yeah, clearly, you got it right. Our estimate of the flu vaccination rate in the ANCHOR study, which was based on prior clinical studies in the prior years, was 65%, given the fact that we are enrolling subjects with moderate to severe comorbidities, immunocompromised and more recently, over 65, all three populations, which tend to have high vaccination rates. And clearly, that has an impact on our -- the powering of the study.

太好了，謝謝你，西莫斯。是的，顯然你猜對了。根據前幾年的臨床研究，我們估計 ANCHOR 研究的流感疫苗接種率為 65%。考慮到我們招募的受試者患有中度至重度合併症、免疫功能低下，以及最近招募的 65 歲以上人群，這三個人群的疫苗接種率往往較高。顯然，這會對我們的研究產生影響。

We also noted in the Southern Hemisphere, however, this past flu season, that the vaccination rates have trended much lower than that 65% overall. And we also noted some of the vaccine manufacturers who have made similar observations. Because this is an ongoing study, we won't be sharing the vaccination rate because that can change over the course of the season up until the point where we complete enrollment.

然而，我們也注意到，在南半球，上個流感季的疫苗接種率遠低於整體 65%。我們也注意到一些疫苗生產商也提出了類似的觀點。由於這是一項正在進行的研究，我們不會公佈疫苗接種率，因為在完成招募之前，疫苗接種率可能會隨著季節的變化而變化。

But let me turn it over to Nicole to see if she has any additional comments on that.

不過，我還是把這個問題交給妮可，看看她還有沒有其他補充意見。

Thank you very much, Jeff. Yeah, I think you stated it very nicely that we had predicted a kind of a historical vaccination rate of around 60%. But really, our goal was to have vaccination be optional, which is in the trial and to really capture the real-world kind of incidence of vaccination.

非常感謝你，傑夫。是的，我認為你表達得很好，我們曾預測歷史疫苗接種率約為 60%。但實際上，我們的目標是讓疫苗接種成為一種選擇，這正是試驗的目的，並且要真正捕捉到現實世界中疫苗接種的發生率。

As we know this therapy will work well, particularly in participants who are unvaccinated, but we expect it to work well vaccinated as well. And so we will continue to follow this, and we are pleased with how enrollment is going so far.

我們知道這種療法效果會很好，尤其是對於未接種疫苗的參與者，但我們預計它對已接種疫苗的參與者也同樣有效。因此我們將繼續關注此事，我們對目前的招生情況感到滿意。

I also want to add that if we do end up seeing kind of a lower vaccination rate than expected, we -- this may end up actually kind of favoring the results of the trial, as you can imagine, because there may be a higher kind of event rate in the placebo arm based on capturing symptoms. So we are prepared for whatever comes, but it's an interesting time certainly to be doing an anti-influenza study.

我還想補充一點，如果我們最終看到的疫苗接種率低於預期，這實際上可能會有利於試驗結果，正如你所想，因為根據症狀捕捉，安慰劑組的事件發生率可能會更高。所以我們已經做好了應對任何情況的準備，但在這個時期進行抗流感研究確實很有意思。

And then, Seamus, to answer the second part of your question regarding manufacturing scale-up, I'll turn that question over to Shane Ward, our COO.

然後，Seamus，為了回答你關於生產規模擴大的第二部分問題，我將把這個問題交給我們的營運長Shane Ward。

Thanks, Jeff. I think we have talked quite a bit about the work we're doing with our partner, WuXi, who has worked with us on manufacturing since the initial stages of clinical development, has supported our clinical trials and will be the site of manufacturing for our BLA submission and planned commercialization.

謝謝你，傑夫。我認為我們已經就與合作夥伴藥明康德所進行的工作進行了許多討論。藥明康德從臨床開發的初期階段就與我們在生產方面展開合作，支持我們的臨床試驗，並將是我們生物製品許可申請和計劃商業化生產的生產基地。

At WuXi, the process characterization and PPQ activities necessary for BLA readiness are progressing well towards our target dates and that provides us more than adequate capacity for launching given our expectations of potential market demand given the population as we now understand it.

在藥明康德，生物製品許可申請 (BLA) 所需的製程表徵和製程性能驗證 (PPQ) 活動正朝著我們的目標日期穩步推進，鑑於我們目前對人口狀況的了解，這為我們提供了充足的產能，以應對潛在的市場需求。

The bigger question is how do we meet the potentially much greater demand during the life cycle of the product. And we have separate activities underway for further scale up. The first one of those is expanding to a parallel US supply chain. We've talked about our recent receipt of BARDA funding and the primary purpose of that funding is to support standing up a full US.

更大的問題是，我們如何在產品生命週期中滿足可能更大的需求。我們目前正在進行其他活動，以進一步擴大規模。第一項舉措是拓展到平行的美國供應鏈。我們已經談到了我們最近收到的 BARDA 資金，該資金的主要目的是支持建立一個完整的美國。

commercial supply chain for all nodes of manufacturing. And the tech transfer kickoffs have already begun for those, and we have a time frame that provides that additional capacity to come online shortly after our anticipated approval. And then the third piece is that we are looking at potential larger capacity global manufacturers who could come online a couple of years after that combined US, and WuXi supply chain to add the final bit of capacity that we would need for expansion into the largest possible population.

涵蓋所有製造環節的商業供應鏈。這些專案的技術轉移工作已經開始，我們制定了一個時間表，確保在預期獲得批准後不久，額外的產能就能投入使用。第三點是，我們正在尋找一些潛在的、產能較大的全球製造商，他們可能會在美國和藥明康德供應鏈合併後的幾年內上線，以增加我們向盡可能多人口地區擴張所需的最後產能。

Eric Schmidt, Cantor.

埃里克·施密特，坎托爾。

Another CMC question, if I could. What is the scale at WuXi currently that you hope to launch with? And I know you've said that CMC is rate limiting to a BLA filing. What exactly within CMC is the rate-limiting effect?

如果可以的話，我還想問一個CMC方面的問題。目前藥明康德希望以多大的規模開展業務？我知道您曾說過 CMC 對 BLA 申請有速率限制。CMC中究竟是什麼因素是速率限制步驟？

And then if I can squeeze another one in for Les on the data that you were presenting a couple of weeks back. Do we have any sense of resistance to CD388 and for those patients who weren't protected, why they weren't protected?

然後，如果我能再擠出一點時間，就你幾週前展示的數據，再給萊斯提個問題。我們是否對 CD388 有任何抵抗力？對於那些未受保護的患者，他們為什麼沒有受到保護？

Great questions, Eric. First part, obviously, we'll turn that over to Shane Ward. Shane?

埃里克，問得好。第一部分，顯然，我們將交給肖恩·沃德來解答。肖恩？

Right. Thank you. Yeah, the capacity at WuXi based on our initial scale that we are validating will provide for around production of 5 million doses per year at the 450-milligram dose level. There's some variability to that, and we may be able to scale up further. WuXi certainly has further capacity, but that is the expectation in terms of the initial supply chain, which then we will add the US supply chain to shortly thereafter.

正確的。謝謝。是的，根據我們正在驗證的初始規模，藥明康德的產能每年可生產約 500 萬劑 450 毫克劑量的疫苗。這其中存在一些變數，我們或許可以進一步擴大規模。藥明康德當然還有更大的產能，但這只是對初期供應鏈的預期，之後我們很快就會將美國供應鏈也納入其中。

In terms of your question about CMC being rate limiting, that's really all of the qualification requirements as we move forward to BLA readiness. So as was referenced earlier, we are transitioning from the clinical trial formulation or clinical trial configuration, which is three injections using prefilled syringes to a commercial configuration that will be a single vial containing the full dose.

關於您提出的 CMC 是否具有速率限制的問題，這實際上是我們推進 BLA 準備工作的所有資格要求。如前面所提到的，我們正在從臨床試驗配方或臨床試驗配置（即使用預填充注射器進行三次注射）過渡到商業配置（即包含全部劑量的單一小瓶）。

So we are transitioning to the vial configuration, scaling up, doing full process characterization and then final qualification. And those steps are really why it's rate limiting and why we are gating in terms of the company's timing for filing the BLA. But we have an aggressive timeline for all of those pieces, and WuXi has worked closely with us in developing an integrated plan for each of the nodes to be qualified on an accelerated schedule, which gives us the ability for that 2027 filing time.

因此，我們正在過渡到小瓶配置，擴大規模，進行完整的製程表徵，然後進行最終驗證。正是這些步驟導致了速度限制，也正是這些步驟導致我們對公司提交生物製品許可申請的時間有所限制。但我們為所有這些環節製定了積極的時間表，藥明康德與我們密切合作，制定了一項綜合計劃，以加快每個節點的認證進度，這使我們能夠在 2027 年提交申請。

And Eric, could you repeat the second part of your question for Les?

艾瑞克，你能把你的問題的第二部分再重複一遍給萊斯聽嗎？

Yeah, that was just on anything we may have learned about resistance or why the product wasn't fully protected for those patients who did get breakthrough infections.

是的，那隻是關於我們可能了解到的有關抗藥性或為什麼該產品不能完全保護那些發生突破性感染的患者的任何資訊。

Yeah, go ahead, Les.

好的，請便，萊斯。

Hi Eric, that's a great question. So at IDWeek, we presented our updated Phase IIb data, which included breakdowns of the timing of influenza cases in all the trial arms across the parallel at the European working group and Valencia, we presented comprehensive PK/PD and exposure response modeling analyses.

嗨，艾瑞克，這是一個很好的問題。因此，在 IDWeek 會議上，我們展示了更新的 IIb 期數據，其中包括歐洲工作組和瓦倫西亞會議上所有試驗組中流感病例時間的細分數據，我們展示了全面的 PK/PD 和暴露反應建模分析。

And I think the PK/PD and exposure response modeling analyses get to the heart of your question that there isn't a red line, a concentration threshold where you're going to get 100% protection with a neuraminidase inhibitor.

我認為 PK/PD 和暴露反應模型分析能夠解答你的問題，那就是神經氨酸酶抑制劑並沒有一條紅線，一個濃度閾值，超過這個閾值就能獲得 100% 的保護。

There will be breakthrough infections if there's potentially a high inoculum, it's called an inoculum effect where you could see infections. And so the fact that we saw infections doesn't mean that there was resistance. So we haven't seen evidence non-clinically, that this should be a molecule that's susceptible to resistance, but we are conducting the virology next-generation sequencing on the infections that were observed in the placebo and in the treatment arms, and we will report that data in due course once we have the analyses completed.

如果接種量可能很高，就會出現突破性感染，這稱為接種效應，在這種情況下可能會出現感染。因此，我們發現感染病例並不意味著存在抵抗力。因此，我們尚未在非臨床方面看到證據表明該分子容易產生抗藥性，但我們正在對安慰劑組和治療組中觀察到的感染進行病毒學下一代定序，一旦分析完成，我們將在適當的時候報告這些數據。

Brian Abrahams, RBC Capital Markets.

Brian Abrahams，加拿大皇家銀行資本市場。

Hi, good evening, thanks for taking my question and, congrats on all the progress. I was wondering if you could talk a little bit more about how the inclusion of healthy individuals over 65 impacts your assumptions for vaccine rates, event rates and powering?

您好，晚上好，感謝您回答我的問題，也祝賀您的所有進展。我想請您再詳細談談，將 65 歲以上的健康個體納入研究範圍，會對您關於疫苗接種率、事件發生率和統計功效的假設產生哪些影響？

And then secondarily, also trying to understand the implications of a potential December enrollment completion. I guess I'm curious what was the median time on study during flu season, that your event rate projections had assumed and whether December completion potentiates average accrual of either greater or fewer potential influenza events than you had maybe originally assumed in your powering. Thanks.

其次，也要了解 12 月入學人數可能達到高峰的影響。我想問的是，在流感季節，你們的事件發生率預測假設的平均研究時間是多少？ 12 月的研究完成是否會導致潛在的流感事件平均累積量比你們最初在計算功率時假設的更多或更少？謝謝。

Great Brian. Nicole, do you want to, take those questions?

偉大的布萊恩。妮可，你願意回答這些問題嗎？

Absolutely. Hi Brian, thanks for the questions. For your first question, so the addition of 65+ is really kind of a fascinating addition to the trial because 65+ is a broad group, as so it's 65 healthy and it's 65 mild to moderate, kind of a mild comorbidities, so to speak. In fact, we think in a lot of ways that it helps the study.

絕對地。嗨，布萊恩，謝謝你的提問。對於你的第一個問題，將 65 歲以上人群納入試驗確實是一個非常有趣的補充，因為 65 歲以上人群是一個廣泛的群體，既包括 65 歲健康的人，也包括 65 歲患有輕度至中度合併症的人。事實上，我們認為它在許多方面都有助於這項研究。

So this is a much easier population to enroll as you can imagine and we, it would probably expedite enrollment which it seems to have done so far for us as well. In terms of kind of background attack rate in this population, this is a population that tends to see more, flu events in trials.

因此，正如你所想，這部分人群更容易招募，而且這可能會加快招募速度，到目前為止，它似乎也確實為我們做到了這一點。就該族群的背景感染率而言，該族群在試驗中往往會發生更多流感事件。

And I think that's because there isn't as much protection and cocooning, these are our own relatives and people who are going out and seeing their grandchildren and out and about and not protecting themselves as much as a traditional kind of high-risk or immunocompromised participant might.

我認為這是因為防護措施和隔離措施不夠完善，這些人都是我們的親戚，他們會外出探望孫輩，四處走動，並沒有像傳統的高風險或免疫功能低下的參與者那樣做好自我保護。

And so when we did all the math and put these populations together, we realized that our placebo attack rate didn't really change very much. It was still right around 1.5%. I think we expect this 65+ population to have a higher background vaccination rate, about, 65% or more. So far we're still monitoring that, but in general, I think it helped us to have this population on the trial and did not affect our sample size or our power assumptions in any negative way.

因此，當我們進行所有計算並將這些人群放在一起時，我們發現安慰劑攻擊率實際上並沒有太大變化。仍在1.5%左右。我認為我們預計 65 歲以上人群的背景疫苗接種率會更高，大約在 65% 或以上。目前我們仍在監測，但總的來說，我認為讓這部分人群參與試驗對我們有所幫助，並且沒有對我們的樣本量或功效假設產生任何負面影響。

Is your second question for the December analysis. So what we mean by that is we will enroll everybody by December. Of course, the end point of the trial is still a six-month follow-up, so it's the 24 weeks influenza rate, or event rate, and so all those participants would still be followed for to the flu season if that helps. So it'd be similar to our phase 2B study where we looked at that at the end of flu season and had results in June.

你的第二個問題是針對十二月分析的。所以我們的意思是，我們將在12月之前完成所有學生的入學手續。當然，試驗的終點仍然是六個月的隨訪，也就是 24 週的流感發病率或事件發生率，因此，如果這有幫助的話，所有這些參與者仍將被隨訪到流感季節。所以這和我們之前的 2B 期研究類似，我們在流感季末進行了這項研究，並在 6 月得到了結果。

And I would add to Nicole's remarks that so far we have not seen any early signs of influenza in North America. That's important. We want to fully enroll this study before we see the first peak or a peak of influenza this this year.

我還要補充妮可的觀點，到目前為止，我們還沒有在北美看到任何流感的早期跡象。這很重要。我們希望在今年流感疫情的第一個高峰到來之前，完成這項研究的全部招募工作。

So far it is resembling last year when we're enrolling the 5,000-participant phase to the Navigate study where we fully enrolled it about a week and a half, two weeks before the first peak of the flu season. So far, fingers crossed we're tracking to a similar timeline here.

到目前為止，情況與去年類似，當時我們正在招募 5000 名參與者加入 Navigate 研究階段，我們在流感季第一個高峰期前大約一周半到兩週完成了全部招募工作。目前來看，但願我們這邊的進度也能跟上。

Joseph Stringer, Needham & Company.

Joseph Stringer，Needham & Company。

Hi, thanks so much for taking our question. Just wondering if you could give us a sense for how you're thinking about potential real-world uptake or penetration of CD 388 within the individual patient segments. I think at a previous R&D Day you had some initial survey work that you've done on this, but just given the potential for, subsequent, developments here for a larger addressable patient population.

您好，非常感謝您回答我們的問題。我想知道您能否談談您如何看待 CD 388 在各個患者群體中的潛在實際應用或滲透情況。我認為在先前的研發日上，你們已經就此進行了一些初步的調查工作，但考慮到後續發展可能惠及更廣泛的患者群體。

How should we think about that, maybe as a representative example, what would be the realistic uptake in, say, a severe immunocompromised patient versus, an otherwise healthy 50-year-old with a mild comorbidity. Yeah.

我們該如何看待這個問題呢？舉個例子，比如說，對於一個免疫功能嚴重受損的患者，實際的接受度會是多少？而對於一個身體健康、有輕微合併症的 50 歲患者，實際的接受度又會是多少？是的。

Yeah, these are important questions, Joey, and we'll be addressing those in detail at our R&D Day event on December 15. But let me turn it over to Jim Vital, to address those to the extent we can now. Jim.

是的，這些都是重要的問題，喬伊，我們將在 12 月 15 日的研發日上詳細討論這些問題。但現在讓我把麥克風交給吉姆‧維塔爾，讓他盡可能回答這些問題。吉姆。

Yeah sure, Jeff, thanks for the question. Certainly an important one. The scope of the approved label is expected to be quite broad and so certainly commercial reach will factor into the adoption and sort of how fast we get to peak sales over time.

當然可以，傑夫，謝謝你的提問。當然，這很重要。核准標籤的適用範圍預計會相當廣泛，因此商業覆蓋率肯定會影響其普及程度，以及我們隨著時間的推移達到銷售高峰的速度。

But I also want to point you to some interesting data in our current corporate deck. I think it's slide 20, where we described the data from our primary market research showing that prescribers have interest in this product in terms of their perception of the burden of flu and the importance of preventing it in subjects with moderate to severe comorbidities, but also these mild forms of comorbidities.

但我還想向您指出我們目前公司宣傳資料中的一些有趣數據。我認為是第 20 張幻燈片，其中我們描述了來自我們主要市場調查的數據，表明處方醫生對該產品感興趣，因為他們認為流感負擔沉重，並且對於患有中度至重度合併症的患者以及患有輕度合併症的患者來說，預防流感非常重要。

And so there's a very broad market opportunity here with strong physician interests across the scope of the populations included in the phase 3 study.

因此，這裡存在著非常廣泛的市場機會，在 3 期研究涵蓋的人群中，醫生們對此表現出了濃厚的興趣。

Sarah Nik, H.C. Wainwright.

Sarah Nik，H.C. Wainwright。

Hi, everyone, and thanks for taking the question. And again, congrats on all the progress. My question is more actually looking, maybe for opportunities beyond even the seasonal flu, as you've highlighted previously, CD 388's universal activity. Against all flu strains including H5N1.

大家好，感謝大家回答這個問題。再次恭喜你們取得的所有進展。我的問題其實是想尋找一些機會，甚至是季節性流感以外的機會，正如你之前強調的，CD 388 具有普遍的活性。可對抗包括H5N1在內的所有流感病毒株。

So, just wondering beyond the BA funding at this point, what specific clinical or pre-clinical work is planned to formally establish its utility in a pandemic setting, and do you think this could create a separate regulatory pathway or government, stockpiling opportunity distinct, from the seasonal flu. Thank you.

所以，除了目前的BA資助之外，還有哪些具體的臨床或臨床前工作計畫來正式確定其在大流行環境下的效用？您認為這是否會創造一條與季節性流感不同的監管途徑或政府儲備機會？謝謝。

Yeah, thanks, Sarah. Les, do you want to take that question about, any other studies we might be performing to evaluate CD 388 in pandemic.

謝謝你，莎拉。萊斯，你想回答一下關於我們可能正在進行的其他研究來評估 CD 388 在大流行中的作用的問題嗎？

Yeah, so we're working closely with Richard Webby's lab at Saint Jude's Hospital, and, thus far we, at a meeting earlier this summer we presented data against the pandemic H5N1 strain in ferrets where we demonstrated robust efficacy with H5N1 at exposures that are consistent with the exposures at the high dose in human subjects.

是的，我們正在與聖裘德醫院的理查德·韋比實驗室密切合作，到目前為止，在今年夏天早些時候的一次會議上，我們展示了針對雪貂身上大流行的 H5N1 毒株的數據，證明在與人類受試者高劑量暴露一致的暴露水平下，H5N1 具有強大的有效性。

We're going to continue work with them on, other pandemic strains, as well as mutant, as, mutant strains that are resistant to neuroiniddase inhibitors. Thus far all of the strains that we've tested that have resistance against neuroiniddase inhibitors, we retain activity against those without a shift. So that we're going to do in vitro studies that will follow-up on the ferret efficacy study that we ran with Wey's lab at Saint Jude.

我們將繼續與他們合作，研究其他流行病毒株以及對神經氨酸酶抑制劑具有抗性的突變病毒株。到目前為止，我們測試過的所有對神經氨酸酶抑制劑有抗性的菌株，對那些沒有發生轉變的菌株仍然保持活性。因此，我們將進行體外研究，以跟進我們與聖裘德兒童研究醫院 Wey 實驗室進行的雪貂療效研究。

This concludes our question-and-answer session. I would like to turn the conference back over to Doctor Jeff Stein for any closing remarks.

我們的問答環節到此結束。我謹將會議交還給傑夫‧斯坦博士，請他作總結發言。

Well, thank you all for joining us today. We greatly appreciate your interest in Cidara, and hope that you enjoy your evening. Thank you.

謝謝各位今天蒞臨。我們非常感謝您對 Cidara 的關注，並祝您晚安。謝謝。

The conference has now concluded. Thank you for attending today's presentation. You may now disconnect.

會議已經結束。感謝各位參加今天的報告會。您現在可以斷開連線了。