Inovio Pharmaceuticals Inc (INO) 2026 Q1 法說會逐字稿

Good afternoon, ladies and gentlemen, and welcome to the Inovio first-quarter 2026 financial results conference call. (Operator Instructions) This call is being recorded on Wednesday, May 13, 2026.

各位女士、各位先生，下午好，歡迎參加 Inovio 2026 年第一季財務業績電話會議。（接線員指示）本次會議於 2026 年 5 月 13 日（星期三）錄音。

I would now like to turn the conference over to Jennie Willson. Please go ahead.

現在我想把會議交給 Jennie Willson。請開始。

Good afternoon, and thank you for joining the Inovio first-quarter 2026 financial results conference call. Joining me today on today's call are Dr. Jacqui Shea, President and Chief Executive Officer; Dr. Mike Sumner, Chief Medical Officer; Steve Egge, Chief Commercial Officer; and Peter Kies, Chief Financial Officer.

各位下午好，感謝您參加 Inovio 2026 年第一季財務業績電話會議。今天與我一同出席的有：總裁兼執行長 Jacqui Shea 博士；首席醫療長 Mike Sumner 博士；首席商務長 Steve Egge；以及財務長 Peter Kies。

Today's call will review our corporate and financial information for the quarter ended March 31, 2026, as well as provide a general business update. Following prepared remarks, we will conduct a question-and-answer segment.

今天的會議將回顧截至 2026 年 3 月 31 日之季度的公司與財務資訊，並提供一般業務最新進展。在預先準備的發言之後，我們將進行問答環節。

During the call, we will be making forward-looking statements regarding future events and the future performance of the company. These statements relate to our business plans to develop Inovio's DNA medicines platform, including the FDA's ongoing review of our BLA for INO-3107, including the October 30, 2026, PDUFA target date and our yet-to-be scheduled meeting with the FDA to discuss eligibility for the accelerated approval program, our belief that INO-3107 fulfills the criteria for accelerated approval; the potential benefits of INO-3107, including our belief that it has a positively differentiated product profile and the potential to become the preferred product by patients and their physicians, if approved; the anticipated commercial launch of INO-3107, if approved, and our engagement of commercial partners in preparation for a potential launch; our collaboration with Akeso Inc., to evaluate INO-5412 in combination with a novel dual checkpoint inhibitor for the potential treatment of GBM, the advancement of our DPROT technology platform, capital resources, including our estimated operational net cash burn of approximately $18 million for the second quarter of 2026 and the expected sufficiency of our cash resources into first quarter of 2027 and our expectations regarding competition, market size and acceptance of INO-3107 if approved.

在本次會議中，我們將就未來事件及公司未來表現作出前瞻性陳述。這些陳述涉及我們推進 Inovio DNA 藥物平台的業務計畫，包括 FDA 對 INO-3107 生物製劑許可申請（BLA）的持續審查，包括 2026 年 10 月 30 日的 PDUFA 目標日期，以及我們尚未排定、將與 FDA 討論是否符合加速核准計畫資格的會議；我們相信 INO-3107 符合加速核准的標準；INO-3107 的潛在效益，包括我們相信其具備正向差異化的產品特性，且若獲核准，可能成為病患及其醫師偏好的產品；若獲核准，INO-3107 預期的商業上市，以及我們為潛在上市所進行的商業合作夥伴洽談與準備；我們與 Akeso Inc. 的合作，以評估 INO-5412 與一種新型雙重免疫檢查點抑制劑合併用於膠質母細胞瘤（GBM）潛在治療；我們 DPROT 技術平台的推進；資本資源，包括我們估計 2026 年第二季營運淨現金消耗約 1,800 萬美元、以及我們預期現金資源可支應至 2027 年第一季；以及我們對於若 INO-3107 獲核准之競爭、市場規模與市場接受度的預期。

All of these statements are based on the beliefs and expectations of management as of today. Actual events or results could differ materially. We refer you to the documents we file from time to time with the SEC, which under the heading Risk Factors identify important factors that could cause actual results to differ materially from those expressed by the company verbally as well as statements made within this afternoon's press release.

上述所有陳述均基於管理層截至今日的信念與預期。實際事件或結果可能存在重大差異。我們建議您參閱我們不時向美國證券交易委員會（SEC）提交的文件，其中在「風險因素」標題下列示可能導致實際結果與公司口頭陳述以及今日下午新聞稿中所載陳述存在重大差異的重要因素。

This call is being webcast live, and a link can be found on our website, ir.inovio.com, and a replay will be made available shortly after this call is concluded.

本次會議將進行線上即時網路直播，連結可於我們網站 ir.inovio.com 找到，並將於會議結束後不久提供重播。

I will now turn the call over to Inovio's President and CEO, Dr. Jacqui Shea.

現在我將把電話交給 Inovio 總裁兼執行長 Jacqui Shea 博士。

Good afternoon, and thank you to everyone for joining today's call. These are very busy times at Inovio as we remain focused on achieving our top priority, advancing our lead candidate, INO-3107 through the regulatory process and toward its October 30 target PDUFA date.

各位下午好，感謝大家參加今天的會議。對 Inovio 而言，這段時間非常忙碌，我們仍專注於達成最優先事項：推進我們的主力候選產品 INO-3107 完成法規審查流程，並朝 10 月 30 日的 PDUFA 目標日期邁進。

Our goal is to ensure that every patient with recurrent respiratory papillomatosis or RRP has access to a therapeutic option that can work for them to reduce the need for surgery. If approved, we believe that our innovative therapy has the potential to become the preferred product for patients suffering from RRP, a rare and debilitating disease of the respiratory tract with a critically high unmet need for effective nonsurgical treatment options. The BLA for 3107 has been in active review since December when the FDA accepted the file for review under the accelerated approval program.

我們的目標是確保每位復發性呼吸道乳頭狀瘤病（RRP）患者都能取得一種適合他們、可降低手術需求的治療選項。若獲核准，我們相信這項創新療法有潛力成為罹患 RRP 患者的首選產品；RRP 是一種罕見且致殘的呼吸道疾病，對有效的非手術治療選項存在極高且迫切的未被滿足醫療需求。3107 的 BLA 自 12 月 FDA 以加速核准計畫受理並進入審查以來，一直處於積極審查中。

While Mike will provide a more in-depth regulatory update, I'd like to comment on a couple of key highlights. The FDA recently completed their standard mid-cycle review with no new significant issues being raised and scheduled the late cycle review for the third quarter. As you will recall from our last quarterly update, the FDA had previously agreed to an informal meeting to discuss the potential review issue they noted in their file acceptance letter regarding eligibility for review under the accelerated approval program.

Mike 將提供更深入的法規更新，但我想先談幾個重點。FDA 近期完成其標準的期中審查（mid-cycle review），未提出新的重大問題，並已將後期審查（late cycle review）排定於第三季進行。如您從我們上次季度更新所記得，FDA 先前已同意召開一場非正式會議，以討論其在受理函中提及、關於是否符合加速核准計畫審查資格的潛在審查議題。

As a part of communications about the mid-cycle review, they reiterated their intent to schedule that meeting, and we look forward to the discussion. While we made progress with 3107 on the regulatory front, we've been advancing our commercial readiness plans, including continuing to gather key strategic insights from the RRP community. And while the majority of our resources are focused on 3107, we're continuing to leverage the power of partnerships to advance other promising candidates in our pipeline, including an exciting opportunity to work with Akeso and the Dana-Farber Cancer Institute to build on our previous immuno-oncology work in glioblastoma or GBM.

作為期中審查溝通的一部分，FDA 重申其安排該會議的意向，我們期待進一步討論。在 3107 的法規面向取得進展的同時，我們也在推進商業化就緒計畫，包括持續從 RRP 社群蒐集關鍵策略洞見。此外，雖然我們大部分資源聚焦於 3107，我們仍持續運用合作夥伴關係的力量推進產品線中其他具潛力的候選產品，包括與 Akeso 及 Dana-Farber 癌症研究所合作、在我們先前於膠質母細胞瘤（GBM）免疫腫瘤學工作的基礎上拓展的一項令人振奮的機會。

We're also advancing our innovative next-generation candidates and I'm pleased to have recently presented promising preclinical data on our DNA-encoded protein or DPROT technology work targeting Factor VIII production in hemophilia A and announced 2 new rare disease targets in Fabry disease and hypophosphatasia for the platform. We are laser-focused on these strategic priorities and excited about what's ahead for Inovio as we work to deliver on the promise of DNA medicine for patients.

我們也在推進創新的下一代候選產品。我很高興近期已發表我們 DNA 編碼蛋白（DPROT）技術在血友病 A 之第八凝血因子（Factor VIII）產生方面的具前景臨床前數據，並宣布該平台新增兩個罕見疾病標的：法布瑞氏症（Fabry disease）與低磷酸酶症（hypophosphatasia）。我們將高度聚焦於這些策略優先事項，並對 Inovio 的未來感到振奮，因為我們致力於為患者實現 DNA 藥物的承諾。

I'll now turn it over to Mike for some additional details on our regulatory progress with 3107. Mike?

接下來我把時間交給 Mike，請他補充 3107 法規進展的更多細節。Mike？

Thanks, Jacqui. As Jacqui noted, since our BLA for INO-3107 was accepted for review under the accelerated approval program in December of 2025, the FDA has been actively reviewing our submission. We have been responding to routine requests for information and meeting regular top milestones in the review process, including the FDA completing its mid-cycle review of our BLA, where no new significant issues were raised.

謝謝你，Jacqui。如 Jacqui 所提，自 2025 年 12 月我們的 INO-3107 BLA 以加速核准計畫獲 FDA 受理審查以來，FDA 一直在積極審查我們的申請資料。我們持續回覆例行的資訊要求，並按期達成審查流程中的重要里程碑，包括 FDA 完成對我們 BLA 的期中審查，且未提出新的重大問題。

At the time of the mid-cycle review, the FDA indicated that it is continuing to review the assessment aid we submitted in February, which outlined our rationale for accelerated approval program eligibility. They also reiterated their intent to schedule the previously agreed to informal meeting to discuss this potential review issue, which they noted in their file acceptance letter. In addition, we reported last quarter that we had submitted an updated protocol for our confirmatory trial to the IND, which is required under the accelerated approval program.

在期中審查時，FDA 表示其仍在審查我們於 2 月提交的評估輔助文件（assessment aid），該文件闡述我們對於符合加速核准計畫資格的理由。他們也重申其安排先前已同意之非正式會議的意向，以討論其在受理函中提及的這項潛在審查議題。此外，我們在上季也報告已向 IND 提交確認性試驗的更新方案，這是加速核准計畫所要求的。

We are waiting for feedback from the agency on both the informal meeting and the confirmatory trial protocol so we can finalize the study design. We look forward to having the opportunity to discuss these issues further with the FDA and to emphasize why we believe that 3107 fulfills the criteria for accelerated approval by meeting a significant unmet need and providing a meaningful therapeutic benefit over existing treatments.

我們正等待主管機關就非正式會議與確認性試驗方案提供回饋，以便我們最終定案研究設計。我們期待有機會與 FDA 進一步討論這些議題，並強調我們為何相信 3107 透過滿足重大未被滿足需求、且相較既有治療提供具意義的治療效益，而符合加速核准的標準。

I'd like to take a moment now to elaborate on that rationale. Based on published FDA guidance, our eligibility depends on the ability of 3107 to provide a meaningful therapeutic benefit over existing treatments and the ability to meet a remaining critical unmet need among patients. We believe that 3107 meets both of these criteria based on 3 factors: First, effectiveness as demonstrated in our Phase I/II trial, where the vast majority of patients experienced a 50% to 100% reduction in surgery in year 1 and with continued clinical improvement in year 2.

我現在想花一點時間進一步闡述該理由。根據已發布的 FDA 指引，我們的資格取決於 3107 是否能相較既有治療提供具意義的治療效益，以及是否能滿足患者仍存在的關鍵未被滿足需求。我們相信 3107 基於三項因素符合這兩項標準：第一，療效已在我們的第一/二期試驗中展現，絕大多數患者在第 1 年手術次數減少 50% 至 100%，且在第 2 年持續出現臨床改善。

Second, an improved safety profile that does not include required surgery to maintain minimal residual disease during the dosing window. And third, a differentiated mechanism of action that does not come with the risk of reduced clinical effectiveness due to known immune factors that impact the efficacy of the approved product, including preexisting neutralizing antibodies or an immunosuppressive tumor microenvironment, thus providing an important opportunity to treat patients who are not served by existing therapy. It is important to emphasize again that at the heart of our belief in 3107's eligibility for accelerated approval are the patients, patients who face the risk of permanent damage to the vocal cords and significant social, emotional and financial costs every time they require surgery to manage their disease. Every surgery matters to patients, and we believe that every patient should have access to a treatment option that works for them to reduce the need for surgery.

第二，改善的安全性特徵，在給藥期間不包含為維持最低殘餘疾病狀態而必須進行的手術。第三，具差異化的作用機轉，不會因已知會影響已核准產品療效的免疫因素（包括既存的中和抗體或免疫抑制性的腫瘤微環境）而帶來臨床有效性降低的風險，因此提供一個重要機會來治療現有療法未能涵蓋的患者。再次強調，我們相信 3107 具備加速核准資格的核心在於患者——這些患者每次需要手術來控制疾病時，都面臨聲帶永久損傷的風險，以及重大的社會、情緒與財務成本。每一次手術對患者都很重要，我們相信每位患者都應該能獲得一個適合他們、可降低手術需求的治療選項。

A compelling example of the remaining unmet need for therapeutic options for RRP patients is the fact that the recently approved gorilla adenoviral-based immunotherapy does not work for a number of patients according to that product's published data. Further, the treatment regimen for this product requires surgery prior to the third and fourth doses if visible papillomas are present to maintain a state of minimal residual disease. This means that nonresponders or the patients for whom this product didn't work, had 2 surgeries during the treatment window and then saw no clinical benefit or improvement in the following year according to the published clinical trial data. That's why additional treatment options are so critical for the RRP community. There will continue to be patients not served by existing therapy.

RRP 患者在治療選項上仍存在未被滿足需求的一個有力例證是：根據該產品已發表的數據，近期核准的 Gorilla 腺病毒載體免疫療法對部分患者無效。此外，該產品的治療方案要求在第三與第四劑之前，若仍可見乳頭狀瘤，需先進行手術以維持最低殘餘疾病狀態。這意味著，對於無反應者或該產品對其無效的患者，在治療期間接受了 2 次手術，但根據已發表的臨床試驗數據，在接下來一年仍未見任何臨床獲益或改善。這就是為什麼對 RRP 社群而言，額外的治療選項如此關鍵。仍將持續有患者無法從現有療法中受益。

This need was highlighted in a recently published RRP Foundation-sponsored position statement, where 16 leading RRP physicians outlined a contemporary evidence-based approach to the management of RRP in adults. They highlighted the benefits of HPV-specific immunotherapy to address the underlying disease that causes RRP and recommended HPV-specific immunotherapy as the preferred first-line therapy for adults, including the recently approved immunotherapy and 3107 should it be approved.

這項需求在近期發表、由 RRP Foundation 贊助的立場聲明中被凸顯；16 位 RRP 領域的權威醫師概述了以當代證據為基礎的成人 RRP 管理方法。他們強調 HPV 特異性免疫療法可針對導致 RRP 的根本疾病，並建議將 HPV 特異性免疫療法作為成人的首選一線治療，包括近期核准的免疫療法，以及若獲核准的 3107。

These are some of the key discussion points we've provided to the FDA, and we look forward to having the opportunity to discuss them further. In the meantime, we will continue working collaboratively with the agency to advance our BLA through the regulatory process.

以上是我們提供給 FDA 的一些關鍵討論要點，我們期待有機會進一步討論。同時，我們將持續與主管機關協作，推進我們的 BLA 依循法規流程向前。

I'll now turn it over to Steve for a commercial update. Steve?

接下來我把時間交給 Steve，進行商業面更新。Steve？

Thanks, Mike. On the commercial front, we are continuing to advance our commercial readiness plans in anticipation of a potential U.S. approval in 2026, and we're planning to manage commercialization ourselves with the support of a contract sales organization. We've completed targeting, segmentation and product positioning work that supports a positively differentiated product profile. We've also engaged or identified key commercial partners, including a third-party logistics provider, specialty distributor, specialty pharmacy, patient hub and agency of record.

謝謝，Mike。在商業方面，我們正持續推進商業化準備計畫，以因應 2026 年在美國可能獲得核准；我們規劃在合約銷售組織的支援下自行負責商業化。我們已完成目標客群鎖定、分眾與產品定位工作，以支持具正向差異化的產品特徵。我們也已接洽或確認關鍵商業合作夥伴，包括第三方物流供應商、專科經銷商、專科藥局、病患支援中心（patient hub）以及主責代理商（agency of record）。

In recent months, we've also been watching and incorporating key learnings from the launch of our competitors' recently approved product, building where they seem to be having success or challenges and optimizing our own commercial plans accordingly. Importantly, there are key differences between the Gorilla adenoviral-based product and 3107. And some of the challenges our competitor faced will not impact our launch, including the fact that we don't require an ultra-cold chain and we don't require surgery to maintain minimum residual disease during the dosing window.

近幾個月來，我們也持續觀察並納入競品近期核准產品上市的關鍵經驗，針對其看似成功或面臨挑戰之處加以借鏡，並相應優化我們自身的商業計畫。重要的是，Gorilla 腺病毒載體產品與 3107 之間存在關鍵差異。而競品所面臨的一些挑戰不會影響我們的上市，包括我們不需要超低溫冷鏈，也不需要在給藥期間為維持最低殘餘疾病狀態而進行手術。

We've also continued to gather important insights from the RRP community and recently attended the Combined Otolaryngology Spring Meeting or COSM, a key conference for laryngologists. In conversations at that conference with physicians specializing in the treatment of RRP, we heard repeatedly that there continues to be a high unmet need for effective immunotherapy options that work for each patient. Both patients and doctors are highly motivated and very receptive to new treatments, which indicates that this market has significant unmet need and commercial opportunity, particularly for a potential product like 3107 that has a positively differentiated product profile across efficacy, tolerability and simplicity of the treatment regimen.

我們也持續從 RRP 社群蒐集重要洞見，並於近期參加了 Combined Otolaryngology Spring Meeting（COSM），這是喉科醫師的重要會議。在該會議與專精 RRP 治療的醫師交流時，我們反覆聽到：仍然高度缺乏能對每位患者都有效的免疫療法選項。患者與醫師都高度投入且非常願意接受新療法，顯示此市場存在顯著未被滿足的需求與商業機會，尤其是像 3107 這樣在療效、耐受性與治療方案簡便性方面具正向差異化產品特徵的潛在產品。

I'll now turn it back to Jacqui for a pipeline update. Jacqui?

接下來我把時間交回給 Jacqui，進行產品線更新。Jacqui？

Thanks, Steve. With our resources focused primarily on 3107, partnerships will continue to be a key part of our strategy to advance other promising candidates in our pipeline. In March this year, we announced an innovative collaboration with Akeso to evaluate INO-5412 in combination with their novel dual checkpoint inhibitor as a potential treatment for glioblastoma, the most common and aggressive form of brain cancer. The study, which builds on our previous promising research in GBM will be part of a Phase II adaptive platform trial sponsored by the Dana-Farber Cancer Institute. We've also continued to advance our exciting next-generation DNA medicine platform and shared research on our DNA-encoded protein or DPROT technology targeting Factor VIII at several key conferences, including the World Federation of Hemophilia Global Conference and the American Society of Gene and Cell Therapy Annual Meeting.

謝謝，Steve。在我們的資源主要聚焦於 3107 的同時，合作夥伴關係仍將是我們推進產品線中其他具潛力候選項目的關鍵策略。今年 3 月，我們宣布與 Akeso 展開創新合作，評估 INO-5412 與其新型雙重檢查點抑制劑聯合使用，作為治療膠質母細胞瘤（glioblastoma）的潛在方案；膠質母細胞瘤是最常見且最具侵襲性的腦癌類型。該研究建立在我們先前於 GBM 的正向研究基礎上，將納入由 Dana-Farber Cancer Institute 贊助的第二期自適應平台試驗。我們也持續推進令人振奮的下一代 DNA 藥物平台，並在多個重要會議上分享我們針對第八凝血因子（Factor VIII）的 DNA 編碼蛋白（DPROT）技術研究，包括世界血友病聯盟全球大會以及美國基因與細胞治療學會年會。

Building on the promising DNA-encoded monoclonal antibody research published in Nature Medicine last year, our DPROT technology aims to enable long-term protein expression within the body and address the shortcomings of conventional therapeutic protein or enzyme replacement therapies. Based on positive preclinical data on Factor VIII production for hemophilia A, Inovio is developing additional DPROT indications in the rare disease space, including Fabry disease and hypophosphatasia, and is in discussions with potential partners to accelerate development of this promising platform.

在去年發表於《Nature Medicine》的 DNA 編碼單株抗體研究之正向成果基礎上，我們的 DPROT 技術旨在使蛋白質能在體內長期表達，並解決傳統治療性蛋白或酵素替代療法的不足。基於針對血友病 A 的第八凝血因子產生之正向臨床前數據，Inovio 正在罕見疾病領域開發更多 DPROT 適應症，包括法布瑞氏症（Fabry disease）與低磷酸酶症（hypophosphatasia），並正與潛在合作夥伴洽談，以加速此一具前景平台的開發。

Now I'll turn it over to our CFO, Peter Kies for a financial update. Peter?

現在我把時間交給我們的財務長 Peter Kies，進行財務更新。Peter？

Thanks, Jacqui. Today, I'd like to provide an overview of Inovio's financial results for the first quarter of 2026. As Jacqui noted, our primary goal is to advance INO-3107 towards approval and we remain focused on optimizing resources to support that program.

謝謝，Jacqui。今天我想概述 Inovio 於 2026 年第一季的財務結果。如 Jacqui 所提，我們的首要目標是推進 INO-3107 走向核准，我們仍專注於最佳化資源配置以支持該計畫。

I am pleased to report that the company strengthened its balance sheet with an underwritten public equity offering in April 2026. Net proceeds from the offering after deducting underwriter discounts, commissions and offering expenses were approximately $16 million. We finished the first quarter of 2026 with $37.7 million in cash, cash equivalents and short-term investments compared to $58.5 million as of December 31, 2025.

我很高興報告，公司於 2026 年 4 月透過承銷公開發行普通股強化了資產負債表。扣除承銷折讓、佣金與發行費用後，本次發行的淨募資款約為 1,600 萬美元。我們在 2026 年第一季結束時的現金、約當現金及短期投資為 3,770 萬美元，相較於 2025 年 12 月 31 日的 5,850 萬美元。

With the addition of the April public offering, we have extended our estimated cash runway into the first quarter of 2027 beyond the target PDUFA date of INO-3107. This projection includes an operational net cash burn estimate of approximately $18 million for the second quarter of 2026. These cash runway projections do not include any further capital raise activities that we may undertake and are based on current projections and assumptions.

加上 4 月的公開發行後，我們將預估現金可支撐期間延長至 2027 年第一季，並超過 INO-3107 的目標 PDUFA 日期。此預估包含 2026 年第二季營運淨現金消耗約 1,800 萬美元的估計。上述現金可支撐期間的預估未包含我們可能進行的任何進一步募資活動，並係基於目前的預測與假設。

Turning to our results to the first quarter. Operating expenses dropped from $25.1 million in the first quarter of 2025 to $21.9 million in the first quarter of 2026, a 13% decrease. Inovio's net loss for the first quarter of 2026 was $19.7 million or $0.28 per share basic and dilutive compared to a net loss of $19.7 million or $0.51 per share basic and dilutive for the first quarter of 2025.

接著說明我們第一季的業績。營運費用由 2025 年第一季的 2,510 萬美元降至 2026 年第一季的 2,190 萬美元，下降 13%。Inovio 於 2026 年第一季的淨損為 1,970 萬美元，基本及稀釋後每股虧損 0.28 美元；相較之下，2025 年第一季淨損同為 1,970 萬美元，但基本及稀釋後每股虧損為 0.51 美元。

As a reminder, you can find our full financial statements in this afternoon's press release as well as in our quarterly report on Form 10-Q filed with the SEC. And with that, I'll turn it back over to Jacqui.

提醒一下，您可以在今天下午發布的新聞稿以及我們向美國證券交易委員會（SEC）提交的 Form 10-Q 季度報告中，查閱我們完整的財務報表。接下來我把時間交還給 Jacqui。

Thanks, Peter. I'd now like to pause to open up the call to answer any questions you might have. Operator?

謝謝你，Peter。我現在想先暫停一下，開放電話會議來回答各位可能有的問題。接線員？

(Operator Instructions) Your first question comes from Ted with Piper Sandler.

(接線員指示) 您的第一個問題來自 Piper Sandler 的 Ted。

And it sounds like things are going well with the review. I saw the PAPZIMEOS numbers of $21.6 million were pretty good. Any comments? Can you expand a little more about how you expect to launch and differentiate versus the currently marketed product?

聽起來審查進展得很順利。我看到 PAPZIMEOS 的 2,160 萬美元數字相當不錯。有什麼評論嗎？你們能否再多談一些，預期將如何上市，以及相較於目前已上市產品要如何做出差異化？

Ted, nice to hear from you. Steve, do you want to take that one?

Ted，很高興聽到你的聲音。Steve，你要回答這題嗎？

Sure. Ted, yes, so we saw the Precigen performance as well and kind of how we're thinking about the market and how we'll compete is we have, we think, a positively differentiated product profile when we look across efficacy, tolerability and the simplicity of the treatment regimen. So we think we can be well differentiated in the market. And then the overall market opportunity itself, there's a very significant number of patients 14,000 RRP patients is the most kind of recently published number, although that's quite dated.

當然。Ted，是的，我們也看到了 Precigen 的表現；至於我們如何看待市場以及如何競爭，我們認為在療效、耐受性以及治療方案的簡便性等面向綜合來看，我們的產品特性具有正向差異化。因此我們認為能在市場上形成明顯差異。另外就整體市場機會而言，患者數量非常可觀；最近一次發表的數字是 14,000 名 RRP 患者，雖然那個數據其實相當久了。

And our own estimate of claims data demonstrates that, that's probably a significant underestimate. I think Precigen has noted 27,000 RRP patients. So by the time we get to market, we expect Precigen or PAPZIMEOS would have had single-digit penetration into the market in the first year. So the vast majority of the opportunity will remain by the time we get to market. And we do expect to be a fast following second entrant, and there's many examples of second entrants fast following that do very, very well in the market and in some cases, take market leadership.

而我們自己對理賠（claims）資料的估算顯示，那很可能是嚴重低估。我想 Precigen 曾提到有 27,000 名 RRP 患者。因此等到我們進入市場時，我們預期 Precigen 或 PAPZIMEOS 在第一年對市場的滲透率會是個位數百分比。所以等我們上市時，絕大多數的市場機會仍然存在。我們也預期會成為快速跟進的第二個進入者；市場上有很多第二進入者快速跟進且表現非常、非常好的例子，甚至在某些情況下還能取得市場領導地位。

And if I can just add here, Steve. I think PAPZIMEOS launch also gives us the opportunity to learn from their successes and challenges, and we'll certainly be baking those learnings into our go-to-market plans as well. So I think the progress that we see Precigen making in the marketplace is really an indication of the high unmet need for these patients for therapeutic alternatives to surgery. And we're really excited by the opportunity for 3107.

如果我可以在這裡補充一下，Steve。我認為 PAPZIMEOS 的上市也讓我們有機會從他們的成功與挑戰中學習，我們也一定會把這些學到的經驗納入我們的上市（go-to-market）計畫。因此，我們看到 Precigen 在市場上的進展，其實也反映出這些患者對於可替代手術的治療選項存在高度未被滿足的需求。我們對 3107 的機會感到非常興奮。

Very great. And then if I may, just a quick follow-up. When it comes to the informal meeting with FDA, what are the primary issues that you intend to discuss there?

非常好。如果可以的話，我再追問一個簡短問題。關於與 FDA 的非正式會議，你們打算在那裡討論的主要議題是什麼？

Mike?

Mike？

Yes, happy to. So I mean this is really about confirming accelerated approval eligibility. I think it's -- I don't think we'll have much discussion around the clinical unmet need. That's very obvious, I think, to everybody concerned. So it's really around the discussion of meaningful therapeutic benefit.

好的，很樂意。所以這主要是要確認是否符合加速核准（accelerated approval）的資格。我想——我不認為我們會在臨床未被滿足需求上有太多討論。我認為對所有相關方來說那點非常明顯。因此重點會放在「具意義的治療效益」的討論上。

And as we've stated, we firmly believe we have a significant safety advantage without the requirement for those minimal residual disease surgeries and our differentiated mechanism of action will enable us to treat patients that the existing product won't be able to.

正如我們所說，我們堅信在不需要進行那些最小殘存病灶（minimal residual disease）手術的前提下，我們具有顯著的安全性優勢；而且我們差異化的作用機轉將使我們能治療現有產品無法治療的患者。

Great. That's really helpful. And I appreciate how you laid it out on the call earlier.

很好。這真的很有幫助。也謝謝你們先前在電話會議中把它講得很清楚。

Your next question comes from Jay with Oppenheimer.

下一個問題來自 Oppenheimer 的 Jay。

Can you just maybe talk about some of the key discussion points with the FDA when you meet on 3107 and remind us any additional data or evidence that you have submitted or will submit, and then I had a follow-up, if I could, please.

你們能否談談與 FDA 針對 3107 會面時的一些關鍵討論重點，並提醒我們你們已提交或將提交的任何額外數據或證據？然後如果可以的話，我還有一個追問。

Yes, sure. So nice to hear from you, Jay. So I'll start off, and then I'll ask Mike to chip in here. So if you remember, we submitted our assessment to FDA back in February. Ahead of the informal meeting, and that was in direct response to a request from the agency.

好的，當然。Jay，很高興聽到你的聲音。我先開始，然後請 Mike 也補充一下。如果你還記得，我們在 2 月向 FDA 提交了我們的評估資料。那是在非正式會議之前提交的，而且是直接回應主管機關的要求。

And that assessment aid doesn't include any new clinical data, but it did include additional analysis of the data that we performed. So at the upcoming meeting, as Mike indicated, what we principally expect to be discussing with the agency are the arguments that we've laid out in the BLA and also in the assessment aid as to why we believe 3107 is eligible for review under the accelerated approval pathway. And that's really around providing meaningful therapeutic benefit over the existing product and that we -- that 3107 has the potential to meet this unmet need.

那份評估輔助文件（assessment aid）不包含任何新的臨床數據，但包含了我們所做的額外數據分析。因此在即將到來的會議上，如同 Mike 所指出的，我們主要預期與主管機關討論的是：我們在 BLA 以及評估輔助文件中所提出的論點，說明為何我們相信 3107 符合加速核准途徑下的審查資格。而這主要圍繞在：相較於現有產品能提供具意義的治療效益，以及 3107 有潛力滿足這項未被滿足的需求。

Mike, do you want to go into some more specifics?

Mike，你要再更具體說明一些嗎？

I mean I certainly can. I mean, in terms of the differentiated mechanism of action, which should enable us to treat a different patient population than PAPZIMEOS. We've talked previously about the presence of neutralizing antibodies to the gorilla adenoviral platform that will decrease the immune response that those patients will see with PAPZIMEOS. And we've also talked about the requirement that they have for performing those minimal residual disease surgeries is based on the immunosuppressive papilloma microenvironment that they utilize those surgeries to overcome.

我當然可以。就差異化的作用機轉而言，這應該能讓我們治療與 PAPZIMEOS 不同的患者族群。我們先前談過，針對大猩猩腺病毒載體平台（gorilla adenoviral platform）存在的中和抗體，會降低這些患者在使用 PAPZIMEOS 時所能產生的免疫反應。我們也談過，他們之所以需要進行那些最小殘存病灶手術，是因為其所利用的免疫抑制性乳頭狀瘤微環境，而他們用手術來克服這一點。

We have shown with that data that was published in Nature Communications that the elements that Precigen identified did not impact the efficacy of INO-3107. So we believe there's a meaningful therapeutic difference of the two products. And I think also into -- when you look at the two different platforms, we've talked about DNA medicines having the capability of redosing and continuing to generate that T cell response.

我們已透過發表於《Nature Communications》的數據顯示，Precigen 所指出的那些因素並不影響 INO-3107 的療效。因此我們相信這兩個產品之間存在具意義的治療差異。另外，當你比較兩種不同平台時，我們也談過 DNA 藥物具備可重複給藥（redosing）的能力，並能持續產生 T 細胞反應。

RRP is a chronic viral illness, and we believe redosing is going to be an important part of the treatment regimen to ensure these patients can hopefully become surgery-free long term.

RRP 是一種慢性病毒性疾病，我們相信重複給藥將會是治療方案中重要的一部分，以確保這些患者有望在長期上不再需要手術。

Great. That's super helpful. And If I could just ask one follow-up. Could you please talk about when you expect to receive feedback from the FDA on the confirmatory study design? And what sort of feedback do you expect to receive from the FDA?

很好。這非常有幫助。如果我可以再追問一個問題。你們預期何時會收到 FDA 對確證性研究（confirmatory study）設計的回饋？以及你們預期 FDA 會給出什麼樣的回饋？

Yes. Great question, Jay. I mean we expect -- as the confirmatory trial design is really linked to the review pathway and the requirement to conduct that confirmatory trial. We would expect that feedback to be linked to the informal meeting. So I think as part of the informal meeting and discussion of the review pathway, we would hope to learn when we'll get feedback on that confirmatory trial design.

是的。Jay，問得很好。我的意思是——由於確證性試驗設計其實與審查途徑以及是否需要進行該確證性試驗的要求密切相關。我們預期那項回饋會與非正式會議相連結。因此我想，作為非正式會議的一部分，在討論審查途徑時，我們希望能了解何時會收到關於該確證性試驗設計的回饋。

Okay. Great. We look forward to that.

好的。很好。我們期待後續進展。

Your next question comes from Sudan with Stephens.

下一個問題來自 Stephens 的 Sudan。

Great to hear all the progress. So to piggyback off the confirmatory trial questions that was just asked, since there is still a high unmet medical need for RRP despite the PAPZIMEOS being on the market, can you let us know to what capacity you're still treating old or new patients with 3107? And secondly, will you get any longer-term durability of response data from 3107 this year since if I remember correctly, 3107 shined on the previous long-term analysis readout.

很高興聽到所有進展。延續剛才關於確證性試驗的問題，鑑於即使 PAPZIMEOS 已在市場上，RRP 仍存在高度未被滿足的醫療需求，你們能否說明目前仍以 3107 治療既有或新患者的規模到什麼程度？第二，今年你們是否會取得 3107 更長期的反應持久性（durability of response）數據？因為如果我沒記錯，3107 在先前的長期分析讀出中表現非常亮眼。

Yes. Thanks, Sudan, for the great question. Our original Phase I/II trial incorporated the initial 4-dose regimen. And then the follow-up data was actually just a retrospective look back on the same patient population with no additional dosing. So we -- at present, we do not have any planned readout of data on 3107 with continued dosing.

是的。謝謝你，Sudan，提出這個很棒的問題。我們最初的第一/二期試驗納入了最初的 4 劑療程。而後續資料其實只是對同一批病人族群進行回溯性回顧，並沒有額外給藥。因此，我們——目前並沒有任何針對 3107 持續給藥的資料讀出規劃。

We have previously talked about data that we have for INO-3100, which has shown the ability to continue to generate a T cell response 6 to 9 months after the completion of the initial treatment. So we are excited to discuss with the agency what a redosing strategy will look like following approval of 3107 if it gets approved.

我們先前談過我們針對 INO-3100 所取得的資料，顯示在完成初始治療後 6 到 9 個月，仍能持續產生 T 細胞反應。因此，若 3107 獲得核准（如果獲批），我們很期待與主管機關討論核准後的再給藥策略會是什麼樣子。

Great. And secondly, I just wanted to ask also, how have your interactions with the RRP Foundation been recently as you near your PDUFA date amidst also the PAPZIMEOS launch currently happening? Do you feel like you have an opportunity here to further utilize this patient advocacy group as a resource to specifically reach the community setting that PAPZIMEOS, I think, currently has only penetrated about 25% to date?

很好。第二個問題，我也想請教，隨著你們在 PDUFA 日期臨近、同時 PAPZIMEOS 目前也正在上市推廣，你們最近與 RRP Foundation 的互動如何？你們是否覺得有機會進一步把這個病友倡議團體作為資源，特別去觸及社區端（community setting）；我想 PAPZIMEOS 目前在那邊的滲透率到目前為止大約只有 25%？

Yes, that's a great question, Sudan. And I'm very pleased to say that the RRPF Foundation and the RRP community have been incredibly supportive. And we're very much aligned with them. We recognize that the current approved therapy doesn't work for all RRP patients and every RRP patient deserves a therapy that works for them. Every surgery matters to RRP patients.

是的，這是個很好的問題，Sudan。我很高興地說，RRPF Foundation 與 RRP 社群一直給予我們極大的支持。而且我們與他們的目標非常一致。我們認知到目前已核准的療法並非對所有 RRP 病患都有效，而每一位 RRP 病患都值得擁有對他們有效的治療。對 RRP 病患而言，每一次手術都很重要。

So our goals are very much aligned with the foundations. We continue to work very closely with them, and we are very grateful for their support. And we'll obviously continue to work with them going forward. But yes, understanding the patient perspective and the remaining unmet need is absolutely critical to what we're trying to do with 3107.

因此，我們的目標與基金會非常一致。我們持續與他們非常密切合作，也非常感謝他們的支持。未來我們當然也會繼續與他們合作。不過是的，理解病患觀點以及仍未被滿足的需求，對於我們想用 3107 達成的事情而言至關重要。

Yin with H.C. Wainwright.

H.C. Wainwright 的 Yin。

This is [Jan De] sitting in for Yi Chen. I have 2 questions. The first is with respect to the FDA. Do you expect the resignation of the current FDA commissioner to introduce any sort of uncertainty in the review timeline of INO-3107.

我是 [Jan De]，代替 Yi Chen。我有兩個問題。第一個是關於 FDA。你們是否預期現任 FDA 局長的辭職，會對 INO-3107 的審查時程帶來任何不確定性？

Yes. I don't think we can really comment on the inner workings of the FDA at the moment. But what I can say is we continue to engage with our review team and respond to information requests and the review seems to be proceeding. As we commented around the mid-cycle review, they did reiterate that they're still reviewing the assessment aid and that they plan to schedule the informal meeting. And we encourage them to -- we continue to encourage them to schedule that meeting.

是的。我想我們目前確實無法評論 FDA 內部運作。但我可以說的是，我們持續與審查團隊互動、回覆資訊要求，而審查看起來仍在進行中。如同我們在期中審查（mid-cycle review）時所提到的，他們確實重申仍在審查評估輔助文件（assessment aid），並計畫安排非正式會議。我們也鼓勵他們——我們持續鼓勵他們安排那場會議。

We're keen to discuss it with them. So I think where we are at the moment is really just continuing to progress the review, and we're very keen to have that discussion.

我們很希望能與他們討論。所以我想我們目前就是持續推進審查流程，而我們也非常期待能進行那場討論。

And my second question is about when INO-3107 gets approval. So once that happens, what would be your marketing strategy or approach to seize market share from PAPZIMEOS?

我的第二個問題是關於 INO-3107 若獲得核准。一旦發生，你們的行銷策略或做法會是什麼，以從 PAPZIMEOS 手中取得市占？

Yes. So we believe, as Steve, I think, has outlined, we believe that 3107 has a positively differentiated product profile and could become the product of choice for patients and RRP physicians. And this is really based on its clinical effectiveness, our tolerability and a simple patient-centric approach to treatment with 3107. So clearly, we've been learning from the PAPZIMEOS launch. We'll be incorporating key learnings from their launch into our go-to-market plan, and we're planning to be a fast follower.

是的。我們相信，正如 Steve 我想已經概述的，我們相信 3107 具有正向差異化的產品特性，並可能成為病患與 RRP 醫師的首選產品。這主要基於其臨床有效性、我們的耐受性，以及以病患為中心、簡單的 3107 治療方式。因此很明顯地，我們一直在從 PAPZIMEOS 的上市推廣中學習。我們會把他們上市的關鍵學習納入我們的上市策略（go-to-market plan），並計畫成為快速跟進者（fast follower）。

As Steve also outlined, we're expecting the vast majority of the prevalent pool or the available market to still be available when we come to market if approved. And there are also new patients being diagnosed every year. So we think we're in a good position to be able to compete with the approved product. Steve, anything you want to add?

如同 Steve 也提到的，我們預期在我們若獲核准並上市時，現有患者池（prevalent pool）或可觸及市場的大多數仍會存在。而且每年也都有新病患被診斷出來。因此我們認為，我們具備良好條件與已核准產品競爭。Steve，你還想補充什麼嗎？

No, I think that's good.

沒有，我覺得這樣很好。

Liang Cheng, Jefferies.

Jefferies 的 Liang Cheng。

This is Liang Cheng on for Roger Song. I guess from us, how should we think about the potential label at approval, particularly the eligible patient population and anticipated restrictions versus the Phase I/II population?

我是 Liang Cheng，代替 Roger Song。我想從我們的角度，核准時的潛在標籤（label）應該如何看待，特別是符合資格的病患族群，以及相較於第一/二期族群可能的限制？

Liang, nice to hear from you. It's a great question. So we would expect to have a very similar label to the approved product. And that's based on the fact that we recruited patients who have had between 2 to 8 surgeries in the prior year. In our patient population, we had a good balance of patients who were infected with HPV-6 and 11 and even a combination of them both.

Liang，很高興聽到你的聲音。這是個很好的問題。因此我們預期會有一個與已核准產品非常相似的標籤。這是基於我們招募的病患在前一年接受過 2 到 8 次手術。在我們的病患族群中，感染 HPV-6 與 HPV-11 的病患比例相當均衡，甚至也有同時感染兩者的病患。

And we have no evidence from our mechanism of action that efficacy would depend on severity of disease or number of surgeries in the prior year. So we would expect a very similar label with no restrictions.

而且從我們的作用機轉來看，沒有任何證據顯示療效會取決於疾病嚴重程度或前一年手術次數。因此我們預期會是非常相似、且沒有任何限制的標籤。

Okay. Got it. And at a higher level, what -- how are you thinking about pricing relative to Precigen?

好的。了解。那在更高層次上，你們如何思考相對於 Precigen 的定價？

Yes. So we are thinking of rare disease pricing. Clearly, in our payer research that we've conducted, payers recognize that rare disease pricing is appropriate for this indication. We'll be conducting some price optimization research and we'll be commenting on pricing a bit closer to launch.

是的。因此我們是以罕見疾病定價來思考。很明顯地，在我們所做的付款方研究中，付款方認為針對這個適應症採用罕見疾病定價是合適的。我們也會進行一些價格最佳化研究，並會在更接近上市時再對定價做更多評論。

There are no further questions at this time. I will turn the call back over to Jacqui Shea.

目前沒有其他問題。我將把電話會議交回給 Jacqui Shea。

Thank you. As we've outlined here today, Inovio is intent on meeting the milestones ahead for INO-3107 and the other promising candidates in our pipeline. We're motivated by the potential opportunity to deliver on the promise of DNA medicines for RRP patients and grateful for the support from the RRP Foundation and larger RRP community. They have waited so long for relief from the devastating impact of their disease. We believe every patient deserves access to a treatment option that works for them because every patient matters and every surgery matters.

謝謝。如同我們今天在此所概述的，Inovio 致力於達成 INO-3107 以及我們產品線中其他具前景候選項目的後續里程碑。我們受到一個潛在機會所驅動：為 RRP 病患實現 DNA 藥物的承諾；同時也感謝 RRP Foundation 與更廣大的 RRP 社群所給予的支持。他們等待從疾病帶來的毀滅性影響中獲得緩解已經很久了。我們相信每位病患都應該能取得對他們有效的治療選項，因為每位病患都很重要，而每一次手術都很重要。

We're working every day to help make that vision a reality. Thank you for your attention, and good evening, everyone.

我們每天都在努力，讓那個願景成為現實。謝謝各位的關注，祝各位晚安。