Inovio Pharmaceuticals Inc (INO) 2025 Q1 法說會逐字稿

Good afternoon, ladies and gentlemen. Welcome to the Inovio first -quarter 2025 financial results conference call.

女士們、先生們，午安。歡迎參加 Inovio 2025 年第一季財務業績電話會議。

(Operator Instructions) This call is being recorded today, Tuesday, May 13, 2025.

（操作員指示）本次通話於今天（2025 年 5 月 13 日，星期二）進行錄音。

I would now like to turn the conference over to Jennie Willson, Director of Communications. Jennie, please go ahead.

現在我想將會議交給通訊主管珍妮威爾森 (Jennie Willson)。珍妮，請繼續。

Good afternoon. Thank you for joining the Inovio first-quarter 2025 financial results conference call.

午安.感謝您參加 Inovio 2025 年第一季財務業績電話會議。

Joining me today on today's call are Dr. Jacque Shea, President and Chief Executive Officer; Dr. Mike Sumner, Chief Medical Officer; Peter Kies, Chief Financial Officer; and Steve Egge, Chief Commercial Officer.

今天參加電話會議的有總裁兼執行長 Jacque Shea 博士、首席醫療官 Mike Sumner 博士、財務長 Peter Kies 和首席商務官 Steve Egge。

Today's call will review our corporate and financial information for the quarter ended March 31, 2025, as well as provide a general business update. Following prepared remarks, we will conduct a question-and-answer session.

今天的電話會議將回顧我們截至 2025 年 3 月 31 日的季度的公司和財務信息，並提供一般業務更新。在準備好的發言之後，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 events relate to our business plans to develop Inovio's DNA medicines platform, which include clinical and regulatory developments and timing of clinical data readouts and planned regulatory submissions, along with capital resources and strategic matters.

在電話會議中，我們將就未來事件和公司未來表現做出前瞻性陳述。這些事件與我們開發 Inovio 的 DNA 藥物平台的業務計劃有關，其中包括臨床和監管發展、臨床數據讀數和計劃監管提交的時間，以及資本資源和戰略事項。

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.

所有這些聲明都是基於管理層今天的信念和期望。實際事件或結果可能會有重大差異。我們請您參閱我們不時向美國證券交易委員會提交的文件，這些文件在「風險因素」標題下，列出了可能導致實際結果與公司口頭表達的結果以及今天下午的新聞稿中所作的聲明存在重大差異的重要因素。

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

本次電話會議正在進行網路直播。您可以在我們的網站 ir.inovio.com 上找到連結。本次通話結束後不久，我們將提供重播。

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

現在我將電話轉給 Inovio 總裁兼執行長 Jacque Shea 博士。

Good afternoon. Thank you to everyone for joining today's call.

午安.感謝大家參加今天的電話會議。

First of all, I am very pleased to say that we remain on track to submit our BLA for INO-3107, our lead candidate for the treatment of Recurrent Respiratory Papillomatosis or RRP.

首先，我很高興地說，我們仍在按計劃提交 INO-3107 的 BLA，這是我們治療復發性呼吸道乳頭狀瘤病 (RRP) 的主要候選藥物。

As previously stated, our goal is to begin rolling submissions in mid-2025, complete the submission in the second half, and receive file acceptance by the end of this year. This would allow for a PDUFA date in mid-2026, if we receive priority review.

如前所述，我們的目標是在 2025 年中期開始滾動提交，在下半年完成提交，並在今年年底前收到文件接受。如果我們獲得優先審查，這將允許在 2026 年中期確定 PDUFA 日期。

Our primary focus continues to be the submission of our BLA for 3107. We reported in March that we had resolved the manufacturing issue involving the single-use array component of the SELETRA device. And we have now moved on to the next step: initiating the device Design Verification Testing, known as DV Testing, required for our IND update and BLA submissions.

我們的主要重點仍然是提交 3107 的 BLA。我們在三月報告稱，我們已經解決了涉及 SELETRA 設備一次性陣列組件的製造問題。現在我們進入了下一步：啟動設備設計驗證測試（稱為 DV 測試），這是我們的 IND 更新和 BLA 提交所必需的。

We've also been pursuing targeted opportunities to highlight key data from our completed Phase I/II trial. We were delighted to announce the publication of clinical and immunology data from that trial in Nature Communications in February.

我們也一直在尋找有針對性的機會來突出我們已完成的 I/II 期試驗的關鍵數據。我們很高興地宣布，該試驗的臨床和免疫學數據於二月在《自然通訊》上發表。

We also look forward to the publication of the previously announced data on the longer-term clinical effect of 3107, which has been submitted to a peer-reviewed journal. In addition, we've been very active at scientific conferences this spring, which has been integral to our strategic outreach to healthcare providers and Key Opinion Leaders. Mike will provide an update on that shortly.

我們也期待先前公佈的3107長期臨床效果數據，該數據已提交給同行評審期刊。此外，今年春季我們非常積極地參加科學會議，這對於我們與醫療保健提供者和關鍵意見領袖的策略推廣至關重要。麥克將很快提供有關該問題的最新消息。

On the commercial readiness front, ongoing market research with physicians, patients, and payers continues to support our belief that 3107 has the potential to be the preferred product for patients and physicians, if approved. Steve will provide a brief overview of both the market opportunity and insights from our market research with healthcare providers later in the call.

在商業準備方面，與醫生、患者和付款人進行的持續市場研究繼續支持我們的信念，即 3107 如果獲得批准，則有可能成為患者和醫生的首選產品。史蒂夫將在稍後的電話會議中簡要介紹市場機會以及我們與醫療保健提供者進行的市場研究的見解。

And, finally, a highlight from our earlier-stage pipeline this quarter, with the announcement of promising interim results from an ongoing Phase I proof-of-concept trial evaluating DNA-encoded monoclonal antibodies or dMAbs. We're excited about the potential of this next-generation technology and what it could mean for the future of our DNA medicines pipeline.

最後，本季度我們早期階段研發管線的一個亮點是，我們宣布了正在進行的評估 DNA 編碼單株抗體或 dMAb 的第一階段概念驗證試驗的有希望的中期結果。我們對這項下一代技術的潛力以及它對我們的 DNA 藥物管道未來的意義感到非常興奮。

With that, I'll turn it over to Mike for a brief update on our progress with 3107 and some highlights from our recent presentations. Mike?

接下來，我將把時間交給 Mike，讓他簡要介紹我們在 3107 方面的進展以及我們最近演示的一些亮點。麥克風？

Thanks, Jacque.

謝謝，雅克。

As Jacque noted, we are making steady progress towards our BLA submission goal for INO-3107. The manufacturing issue involving the single-use array component of the device has been resolved. We have begun manufacturing the updated commercial-grade arrays and initiated DV Testing, which is a significant component of the device-related BLA modules.

正如 Jacque 所說，我們正在穩步推進 INO-3107 的 BLA 提交目標。涉及該設備一次性陣列組件的製造問題已解決。我們已經開始製造更新的商業級陣列並啟動 DV 測試，這是與設備相關的 BLA 模組的重要組成部分。

As a reminder, we have completed drafting all the non-device modules, including non-clinical, clinical, and CMC modules. And will request to begin the submission of our BLA under the FDA's rolling submission process in mid-2025. In addition, we remain on track to launch our confirmatory trial targeting more than 20 sites at major US medical centers.

提醒一下，我們已經完成了所有非設備模組的起草，包括非臨床、臨床和 CMC 模組。並將請求於 2025 年中期根據 FDA 的滾動提交流程開始提交我們的 BLA。此外，我們仍計劃在美國主要醫療中心的 20 多個地點進行確認性試驗。

While that work is underway, we've leveraged several important opportunities to engage with the RRP community, sharing why we believe 3107 could be the preferred product for patients and doctors, who are eager for an alternative to surgery.

在進行這項工作的同時，我們利用了幾個重要的機會與 RRP 社區接觸，分享了我們為什麼相信 3107 可能是渴望尋求手術替代方案的患者和醫生的首選產品。

3107 offers the potential for a majority of patients to see significant clinical benefit that improves over time, a favorable safety profile, and a patient-centric treatment regimen.

3107 預計將使大多數患者獲得隨時間推移而改善的顯著臨床益處、良好的安全性和以患者為中心的治療方案。

This spring, we have presented key clinical and safety data at multiple scientific conferences, including the inaugural National HPV Conference, the first scientific conference in the US to focus solely on HPV research and related diseases. It was an excellent opportunity to connect with experts from across sectors and to broaden awareness of both RRP and the potential benefit of INO-3107.

今年春天，我們在多個科學會議上展示了關鍵的臨床和安全數據，包括第一屆全國 HPV 會議，這是美國第一個專注於 HPV 研究和相關疾病的科學會議。這是與各個領域的專家聯繫並加深對 RRP 和 INO-3107 潛在益處的認識的絕佳機會。

This week, Inovio will also be presenting year 2 and 3 durability data at the Combined Otolaryngology Spring meeting, otherwise known as COSM. This is the largest US national meeting for otolaryngologists, the specialist physicians who treat the majority of RRP patients.

本週，Inovio 也將在聯合耳鼻喉科春季會議（也稱為 COSM）上展示第 2 年和第 3 年的耐久性數據。這是美國最大的耳鼻喉科醫生全國會議，耳鼻喉科醫生是治療大多數 RRP 患者的專家。

At these conferences, we've been able to paint a compelling picture of the potential impact of 3107 for the RRP community; for the patients, physicians, and caregivers who know that every single surgery comes with both significant risk and cost.

在這些會議上，我們能夠清楚地描繪出 3107 對 RRP 社區的潛在影響；對於患者、醫生和護理人員來說，他們知道每一次手術都伴隨著巨大的風險和成本。

As a reminder, we completed a Phase I/II open-label trial of INO-3107 called RRP-001 in patients who required at least two surgeries in the previous year for the removal of HPV6 and HPV11-related papillomas. Every surgery performed after the initial dose was counted against the efficacy endpoint in our trial, where we followed the patients for 12 months.

提醒一下，我們完成了 INO-3107 的 I/II 期開放標籤試驗，稱為 RRP-001，針對在過去一年中需要至少兩次手術以切除 HPV6 和 HPV11 相關乳頭狀瘤的患者。在我們的試驗中，初始劑量後進行的每一次手術都計入療效終點，我們對患者進行了 12 個月的追蹤。

We also conducted a retrospective trial called RRP-002, where we collected data on 28 of the original 32 patients to assess the longer-term treatment effect, with a median follow-up of 2.8 years.

我們還進行了一項名為 RRP-002 的回顧性試驗，收集了最初 32 名患者中的 28 名患者的數據，以評估長期治療效果，中位追蹤期為 2.8 年。

The key takeaway from these two studies is clear. We saw a statistically significant reduction in surgeries in the first 12 months following treatment; and that clinical benefit continued to improve beyond the initial one-year period into year 2 and 3 time-frame.

這兩項研究的關鍵結論很明確。我們發現，治療後前 12 個月內手術數量顯著減少；且臨床益處在最初的一年後持續改善，持續到第 2 年和第 3 年。

More specifically, in the first year, 72% of patients saw a 50% to 100% reduction in the number of surgeries, after starting treatment with 3107. With no additional dosing, this number increased to 86% in the second year, with half of the patients requiring no surgeries at all.

更具體地說，在第一年，72% 的患者在開始使用 3107 治療後，手術次數減少了 50% 至 100%。在沒有額外劑量的情況下，第二年這一數字增加到 86%，其中一半患者根本不需要手術。

Moving on to next steps. We are focused on completing the DV Testing and finalizing the device-related aspects of our BLA.

繼續下一步。我們專注於完成 DV 測試並最終確定 BLA 的設備相關方面。

As a reminder, this testing is required for both our BLA submission and to update the IND, before we can dose patients in our confirmatory trial.

提醒一下，在我們的確認性試驗中對患者進行給藥之前，我們的 BLA 提交和更新 IND 都需要進行此項測試。

Our timeline for the BLA remains the same. We plan to request rolling submission and begin submitting our modules in mid-2025 and complete the full submission in the second half of the year. Our goal is to have FDA acceptance of our complete BLA filing by year-end.

我們對 BLA 的時間表保持不變。我們計劃要求滾動提交，並於 2025 年中期開始提交我們的模組，並在下半年完成全部提交。我們的目標是讓 FDA 在年底前接受我們的完整 BLA 申請。

And if we receive priority review, that could allow for a PDUFA date in mid-2026. After that, we plan to finalize our longer-term treatment strategy, with the goal of maintaining or even improving upon the clinical benefit seen to date and submit a proposed protocol to the FDA to support a supplemental BLA in the future.

如果我們獲得優先審查，PDUFA 的生效日期就可以定在 2026 年中期。之後，我們計劃最終確定我們的長期治療策略，目標是維持甚至改善迄今為止所見的臨床益處，並向 FDA 提交擬議方案以支持未來的補充 BLA。

The ability to maintain or increase the immune response over time by continuing treatment is a key feature of our DNA medicines platform and has been demonstrated in our previous work in other HPV-related indications.

透過持續治療來維持或增強免疫反應的能力是我們 DNA 藥物平台的關鍵特性，並且已在我們先前針對其他 HPV 相關適應症的研究工作中得到證實。

And, finally, as we noted last quarter, deployment of our medical science liaison team is planned for this quarter. And I look forward to providing an update at our next quarterly report.

最後，正如我們上個季度所指出的，我們計劃在本季部署醫學科學聯絡團隊。我期待在下一份季度報告中提供最新進展。

I will now turn it over to our Chief Commercial Officer, Steve Egge, for some insights on the market opportunity for 3107. Steve?

現在，我將把主題交給我們的商務長 Steve Egge，請他談談對 3107 市場機會的一些見解。史蒂夫？

Thanks, Mike.

謝謝，麥克。

I'd like to spend a few minutes on the significant market opportunity we see for 3107 and why we believe that 3107 could be the product of choice for patients and providers.

我想花幾分鐘時間談談我們看到的 3107 的巨大市場機遇，以及我們為什麼相信 3107 可以成為患者和供應商的首選產品。

I'll start by describing the market opportunity. RRP is a rare HPV-related disease that affects around 14,000 people in the US. Because HPV vaccination rates are plateauing and because the majority of the adult population remain unvaccinated, risk of RRP remains.

我首先要描述一下市場機會。RRP 是一種罕見的 HPV 相關疾病，影響美國約 14,000 人。由於 HPV 疫苗接種率趨於穩定，且大多數成年人口仍未接種疫苗，因此 RRP 的風險仍然存在。

HPV experts believe the HPV vaccine is unlikely to have a significant impact on RRP prevalence in adults in the near term or for at least a generation. This disease is characterized by persistent work-like growth called papilloma in the respiratory tract.

HPV專家認為，HPV疫苗不太可能在短期內或至少一代人內對成年人的RRP盛行率產生重大影響。這種疾病的特徵是呼吸道中持續出現稱為乳頭狀瘤的類似工作物的生長物。

There are currently no regulatory-approved therapeutic options available and the current standard of care is surgery; often, multiple surgeries a year. These surgeries have the potential to cause irreversible harm to the vocal cords and the surgery does not address the underlying disease, so the papillomas can grow back, repeatedly.

目前尚無監管機構批准的治療方案，目前的治療標準是手術；通常每年進行多次手術。這些手術可能會對聲帶造成不可逆的傷害，而且手術並不能解決潛在的疾病，因此乳頭狀瘤可能會反覆生長。

Patients and doctors have expressed time and again the urgent need for a non-surgical treatment option that addresses the underlying cause of the disease. And this is where we see the potential for 3107. In our trial, 3107 provided significant clinical benefit; was well tolerated; and is delivered via a simple patient-centric treatment regimen.

患者和醫生一再表示，迫切需要一種能夠解決疾病根本原因的非手術治療方案。這就是我們看到 3107 的潛力的地方。在我們的試驗中，3107 提供了顯著的臨床益處；耐受性良好；並且透過簡單的以患者為中心的治療方案進行。

After reviewing our data during market research, many laryngologists commented that about 8 out of 10 patients achieved a 50% to 100% reduction in surgeries; meaning, the vast majority of patients saw a significant benefit from treatment. And this is the data that they found most compelling and that they believe will be most meaningful to patients.

在市場調查期間查看了我們的數據後，許多喉科醫生表示，大約有十分之八的患者手術次數減少了 50% 到 100%；這意味著絕大多數患者都從治療中獲得了顯著的益處。他們認為這些數據最有說服力，對患者最有意義。

Treatment with 3107 was also well tolerated, with the most common adverse events being transient injection site pain and fatigue. And there were no discontinuations.

3107 治療的耐受性也很好，最常見的不良事件是短暫性注射部位疼痛和疲勞。且沒有出現停產的情況。

The simplicity of our treatment regimen is also key. Most notably, 3107 does not require scoping and surgeries during the window as part of the treatment regimen, which is vitally important when every single surgery comes with real risk and cost to patients.

我們的治療方案簡單也是關鍵。最值得注意的是，3107 不需要在治療期間進行檢查和手術，這是至關重要的，因為每一次手術都會為患者帶來真正的風險和費用。

3107 can also be administered in the physician's office and does not require a referral to an infusion center, which leaves the physician in control.

3107 也可以在醫生辦公室內使用，不需要轉診到輸液中心，而由醫生來控制。

These and other market insights gathered to date will be critical, as we continue to advance our commercial planning. We're currently refining our go-to-market model and planning a further build-out of the commercial organization.

隨著我們繼續推進商業規劃，迄今為止收集到的這些和其他市場見解將至關重要。我們目前正在完善我們的市場進入模式並計劃進一步擴大商業組織。

And I look forward to providing more updates on our progress next quarter.

我期待下個季度提供更多有關我們進展的最新消息。

With that, I'll turn it back to Jacque.

說完這些，我就把它交還給雅克。

Thanks, Steve.

謝謝，史蒂夫。

As I mentioned earlier, we're also making important progress with the next generation of DNA medicine, with our DNA-encoded monoclonal antibody or dMAb technology. This technology leverages the strengths of our DNA medicine platform to create precisely designed DNA plasmids that encode for specific monoclonal antibodies.

正如我之前提到的，我們也在下一代 DNA 醫學方面取得了重要進展，利用我們的 DNA 編碼單株抗體或 dMAb 技術。該技術利用我們的 DNA 藥物平台的優勢，創建精確設計的編碼特定單株抗體的 DNA 質粒。

These plasmids can then be delivered directly into muscle cells in the arm, using our Selectra delivery system. The dMAbs are produced within the muscle cells and then, secreted into the blood, where they circulate within the body.

然後，我們可以使用 Selectra 輸送系統將這些質粒直接輸送到手臂的肌肉細胞中。dMAb 在肌肉細胞內產生，然後分泌到血液中，並在體內循環。

This contrasts with conventional monoclonal antibodies, which are manufactured in in vitro systems and then, need to be administered through regular infusions or injections.

這與傳統的單株抗體形成對比，傳統的單株抗體是在體外系統中製造的，然後需要透過定期輸注或註射來給藥。

We are researching the potential of this technology in several disease targets and recently announced interim clinical data from an ongoing Phase I proof-of-concept trial evaluating dMAbs for COVID-19, led by the Wistar Institute, in collaboration with AstraZeneca, the University of Pennsylvania, and Inovio; and funded by DARPA and JPO.

我們正在研究這項技術在多種疾病目標中的潛力，並最近公佈了正在進行的 I 期概念驗證試驗的中期臨床數據，該試驗評估了針對 COVID-19 的 dMAb，該試驗由威斯塔研究所牽頭，與阿斯利康、賓夕法尼亞大學和 Inovio 合作；並由 DARPA 和 JPO 資助。

This trial provided the first clinical proof of concept that dMAbs can be durably and simultaneously produced inside the human body. The data showed long-lasting in vivo antibody production across 72 weeks; no antidrug antibodies or immune rejection of the dMAbs; and the treatment was well tolerated, with no serious adverse events related to treatment.

這項試驗首次提供了臨床概念證明，證明 dMAb 可以在人體內持久且同時產生。數據顯示，72 週內體內抗體的產生持續時間很長；沒有抗藥性抗體或對 dMAbs 的免疫排斥；治療耐受性良好，沒有與治療相關的嚴重不良事件。

Of note, we saw that the express dMAbs successfully bound the SARS-CoV-2 spike protein receptor binding domain, confirming functional activity.

值得注意的是，我們發現表達的 dMAb 成功結合了 SARS-CoV-2 刺突蛋白受體結合域，證實了功能活性。

To be clear, this was a proof-of-concept study of our technology. Inovio does not have plans to develop these dMAbs for COVID-19, going forward.

需要明確的是，這是對我們技術的概念驗證研究。Inovio 目前沒有計劃針對 COVID-19 開發這些 dMAb。

Part of this data will be presented at ASGCT this week by our partners at the Wistar Institute. And a manuscript has been submitted to a leading peer-reviewed journal and is currently available in pre-print on Research Square.

我們在威斯塔研究所的合作夥伴本週將在 ASGCT 上展示部分數據。目前，論文的手稿已提交給一家領先的同行評審期刊，目前可在 Research Square 上進行預印。

We believe this technology has breakthrough potential and could overcome many of the challenges seen with traditional monoclonal antibody production, offering rapid manufacturing, low cost of production, temperature-stable storage and distribution, and the ability to redose.

我們相信這項技術具有突破性的潛力，可以克服傳統單株抗體生產中遇到的許多挑戰，提供快速製造、低成本生產、溫度穩定的儲存和分銷以及重複使用的能力。

dMAb technology could help expand use, reduce cost, and enable access in low resource settings.

dMAb 技術可以幫助擴大用途、降低成本並實現在資源匱乏的環境中應用。

And, importantly, our DNA-based approach has demonstrated sustained antibody production without generating antidrug antibodies, making it a potentially promising long-term solution for conditions requiring continuous therapy.

而且，重要的是，我們基於 DNA 的方法已證明能夠持續產生抗體，而不會產生抗藥性抗體，這使其成為需要持續治療的疾病的潛在有希望的長期解決方案。

We're excited about the potentially broad application we see for this technology, including the potential to leverage the sustained in vivo protein production we have observed to produce other kinds of proteins beyond monoclonal antibodies.

我們對這項技術的潛在廣泛應用感到非常興奮，包括利用我們觀察到的持續體內蛋白質生產來生產單株抗體以外的其他類型蛋白質的潛力。

For instance, to enable protein replacement therapies; and as a potential alternative to gene therapy for some indications.

例如，實現蛋白質替代療法；並作為某些適應症的基因療法的潛在替代方案。

I'll now hand over to our CFO, Peter Kies, for a brief financial update. Peter?

現在我將把時間交給我們的財務長 Peter Kies，讓他簡要地報告財務狀況。彼得？

Thanks, Jacque.

謝謝，雅克。

Today, I'd like to provide an overview of Inovio's financial results for the first-quarter 2025.

今天，我想概述 Inovio 2025 年第一季的財務表現。

As Jackie mentioned, we're focusing resources on advancing our lead candidate, 3107, and our other strategic priorities. And I'm pleased to report that we've been able to support significant progress towards those goals, while continuing to reduce costs.

正如 Jackie 所提到的，我們正集中資源推進我們的主要候選人 3107 和我們的其他策略重點。我很高興地報告，我們已經能夠支持這些目標取得重大進展，同時繼續降低成本。

As you can see here, we've been able to significantly reduce our operating expenses over the past year.

正如您所看到的，我們在過去一年中已經大幅降低了營運費用。

This quarter's operating expenses dropped from $31.5 million in the first quarter of 2024 to $25.1 million in the first quarter of 2025, a 20% decrease.

本季營運費用從2024年第一季的3,150萬美元下降至2025年第一季的2,510萬美元，降幅達20%。

Inovio's net loss for the first quarter of 2025 was $19.7 million or $0.51 per share, basic and dilutive, compared to a net loss of $30.5 million or $1.31 per share, basic and dilutive, for the first quarter of 2024.

Inovio 2025 年第一季的淨虧損為 1,970 萬美元，即每股 0.51 美元（基本和稀釋），而 2024 年第一季的淨虧損為 3,050 萬美元，即每股 1.31 美元（基本和稀釋）。

We finished the first quarter of 2025 with $68.4 million in cash, cash equivalents, and short-term investments, compared to $94.1 million, as of December 31, 2024.

截至 2025 年第一季度，我們的現金、現金等價物和短期投資為 6,840 萬美元，而截至 2024 年 12 月 31 日，我們的現金、現金等價物和短期投資為 9,410 萬美元。

We estimate our cash runway to take us into the first quarter of 2026. This projection includes an operational net cash burn estimate of approximately $22 million for the second quarter of 2025. These cash runway projections do not include any further capital raise activities that we may undertake.

我們估計我們的現金流可以維持到 2026 年第一季。該預測包括 2025 年第二季約 2,200 萬美元的營運淨現金消耗估計。這些現金流預測不包括我們可能進行的任何進一步的融資活動。

As a reminder, you can find our full financial statements in this afternoon's press release, as well as in our quarterly report, Form 10-Q, filed with the SEC today.

提醒一下，您可以在今天下午的新聞稿以及今天向美國證券交易委員會提交的季度報告 10-Q 表中找到我們的完整財務報表。

With that, I'll turn it back over to Jacque.

說完這些，我會把話題交還給雅克。

Thanks, Peter.

謝謝，彼得。

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

現在我想開始回答你們可能提出的任何問題。操作員？

(Operator Instructions)

（操作員指示）

Roy Buchanan, Citizens.

羅伊·布坎南，公民。

To start, maybe -- can you just give any additional detail on the COSM presentation, coming up in a few days? Are you going to have any data beyond year 3?

首先，您能否提供有關幾天後即將舉行的 COSM 演示的更多細節？您會獲得第三年以後的任何數據嗎？

And what else are you going to present other than surgery counts?

除了手術計數之外您還要展示什麼？

Roy, nice to hear from you. Mike, do you want to give some further background on our COSM presentation?

羅伊，很高興收到你的來信。麥克，你想進一步介紹一下我們的 COSM 演示的背景嗎？

Yeah. Absolutely. We'll obviously be focusing on the surgery counts as, obviously, every surgery matters to these patients because of the risk and the cost. But we will be able to show a little bit more color around that.

是的。絕對地。我們顯然會關注手術的數量，因為顯然，由於風險和費用，每一次手術對這些患者都很重要。但我們將能夠展示更多有關它的顏色。

Also, as part of having an oral presentation at COSM, you also get to submit the data to Laryngoscope. So we will hopefully be having that peer-reviewed publication available soon, also; which will, again, provide additional color.

此外，作為在 COSM 進行口頭陳述的一部分，您也可以將資料提交給 Laryngoscope。因此，我們希望很快也能獲得經過同行評審的出版物；這將再次提供更多內容。

Yeah. And I think the real importance of COSM is this is really the primary meeting for otolaryngologists, who are the primary treating physicians for RRP. So it really is a good opportunity to get our data in front of the physicians who really matter for RRP patients.

是的。我認為 COSM 的真正重要性在於它實際上是耳鼻喉科醫生的主要會議，而耳鼻喉科醫生是 RRP 的主要治療醫生。因此，這確實是一個很好的機會，讓我們的數據呈現給真正關心 RRP 患者的醫生。

Okay. Great. And then a follow-on. Can you remind me how many MSLs you plan to onboard?

好的。偉大的。然後是後續內容。您能提醒我您打算搭載多少名 MSL 嗎？

And I had a question about the epidemiology. As we know, the 14,000 number is a bit older. And Precigen is saying 27,000; although, I'm not sure how they're backing that up.

我有一個關於流行病學的問題。我們知道，14,000 這個數字有點老了。Precigen 稱該數字為 27,000；不過，我不確定他們是如何證實這一數字的。

Are you coming up with your own number? Or how do you plan to think about the market? And when might we see an updated number, if you are?

你想出自己的號碼了嗎？或者你打算如何看待市場？如果您願意的話，我們什麼時候可以看到更新後的數字？

Yeah. Both great questions. Maybe, we'll take the epi question, first of all. So, yeah, the 14,000 of active cases here in the US is relatively old data. It's the most commonly cited data by most experts in the field.

是的。這兩個問題都很好。也許，我們先來討論流行病學問題。是的，美國 14,000 例活躍病例是相對較舊的數據。這是該領域大多數專家最常被引用的數據。

And we have been conducting our own research to try and get a better handle on that number. And our own research suggests, in common with many rare diseases, that that might be an underestimate.

我們一直在進行自己的研究，試圖更好地掌握這個數字。我們自己的研究表明，與許多罕見疾病一樣，這一數字可能被低估了。

Steve, do you want to add any other comments to that?

史蒂夫，你還想補充其他評論嗎？

Yeah. The only other thing I would add is -- and this is common in rare disease -- for RRP, there's no diagnostic code, right? So there's no source to go to get a count of the number of patients.

是的。我唯一想補充的是——這在罕見疾病中很常見——對於 RRP，沒有診斷代碼，對嗎？因此沒有資料來源可以統計患者人數。

So we have done some research and continue to do research looking at procedure codes that are used to conduct RRP procedures; even that is not particularly straightforward.

因此，我們做了一些研究，並將繼續研究用於執行 RRP 程序的程序代碼；即使這樣也不是特別簡單。

But based on what we see, we do think the 14,000 is an underrepresentation. But we're continuing to look at this through research.

但根據我們所看到的情況，我們確實認為 14,000 這個數字是被低估的。但我們仍在繼續研究這個問題。

And, as we go forward, we may be able to share more. But, now, we prefer to just be conservative and quote the 14,000.

而且，隨著我們不斷前進，我們也許能夠分享更多。但現在，我們更願意保守一點，報出 14,000 元。

But, again, we do think it's an underrepresentation.

但我們再次認為，這種說法仍然不足。

And, then, that, of course, is the prevalent pool. And, on top of that, there are new cases arising every year. And the figures there are about 1.8 per 100,000 new or instant cases a year.

那麼，那當然就是普遍的池子了。除此之外，每年還會出現新的案件。那裡的數字是每年每10萬例新發或瞬時病例中約有1.8例。

So, actually, this is -- for a rare disease, there is actually a pretty significant pool of patients that need to be addressed.

因此，實際上，對於一種罕見疾病來說，實際上有相當多的患者需要治療。

In terms of the MSLs, Mike, do you want to add (inaudible) about that?

關於 MSL，Mike，你想補充（聽不清楚）嗎？

We haven't specifically guided yet, as to the size of the MSL team. But, as you've heard Steve mentioned before, the actual number of physicians treating RRP is not that large.

關於 MSL 團隊的規模，我們還沒有具體說明。但是，正如史蒂夫之前提到的，實際治療 RRP 的醫生數量並不多。

And so, as we are considering the number of MSLs, it's really about what we think that Key Opinion Leader base will look like; and, also, considering just the geography of where we'll have those interactions and to optimize that.

因此，當我們考慮 MSL 的數量時，我們實際上考慮的是關鍵意見領袖群體將會是什麼樣子；並且，我們還會考慮進行這些互動的地理位置並對其進行最佳化。

Jay Olson, Oppenheimer.

傑伊·奧爾森，奧本海默。

Congrats on the progress. Maybe, to start with, as you look ahead to a potential approval for 3107, do you expect to have a surgery-sparing claim in the label? And how important is the surgery -sparing benefit for differentiation from your competitors?

恭喜你取得進展。也許，首先，當您展望 3107 的潛在批准時，您是否希望在標籤中聲明避免手術？無需手術的優勢對於您與競爭對手的差異化有多重要？

Yeah. That's a great question. Mike?

是的。這是一個很好的問題。麥克風？

It's an interesting terminology. Ultimately, in our discussions with the FDA, they recognize the clinical benefit is that reduction in surgeries. And so, obviously, as you look at our Phase I/II data, we see a statistically significant reduction in those number of surgeries.

這是一個有趣的術語。最終，在我們與 FDA 的討論中，他們認識到臨床益處是減少手術。因此，顯然，當您查看我們的 I/II 期數據時，我們會發現手術數量在統計上顯著減少。

How the FDA will actually approach that terminology, I think, is too early to predict. But I think all parts lead to a reduction in surgeries.

我認為，FDA 實際上將如何處理該術語現在還無法預測。但我認為各個部分都會減少手術。

Okay. Great. And then, since you provided the update on your rolling submission of a BLA, is there any update on your registrational strategy in other geographies outside the US?

好的。偉大的。然後，既然您提供了有關 BLA 滾動提交的更新，那麼您在美國以外其他地區的註冊策略是否有任何更新？

Yeah. Just as a reminder: our confirmatory trial is going to be in patients who've had two or more surgeries in the prior year. It's also going to be a placebo-controlled trial, 2:1 randomization.

是的。提醒一下：我們的確認試驗將針對前一年接受過兩次或兩次以上手術的患者。這也將是一項安慰劑對照試驗，2:1 隨機化。

And in discussions with European regulators and with the UK regulators, they've been very clear that they expect to see placebo-controlled data for approval.

在與歐洲監管機構和英國監管機構的討論中，他們非常明確地表示，他們希望看到安慰劑對照數據以供批准。

So, as Mike mentioned earlier on in the call, after we've started our confirmatory trial and after our BLA is in, the next thing we're really going to be focusing on is getting in a protocol for continued treatment.

因此，正如 Mike 在電話中早些時候提到的那樣，在我們開始確認試驗並且 BLA 完成後，我們接下來真正要關注的是製定持續治療的方案。

That's our next immediate priority. But, certainly, we've had some good discussions with European regulators. And we think we're well aligned with them, in terms of the design of any trials required there.

這是我們的下一個當務之急。但當然，我們已經與歐洲監管機構進行了一些很好的討論。我們認為，就那裡所需的任何試驗的設計而言，我們與他們保持一致。

Okay. Great. And then, for your dMAb platform, we understand that you use COVID-19 as a proof of concept. What are you thinking of, in terms of your initial indication for the dMAb technology?

好的。偉大的。然後，對於您的 dMAb 平台，我們了解您使用 COVID-19 作為概念驗證。就您對 dMAb 技術的初步預期而言，您有何想法？

Yeah. So, outside of those indications that have been disclosed through publications, we haven't disclosed the indications we're currently working on.

是的。因此，除了透過出版物披露的那些適應症之外，我們還沒有披露我們目前正在研究的適應症。

But, as you can imagine, with such a broad and versatile technology, we can apply this to monoclonal antibodies. We can also apply this to proteins that are missing or defective in certain indications, such as enzyme replacement therapies.

但是，正如您所想像的，有瞭如此廣泛和多功能的技術，我們可以將其應用於單株抗體。我們也可以將其應用於某些適應症中缺失或有缺陷的蛋白質，例如酵素替代療法。

So we'll be providing more details, when we have additional data to share there.

因此，當我們有更多數據可以分享時，我們將提供更多詳細資訊。

Sudan Loganathan, Stephens.

蘇丹·洛加納坦，史蒂芬斯。

I appreciate the update today. Congrats on the continued progress towards filing for INO-3107.

我很感謝今天的更新。恭喜 INO-3107 申請繼續取得進展。

My first question is regarding the priority review for 3107. Could receiving priority review status be in any jeopardy, with the potential of another RRP treatment on the market this August/September, potentially, with a competitor's therapeutic? Or is there anything else outstanding that is the final steps to actually making sure that the filing is completed for priority review status?

我的第一個問題是關於 3107 的優先審查。今年 8 月/9 月市場上可能會出現另一種 RRP 療法，並且可能與競爭對手的療法相同，那麼獲得優先審查地位是否會受到威脅？或者還有其他未完成的最後步驟來確保申請能夠獲得優先審查狀態？

Thanks, Sudan. When you look at the guidelines around accelerated approval, it talks to, obviously, both the clinical benefit in, most often, a rare disease for RRP. But it also talks to the difference of the product we're bringing to market.

謝謝，蘇丹。當您查看有關加速審批的指南時，它顯然會談到 RRP 對最常見的罕見疾病的臨床益處。但它也談到了我們推向市場的產品的差異。

I think we've always felt that based on some of the Precigen data, with potentially their efficacy, could be impacted by neutralizing antibodies or by the papilloma microenvironment, neither of which impact INO-3107.

我認為我們一直認為，根據 Precigen 的一些數據，其功效可能會受到中和抗體或乳頭狀瘤微環境的影響，而這兩者都不會影響 INO-3107。

We've always felt there is a population that can only be uniquely treated with 3107. So I don't think -- if we have to have that discussion with the agency, I think we have a very solid rationale of why 3107 should still be brought to market, under the accelerated approval pathway.

我們始終認為，對於某些人群來說，只有 3107 才能提供獨特的治療。因此，我認為——如果我們必須與該機構進行討論，那麼我們有非常充分的理由說明為什麼 3107 仍應透過加速審批途徑推向市場。

And my second question, really quick, is since your filing is expected to be completed by the year end of 2025, is there any plans to add any more data to the actual filing? Or is it already all pencils down, when it comes to adding to the data package or the filing package; and just, now, jumping through the hoops of actually getting it submitted?

我的第二個問題，非常簡短，由於您的申請預計將於 2025 年底完成，是否有計劃在實際申請中添加更多數據？或者，當涉及到添加資料包或文件包時，一切都已經準備好了；現在，只需克服重重困難，真正地提交它？

Yeah. Obviously, one of the reasons we went ahead and performed RRP-002 was to strengthen our overall clinical and safety package. But, as I mentioned earlier on the call, that's all now integrated.

是的。顯然，我們繼續進行 RRP-002 的原因之一是加強我們的整體臨床和安全方案。但是，正如我之前在電話中提到的那樣，現在一切都已經整合在一起了。

So from a clinical perspective, it really is pencils down. And they're ready to go.

因此從臨床角度來看，這確實是放下鉛筆。他們已經準備出發了。

But it's an important point. We got breakthrough therapy designation. And then, our initial discussions with the FDA were just on that initial Phase III 12-month data.

但這是一個重要的觀點。我們獲得了突破性療法認定。然後，我們與 FDA 的初步討論只是針對第三階段的初始 12 個月數據。

And since then, we've really strengthened the package, with very detailed immunology characterization that was published in February in Nature Communications. And then, what we think is very exciting: year 2 and year 3 durability data, where we saw that the clinical benefit that we saw in the first 12-month period continued on into the second 12 months and the third 12-month period; and, actually, improved during the second 12-month period.

從那時起，我們真正加強了這個方案，並在二月在《自然通訊》上發表了非常詳細的免疫學表徵。然後，我們認為非常令人興奮的是：第 2 年和第 3 年的耐久性數據，我們發現在第一個 12 個月期間看到的臨床益處持續到第二個 12 個月和第三個 12 個月期間；並且實際上在第二個 12 個月期間有所改善。

So I think we now have a very compelling package to put in front of the FDA.

所以我認為我們現在有一個非常有吸引力且可行的方案可以提交給 FDA。

That's an excellent point. As we've said on previous calls, we've actually got a three-year history of these patients. So we really can compare like-for-like.

這是一個很好的觀點。正如我們在之前的電話中所說的那樣，我們實際上已經掌握了這些患者三年的病史。因此我們確實可以進行同類比較。

And, as we talked about on the call, we're seeing a very significant and impressive reduction in surgeries. And that only, I think, becomes more impressive, when you look at these patient history.

而且，正如我們在電話中談到的那樣，我們看到手術數量顯著減少。我認為，當你查看這些病人的病史時，這一點才會變得更加令人印象深刻。

So all of that is now incorporated into our clinical modules.

所以所有這些現在都被納入了我們的臨床模組中。

Looking forward to the execution in the second half of this year.

期待今年下半年的執行。

Roger Song, Jefferies.

傑富瑞 (Jefferies) 的羅傑宋 (Roger Song)。

Two questions from us. One is it's very interesting for the year 2, 3 durability data from 3107. Given your initial approval will be based on the current data, how should we think about the pricing, as you continue to accumulate the durability data?

我們有兩個問題。首先，3107 的第 2 年和第 3 年的耐久性數據非常有趣。鑑於您的初步批准將基於當前數據，隨著您繼續累積耐用性數據，我們應該如何考慮定價？

Second question is related to the confirmatory study and then, on commercialization. What's the current thinking about the overall cost for the next steps? And then, how you will -- considering partnership or your own to fund the next steps?

第二個問題與驗證性研究以及商業化有關。目前對於下一步的整體成本有何考量？然後，您將如何—考慮透過合作或自己的方式來資助下一步？

Great. Thank you. They're excellent questions.

偉大的。謝謝。這些問題非常好。

Steve, do you want to comment on our thoughts on pricing for the initial treatment regimen?

史蒂夫，你想評論一下我們對初始治療方案定價的想法嗎？

Yeah. We've done quite a bit of research with payers. In fact, we've spoken with payers that represent about 70% of commercial lives in the US.

是的。我們對付款人進行了大量的研究。事實上，我們已經與代表美國商業生活的約 70% 的付款人進行了交談。

We've gone through the data with them, talked about price ranges in the rare disease space. Payers felt that that was appropriate, given the product, given the benefits.

我們與他們一起研究了數據，討論了罕見疾病領域的價格範圍。付款人認為，考慮到產品和好處，這樣做是合適的。

And the analogy that we've shared -- and we've mentioned this before -- SpringWorks Therapeutics product, OGSIVEO, for desmoid tumors we think is a good analogy. That product is priced at $360,000 per year and that's the guidance that we've provided around pricing and payers seem very open.

我們分享過的類比——我們之前提到過——SpringWorks Therapeutics 產品 OGSIVEO 對於纖維瘤來說，我們認為是一個很好的類比。該產品的價格為每年 36 萬美元，這是我們提供的定價指導，付款人似乎非常開放。

They think that's appropriate. We haven't commented or guided on pricing, in terms of redosing or continued treatment over time or duration. We haven't guided to that.

他們認為這是合適的。我們沒有對重新給藥或持續治療的定價做出評論或指導。我們還沒有對此進行指導。

Just, we've really focused on that initial four doses.

只是，我們真正關注的是最初的四劑。

So in terms of funding, in the US, we plan to bring 3107 to market, ourselves, in the US. We think, with the relatively small number of physicians that we'll need to reach, we'll be able to do that with a small and efficient field force.

因此，就資金方面而言，在美國，我們計劃自行將 3107 推向美國市場。我們認為，由於我們需要聯繫的醫生數量相對較少，因此我們能夠透過一支規模較小但高效的現場隊伍來實現這一目標。

Ex-US, certainly, we're very open to partnering to bring 3107 to market ex-US.

在美國以外，當然，我們非常願意合作將 3107 推向美國以外的市場。

Yi Chen, H.C. Wainwright.

陳毅，H.C.溫賴特。

Does the current tariff policy or the most favorable pricing (technical difficulty) -- or sales from?

現行關稅政策是否還是最優惠定價（技術難度）－或銷售如何？

Yeah. Clearly, this is a rapidly evolving situation. And I think we, like others, are waiting to see how this plays out.

是的。顯然，情況正在迅速變化。我想，我們和其他人一樣，正在等著看事情將如何發展。

But what I would say, at the moment, is we're very much focused on our first approval in the US. That's where our focus is.

但目前我想說的是，我們非常關注在美國的首次批准。這就是我們的重點。

So as a first, US launch, without a product available in another market; that would be something that we wouldn't have to face in those initial launch years. But, clearly, this is going to be very important for the entire sector.

因此，作為首次在美國推出的產品，如果其他市場上沒有產品，那麼在最初的幾年裡我們就不必面對這樣的問題。但顯然，這對整個產業來說都非常重要。

And we'll be paying close attention to this as it evolves.

我們將密切關注事態發展。

Gregory Renza, RBC Capital.

加拿大皇家銀行資本管理公司的 Gregory Renza。

This is [Mitch], on for Greg. We are wondering if you were planning on disclosing baseline characteristics for the patients in the confirmatory trial? And how might HPV genotype affect these trial results, based on the findings you disclosed in your Nature Communications paper?

我是 [Mitch]，代替 Greg。我們想知道您是否計劃在確認性試驗中揭露患者的基線特徵？根據您在《自然通訊》論文中揭露的研究結果，HPV 基因型可能如何影響這些試驗結果？

Yeah. Apologies. We couldn't hear the first part of your question. Could you repeat that?

是的。抱歉。我們聽不清楚您問題的第一部分。你能再說一次嗎？

We are wondering if you were planning on disclosing the baseline characteristics for the patients in the confirmatory trial, related to HPV genotype.

我們想知道您是否計劃揭露與 HPV 基因型相關的確認試驗患者的基線特徵。

I can't remember the details in there. But we've always talked about -- the HPV serotypes was actually very representative of what the normal RRP population is.

我不記得裡面的細節了。但我們一直在談論——HPV 血清型實際上非常能代表正常的 RRP 族群。

So there were -- just over 60% were HPV 6, 30% were HPV 11. And then, there were some patients who were co-infected.

因此，超過 60% 的患者感染的是 HPV 6，30% 的患者感染的是 HPV 11。然後，有一些患者被同時感染。

So exactly what you'd expect to see in the normal RRP population. I think that's in the paper somewhere. But it's been a while since I've read it.

這正是您所期望在正常的 RRP 人群中看到的。我想這在報紙的某處。但我已經很久沒讀過它了。

So that was the split in our Phase I/II trial, Mike. In terms of the confirmatory trial, we're going to try and recruit a patient population that's very representative of the normal RRP patient population, which is, as Mike said, predominantly HPV 6, skews male.

這就是我們 I/II 期試驗的分裂，麥克。在確認性試驗方面，我們將嘗試招募一個非常具代表性的正常 RRP 患者群體，正如 Mike 所說，該群體主要為 HPV 6，且以男性為主。

And so, we'll look to try and recruit the appropriate population; very similar to our Phase I/II, which we do think it was representative.

因此，我們將嘗試招募合適的人群；與我們的第一/第二階段非常相似，我們確實認為它具有代表性。

Yeah. Hopefully, with the targeted 100 patients, that will give us a greater chance to, once again, replicate what the actual RRP population is.

是的。希望透過這 100 名目標患者，我們可以有更大的機會再次複製實際的 RRP 族群。

There are no further ,questions at this time.

目前沒有其他問題。

I'd like to turn the call back to Jacque Shea for final closing comments.

我想將電話轉回給 Jacque Shea，請他發表最後的總結評論。

Thank you.

謝謝。

As I've outlined, here, today, we're making significant progress and remain focused on the catalysts ahead that will help us achieve our primary goals: submitting our BLA for 3107 and being prepared for a swift and efficient commercial launch, if approved.

正如我今天在這裡概述的那樣，我們正在取得重大進展，並將繼續關注未來的催化劑，這將有助於我們實現主要目標：提交 3107 的 BLA，並為迅速、高效的商業發布做好準備（如果獲得批准）。

I'm really excited about how 3107 could change the treatment paradigm for RRP and for the patients who could benefit from it.

我非常興奮 3107 如何改變 RRP 的治療模式以及從中受益的患者。

We're moving forward knowing that each day and each surgery matters to patients and our mission.

我們不斷前進，深知每一天、每場手術對病人和我們的使命都至關重要。

Thank you for your attention. Good evening, everyone.

感謝您的關注。大家晚上好。

Thank you.

謝謝。

Ladies and gentlemen, this concludes your call for today.

女士們、先生們，今天的通話到此結束。

We thank you for participating and ask that you please disconnect your lines.

感謝您的參與，並請您斷開線路。