Precigen Inc (PGEN) 2024 Q4 法說會逐字稿

Good evening, and welcome to the Precigen's Full Year 2024 Financial Results and Business Update Call. At this time, all lines are in listen-only mode. Following the prepared remarks, there will be a question-and-answer session. Please note that this event is being recorded.

晚上好，歡迎參加 Precigen 2024 年全年財務表現和業務更新電話會議。此時，所有線路都處於只聽模式。準備好的發言之後，將有一個問答環節。請注意，此事件正在被記錄。

I would now like to turn the conference over to Steve Harasym from Investor Relations. Please go ahead.

現在我想將會議交給投資者關係部門的史蒂夫·哈拉西姆 (Steve Harasym)。請繼續。

Thank you, Alan, and thank you for everyone joining us this afternoon. With me today are Dr. Helen Sabzevari, President and CEO of Precigen; Harry Thomasian, our CFO; Phil Tennant, our Chief Commercial Officer; and Rutul Shah, our Chief Operating Officer.

謝謝你，艾倫，也謝謝今天下午加入我們的所有人。今天與我一起的有 Precigen 總裁兼執行長 Helen Sabzevari 博士、財務長 Harry Thomasian、首席商務長 Phil Tennant 和營運長 Rutul Shah。

Before we begin, let me briefly review our forward-looking statements. During today's call, we will make various forward-looking statements. Investors are cautioned that our forward-looking statements are based on current expectations and are subject to risks and uncertainties that could cause actual results or outcomes to differ from those indicated by our forward-looking statements. Please read the Safe Harbor statement contained in our most recent SEC filings as well as the risk factors contained in Precigen's filings.

在我們開始之前，讓我先簡單回顧一下我們的前瞻性陳述。在今天的電話會議中，我們將做出各種前瞻性陳述。請投資人注意，我們的前瞻性陳述是基於當前預期，並受風險和不確定性的影響，這些風險和不確定性可能導致實際結果與我們的前瞻性陳述所示的結果不同。請閱讀我們最近的 SEC 文件中包含的安全港聲明以及 Precigen 文件中包含的風險因素。

With that, I would like to now turn the call over to Dr. Sabzevari. Helen?

現在，我想將電話轉給 Sabzevari 博士。海倫？

Thank you, Steve, and thank you to all for taking the time to join us for our year end 2024 update. It is indeed a transformative time for at Precigen and as we are on the verge of commercializing our lead asset, PRGN-2012 in RRP, potentially bringing a treatment to this patient population with high unmet needs.

謝謝你，史蒂夫，也謝謝大家抽出時間來參加我們的 2024 年年終更新。對於 Precigen 來說，這確實是一個變革時期，我們即將在 RRP 中商業化我們的主要資產 PRGN-2012，這可能會為這些具有大量未滿足需求的患者群體帶來治療。

From discovery in 2020, Phase 1 initiation in 2021, Phase 2 with breakthrough designation, an accelerated approval pathway in 2023, followed by publication in science translation and presentation of the groundbreaking data at ASCO in 2024, this program has advanced with a remarkable efficiency and agility.

從 2020 年的發現、2021 年啟動第一階段、第二階段突破性認定、2023 年加速審批途徑，到 2024 年在科學翻譯中發表並在 ASCO 上展示突破性數據，該計劃以驚人的效率和靈活性不斷推進。

We finished 2024 with submission of our BLA and announced FDA's acceptance with priority review with an upcoming PDUFA date of August 27, 2025. The FDA has indicated that they are not currently planning to hold an advisory committee meeting to discuss the BLA.

我們在 2024 年提交了 BLA，並宣布獲得 FDA 的優先審查，PDUFA 的截止日期為 2025 年 8 月 27 日。FDA 表示他們目前不打算召開諮詢委員會會議來討論 BLA。

I would like to now take some time, a few minutes, to recap of our data, which actually recently has come out in Lancet publication. We are extremely excited about PRGN-2012 potential in RRP to not only be the first but the best in the class treatment. Due to significant effect in terms of efficacy, safety, the ease of route of administrations.

現在我想花幾分鐘時間來回顧我們的數據，這些數據實際上最近已在《柳葉刀》出版物上發表。我們對 PRGN-2012 在 RRP 中的潛力感到非常興奮，它不僅是同類治療中的首個，而且是最好的。由於療效顯著、安全性高、給藥途徑簡便。

In our pivotal clinical data, PRGN-2012 demonstrated a statistically significant efficacy. I want to emphasize that our clinical study was designed with a robust, clinically meaningful, and most importantly, a prospectively defined statistical primary efficacy endpoints of complete responses in RRP patients.

在我們的關鍵臨床數據中，PRGN-2012 表現出了具有統計意義的療效。我想強調的是，我們的臨床研究設計具有穩健性、臨床意義，最重要的是前瞻性地定義了 RRP 患者完全緩解的統計主要療效終點。

Our pivotal study met primary efficacy endpoint with 51% complete response rate. That was a statistically significant and handling beat pre-specified success criteria established in alignment with FDA. Complete responses have been durable with median durability of response at 24 months, and all of our Phase 1 complete responders remain surgery free and in complete response three years.

我們的關鍵研究達到了主要療效終點，完全緩解率為 51%。這具有統計意義，且處理超出了與 FDA 一致制定的預先指定的成功標準。完全緩解具有持久性，平均緩解持久性為 24 個月，並且我們所有第 1 階段完全緩解患者在三年內無需進行手術即可保持完全緩解。

This is highly significant and I'm going to stress that the data that was presented at ASCO showing collectively Phase 1 and Phase 2,it has such a close repeats that in our Phase 1, we had 50% complete responders and in Phase 2 52% for the average of 51% complete response in a prospective manner.

這非常重要，我要強調的是，ASCO 上展示的數據共同顯示了第 1 階段和第 2 階段的結果，其重複性非常接近，在第 1 階段，我們有 50% 的完全緩解者，而在第 2 階段，我們有 52% 的完全緩解者，平均而言，以前瞻性的方式，完全緩解率為 51%。

Furthermore, 86% of all of our patients had reduction in the number of surgeries. In multiple publications, we have also shown significant enhancement of HPV 6 and or HPV 11 T-cell responses, which is directly corresponds to the mechanism of action of PRGN-2012 and our clinical responses, and the first set of data was published actually in 2023 in science translation in regard to this.

此外，我們有86%的患者手術次數減少。在多篇出版物中，我們也展示了 HPV 6 和/或 HPV 11 T 細胞反應的顯著增強，這與 PRGN-2012 的作用機制和我們的臨床反應直接相對應，第一組數據實際上於 2023 年在《科學翻譯》中發表。

Also, we have shown that PRGN-2012, it's again because of the differentiation that the gorilla adenovirus has shown it can be repeatedly dosed, not only in our current program of PRGN-2012, which there has been four doses but also in our other programs such as PRGN-2009, which has been dosed more than 20 times.

此外，我們已經證明 PRGN-2012 可以重複注射，這再次證明了大猩猩腺病毒的分化，不僅在我們目前的 PRGN-2012 項目中（已有四劑），而且在我們的其他項目中（例如 PRGN-2009），已註射超過 20 次。

This platform is different than other AAVs or other viral platform that they have a limited amount of time that you can dose them, for instance, once and twice. Gorilla adenovirus it can be repeat dosed and continues to lead to the enhancement of the T-cells in the absence of neutralizing antibodies, significant neutralizing antibodies.

該平台與其他 AAV 或其他病毒平台不同，它們的給藥時間有限，例如一次或兩次。大猩猩腺病毒可以重複給藥，並在沒有中和抗體的情況下繼續導致 T 細胞增強，產生顯著的中和抗體。

And finally, I would like to say that the PRGN-2012, based on all the data that has been shown and the follow-ups, is extremely well tolerated with no dose-limiting toxicity, no treatment-related adverse event greater than Grade 2.

最後，我想說的是，根據已顯示的所有數據和後續情況，PRGN-2012 的耐受性極好，沒有劑量限制性毒性，也沒有高於 2 級的治療相關不良事件。

And as we have already shown, the route of the administration is just a simple subcute administration that can be given at any office. Well, we also have continued with our readiness to hit the ground running post our PDUFA date in August of 2025 as we shift from R&D to commercialization, and Phil, our Chief of Commercials would be speaking to that.

正如我們已經表明的，管理途徑只是一種簡單的亞臨床管理，可以在任何辦公室進行。好吧，我們也將繼續做好準備，在 2025 年 8 月 PDUFA 日期之後從研發轉向商業化，立即開始工作，我們的商業總監 Phil 將會談到這一點。

Finally, I would say a few words about our confirmatory trial, which already has been initiated and has started enrolling patients. Our confirmatory trial in alignment with the FDA is a single-arm no placebo control required with 35 patients to be enrolled, and we are well on our way. Not only we have started and enrolling patients but to move very rapidly in finishing this trial.

最後，我想談談我們的確認性試驗，該試驗已經啟動並開始招募患者。我們與 FDA 合作進行的確認試驗是單臂試驗，無需安慰劑對照，共招募 35 名患者，目前進展順利。我們不僅已經開始招募患者，而且還將非常迅速地完成這項試驗。

So with that, I will now turn the call to Rutul Shah, our Chief Operation Officer, which will be speaking to our CMC and manufacturing readiness. Rutul?

因此，我現在將電話轉給我們的營運長 Rutul Shah，他將談論我們的 CMC 和製造準備。魯圖爾？

Thank you, Helen. I would like to take a few moments to discuss our manufacturing strategy and provide an update. As you are aware, we have made significant commitment and investment to be in control of our GMP manufacturing activities.

謝謝你，海倫。我想花點時間討論一下我們的製造策略並提供最新資訊。如您所知，我們已做出重大承諾和投資來控制我們的 GMP 製造活動。

With that strategic goal in mind, we had built an in-house dedicated GMP facility for our adenovirus drug substance manufacturing back in 2019. We then manufactured all PRGN-2012 drug substance slots utilized in our clinical trials at this facility. Now, we have upgraded the facility, and with a strong leadership and operations team, we are ready to support the commercial launch of PRGN-2012.

本著這個策略目標，我們早在 2019 年就為腺病毒藥物物質生產建立了內部專用 GMP 設施。然後，我們在該設施生產了臨床試驗中使用的所有 PRGN-2012 藥物物質槽。現在，我們已經升級了設施，並擁有強大的領導和營運團隊，我們已準備好支援 PRGN-2012 的商業發布。

In addition, we utilized an established commercial CDMO for fill finish operations for the PRGN-2012 drug product. We have also built significant GMP quality control capabilities in-house to support release testing of PRGN-2012.

此外，我們利用成熟的商業 CDMO 進行 PRGN-2012 藥品的填充完成操作。我們還在內部建立了重要的 GMP 品質控制能力，以支援 PRGN-2012 的發布測試。

Together, our GMP manufacturing and testing strategy is designed to give us better control over more specialized operations, overall timelines and provide independence from external vendors as much as practical while containing costs. As part of the PRGN-2012 BLA, we had completed the process validations as previously aligned with the FDA. We are confident in our ability to supply PRGN-2012 product to meet anticipated demand at launch and beyond.

總而言之，我們的 GMP 製造和測試策略旨在讓我們更好地控制更專業化的營運和整體時間表，並在控製成本的同時盡可能地獨立於外部供應商。作為 PRGN-2012 BLA 的一部分，我們已經完成了先前與 FDA 保持一致的流程驗證。我們有信心提供 PRGN-2012 產品以滿足發佈時及之後的預期需求。

With that, I'd like to now hand over to Phil Tennant, our Chief Commercial Officer, to provide update on PRGN-2012 market opportunity and our commercialization strategy. Phil?

現在，我想將時間交給我們的商務長 Phil Tennant，以提供有關 PRGN-2012 市場機會和我們的商業化策略的最新資訊。菲爾？

Great. Thank you, Rutul. Good afternoon, everyone. The past few months have been incredibly dynamic for our commercialization team as we advanced preparations for the commercial launch around the August 27 PDUFA. And as Helen said, this is a pivotal milestone for us as an organization and I'm excited to share the progress that we've made since our last update.

偉大的。謝謝你，魯圖爾。大家下午好。過去幾個月對我們的商業化團隊來說是異常活躍的幾個月，我們為 8 月 27 日 PDUFA 前後的商業發布做好了充分的準備。正如海倫所說，這對我們組織來說是一個關鍵的里程碑，我很高興分享我們自上次更新以來的進展。

So, we've made some significant strides in our launch readiness, including several major accomplishments. Firstly, we've completed the build-out of our Precigen commercial leadership team. We have very strong and experienced team now across sales, marketing, medical affairs, and market access and distribution who have the experience and the capabilities to guide the launch with precision and impact.

因此，我們在發布準備方面取得了一些重大進展，包括幾項重大成就。首先，我們已經完成了 Precigen 商業領導團隊的組建。我們現在擁有一支非常強大且經驗豐富的團隊，涵蓋銷售、行銷、醫療事務以及市場准入和分銷，他們擁有精準且有影響力地指導產品發布的經驗和能力。

Secondly, we've established a comprehensive commercialization strategy for the US launch and this spans all facets of our operations to ensure we hit the ground running at launch. And then in terms of our broader commercial infrastructure, we're delighted to announce our partnership with EVERSANA to implement the US launch.

其次，我們為美國市場的發布製定了全面的商業化策略，涵蓋了我們營運的各個方面，以確保我們在發佈時能夠順利進行。就我們更廣泛的商業基礎設施而言，我們很高興宣布與 EVERSANA 合作實施美國市場推廣。

As a proven leader in supporting rare disease launches, EVERSANA brings a wealth of experience that complements our vision for PRGN-2012. Together, we are executing on critical launch activities across the spectrum, including the training and deployment of dedicated field teams to ensure a seamless and impactful launch.

作為支持罕見疾病上市領域公認的領導者，EVERSANA 擁有豐富的經驗，這與我們對 PRGN-2012 的願景相輔相成。我們共同執行各領域的關鍵發射活動，包括培訓和部署專門的現場團隊，以確保無縫且有影響力的發射。

Our updated analytics underscore the importance of this work. Our advanced analysis, as shared at JP Morgan, suggests that up to 27,000 adult patients in the US are living with RRP, which is higher than the previous estimates of up to 20,000 and indicative of an even greater unmet need. These data strengthen our resolve to launch PRGN-2012 with a patient-centric focus ensuring timely access to this transformative treatment.

我們最新的分析強調了這項工作的重要性。正如摩根大通所分享的，我們的高級分析表明，美國有多達 27,000 名成年患者患有 RRP，這一數字高於先前估計的 20,000 人，表明未滿足的需求更大。這些數據堅定了我們以患者為中心推出 PRGN-2012 的決心，確保患者能夠及時獲得這種變革性治療。

So in summary, we're moving decisively towards launch driven by an experienced leadership team, a highly capable commercialization partner, and a robust strategy. And these elements position us well to realize the full potential of PRGN-2012 with commercial revenues expected to begin in the second half of 2025. We are primed to hit the ground running given our PDUFA date and that would clearly position us as the first and only medical treatment available for these patients.

總而言之，在經驗豐富的領導團隊、能力超強的商業化合作夥伴和強而有力的策略的推動下，我們正果斷地邁向上市。這些因素使我們能夠充分發揮 PRGN-2012 的潛力，預計商業收入將在 2025 年下半年開始。鑑於我們的 PDUFA 日期，我們已準備好立即開始行動，這將明確使我們成為這些患者可用的第一個也是唯一一個醫療機構。

Back to you, Helen.

回到你身邊，海倫。

Thank you, Phil. Well, as you can see that our level of excitement among Precigen and our teams as obviously PRGN-2012 has been our highest priority and remains to be our highest priority as we take and transform the organization from R&D to the commercial organization.

謝謝你，菲爾。好吧，正如您所看到的，Precigen 和我們的團隊都非常興奮，因為 PRGN-2012 顯然一直是我們的最高優先事項，並且在我們將組織從研發轉變為商業組織時仍將是我們的最高優先事項。

With that in mind, however, I would like to also give you an update on two other programs that has been very exciting and very important. The first one is our PRGN-2009, which is our gorilla adenovirus drug product that targets HPV-16 and HPV-18, which is the lead cause of HPV-related cancers, a total of 5% of all cancers are HPV-related and that includes cervical cancers, head and neck, and anal cancerous indications.

不過，考慮到這一點，我還想向大家介紹另外兩個非常令人興奮且非常重要的專案的最新進展。第一個是我們的 PRGN-2009，這是我們的大猩猩腺病毒藥物產品，針對的是 HPV-16 和 HPV-18，這是 HPV 相關癌症的主要原因，所有癌症中有 5% 與 HPV 相關，其中包括子宮頸癌、頭頸癌和肛門癌。

Our PRGN-2009 have been targeting HPV-16 and HPV-18 and we presented the data the Phase 1 data at ASCO in 2023. We showed not only a very favorable safety and tolerability profile but more importantly, in relapsed-refractory patients that they had failed even all the checkpoint inhibitors.

我們的 PRGN-2009 一直針對 HPV-16 和 HPV-18，我們在 2023 年在 ASCO 上展示了第 1 期資料。我們不僅展示了非常有利的安全性和耐受性，更重要的是，對於復發難治性患者，他們甚至所有檢查點抑制劑都失敗了。

We were able to show for the first time for any drug products in this setting, 30% objective responses. Complete responders as well as partial responders. We had our complete responders spanning close to two years of response.

我們首次能夠證明，在這種環境下，任何藥品的客觀反應率都達到 30%。完全響應者以及部分響應者。我們擁有完整的回應人員，回應時間長達近兩年。

And in these same patients, we have shown and some of them have received excess of 20 doses of PRGN-2009, which has the exact same backbone as PRGN-2012, and have shown not only the neutralizing antibodies are at bay, and not increasing at all, but also clear enhancement of T-cell responses upon redosing of these patients.

在這些相同的患者中，我們已經證明，其中一些患者已經接受了超過 20 劑的 PRGN-2009（其主鏈與 PRGN-2012 完全相同），並且不僅證明中和抗體得到了抑制，根本沒有增加，而且這些患者再次服藥後 T 細胞反應也明顯增強。

So this clearly this clinical data in conjunction with our immunological data points again to the differentiation of our gorilla adenovirus platform than any other platforms. And currently, our PRGN-2009 is advancing at Phase 2, both for cervical cancer and head and neck at NCI, and we continue to expand on that and we will be looking forward in near future to be giving updates on these two programs.

因此，這些臨床數據與我們的免疫學數據相結合，再次清楚地表明了我們的大猩猩腺病毒平台與任何其他平台的差異。目前，我們的 PRGN-2009 正在 NCI 進行第 2 階段研究，用於治療子宮頸癌和頭頸癌，我們將繼續擴展該研究，並期待在不久的將來公佈這兩個項目的最新進展。

At the same time, I'd like to give you an update on our UltraCAR-T platform. As you are aware, our UltraCAR-T platform to our knowledge, is genuinely the only CAR-T platform that can deliver autologous CAR-T of the patients overnight at the setting of a hospital without a centralized manufacturing.

同時，我想向您介紹我們的 UltraCAR-T 平台的最新情況。如您所知，據我們所知，我們的 UltraCAR-T 平台是真正唯一能夠在醫院環境中隔夜為患者提供自體 CAR-T 且無需集中製造的 CAR-T 平台。

It's knee-centralized manufacturing, it's overnight and it's autologous, it contains the car of the interest plus a safety switch, which becomes very important. If anything goes wrong, you can eliminate these cells immediately at the same token with the mechanism of membrane-bound IL-15, which allows actually the persistent and expansion of these cells in a way, the manufacturing of these cells directly in vivo in patients.

它是膝蓋集中製造的，它是一夜之間發生的，它是自體的，它包含感興趣的汽車加上一個安全開關，這變得非常重要。如果出現任何問題，您可以立即消除這些細胞，同時採用膜結合 IL-15 的機制，這實際上允許這些細胞以某種方式持續存在和擴增，直接在患者體內製造這些細胞。

And you do not need in vitro expansion of these cells. Last year, we communicated that we had finished our Phase 1b in AML patients and we also communicated that we are preparing for the presentation for the end of Phase 1b to FDA. Meanwhile, at JP Morgan, we presented further data and quite exciting data that in our belief, it's the first time ever that any company has shown this.

而且你不需要在體外擴增這些細胞。去年，我們告知我們已完成針對 AML 患者的 1b 期試驗，並且我們也告知我們正在準備向 FDA 提交 1b 期試驗結束的報告。同時，在摩根大通，我們展示了進一步的數據和相當令人興奮的數據，我們相信，這是有史以來任何公司第一次展示這些數據。

For the first time, we have developed and discovered a specific biomarkers for the AML patients in our responders versus non-responders after the treatment with the CAR-Ts. And we have shown part of that data at JP Morgan, and I highly encourage people to take a look at that presentation if they have not seen that.

我們首次開發並發現了針對接受 CAR-T 治療後對 AML 患者有反應和無反應的患者的特定生物標記。我們已經在摩根大通展示了部分數據，如果大家還沒看過，我強烈建議大家看一下這個簡報。

We are currently very much excited about putting these material together and we are preparing for our meetings with the FDA to discuss the not only the platform but also our AML data and also to discuss the strategy for a pivotal Phase 2 and a path for approval and starting with the AML. Please stay tuned. We will be giving you update on that level as well.

我們目前非常高興能夠將這些材料整合在一起，我們正在準備與 FDA 會面，不僅討論平台，還討論我們的 AML 數據，並討論關鍵的第 2 階段的策略以及獲得批准和從 AML 開始的途徑。敬請關注。我們也將向您提供該級別的最新資訊。

And finally, I would like to also mention that our UltraCAR-T platform has been generating a very exciting data in an autoimmune setting. And as you know, the requirements for autoimmune settings are you have to have a safe course that you can be actually generated with the price tag that can be used in number of dosing.

最後，我還想提一下，我們的 UltraCAR-T 平台在自體免疫環境中產生了非常令人興奮的數據。如您所知，自體免疫設定的要求是您必須有一個安全的療程，並且您實際上可以透過可用於劑量數量的價格標籤來產生該療程。

And our CAR-T platform meets all of that. Including with safety switches and also the next generation adjustments that it does not require any use of checkpoint inhibitors in them. And with that data, we believe that in the field of autoimmunity, not only we have the ability to be first in the class, but also the best in the class. And this is ongoing and we are very excited about this.

我們的 CAR-T 平台滿足了所有這些要求。包括安全開關以及下一代調整，它不需要使用任何檢查點抑制劑。有了這些數據，我們相信，在自體免疫領域，我們不僅有能力成為同類中的佼佼者，而且能夠成為同類中的佼佼者。這項工作仍在進行中，我們對此感到非常興奮。

So with that update, I would like now to hand over to our CFO, Harry. Harry?

有了這些更新，我現在想把時間交給我們的財務長 Harry。哈利？

Thanks very much, Helen, and welcome to those participating in the call today. This really is an exciting time at Precigen as we prepare for the launch of our first commercial drug product. You've already heard from other members of the Precigen team as to our preparedness toward this goal, and I'd like to spend some time discussing our financial position as we move toward commercialization.

非常感謝，海倫，歡迎今天參加電話會議的各位。這對 Precigen 來說確實是一個激動人心的時刻，因為我們正準備推出我們的第一個商業藥品。您已經從 Precigen 團隊的其他成員那裡聽說了我們為實現這一目標所做的準備，我想花一些時間討論一下我們在走向商業化過程中的財務狀況。

I'll begin by highlighting our financial results for 2024. Overall, we finished the 2024 year with a net loss of $126.2 million or $0.47 per basic and diluted share compared to a net loss of $95.9 million or $0.39 per basic and diluted share in the year ended, December 31, 2023. I'd like to point out that our current year net loss included over $55 million of non-cash charges net.

首先，我將重點放在我們 2024 年的財務表現。整體而言，2024 年我們的淨虧損為 1.262 億美元，即每股基本虧損和稀釋虧損均為 0.47 美元，而截至 2023 年 12 月 31 日的年度淨虧損為 9,590 萬美元，即每股基本虧損和稀釋虧損均為 0.39 美元。我想指出的是，我們今年的淨虧損包括超過 5,500 萬美元的非現金費用淨額。

Our cash burn for 2024, consisting of cash used in operations plus capital expenditures totaled $76.8 million. Our press release and management's discussion and analysis included in our 10-K, both of which were just filed with the SEC, provide further detail as to fluctuations in our statement of operations between 2024 and 2023.

我們 2024 年的現金消耗（包括營運所用現金加上資本支出）總計 7,680 萬美元。我們的新聞稿以及 10-K 文件中包含的管理層討論和分析（兩者都剛提交給美國證券交易委員會）提供了有關 2024 年至 2023 年期間營運報表波動的更多詳細資訊。

With that summary of our operating results, I'd like to spend a little time focused on our financial position as I believe most of you listening in are aware, we raised $79 million at the end of 2024 via a preferred stock issuance, which included the issuance of warrants to purchase common stock.

在總結我們的經營業績之後，我想花一點時間關註一下我們的財務狀況，因為我相信大多數聽眾都知道，我們在 2024 年底透過發行優先股籌集了 7,900 萬美元，其中包括發行購買普通股的認股權證。

We believe that the terms of this preferred share instrument are friendly to the company. The preferred stock carries an 8% dividend of which the first two years are to be paid in kind, beginning with the third year, while dividends will be payable in cash, the payment of such are due, when and if declared by Precigen's Board of Directors. While the preferred shares are convertible into common stock, the conversion feature resets each quarter, thus reducing the dilutive effect of a potential conversion as our stock price increases.

我們相信該優先股工具的條款對公司有利。優先股的股息為 8%，其中前兩年將以實物形式支付，從第三年開始，股息將以現金形式支付，這些股息的支付應在 Precigen 董事會宣佈時進行。雖然優先股可以轉換為普通股，但轉換功能每季都會重置，隨著股價上漲而減少潛在轉換的稀釋效應。

In addition, the company has the ability to redeem the preferred shares for the stated value plus accumulated and unpaid dividends, while the holders do not have the ability to put the shares to the company.

此外，公司有權依規定價值加上累積未付股息贖回優先股，而持有人無權將股份出售給本公司。

You can find more detailed summary of the terms of the preferred stock issuance in the financial statements in our Form 10-K just filed with the SEC or within our 8-K announcing the transaction that was filed with the SEC on December 30th of 2024.

您可以在我們剛剛向美國證券交易委員會提交的 10-K 表格中的財務報表或我們於 2024 年 12 月 30 日向美國證券交易委員會提交的交易公告 8-K 表格中找到有關優先股發行條款的更詳細摘要。

On top of the funds received from the preferred stock issuance, we also monetized in a non-dilutive manner certain intellectual property rights and royalty rights related to non-core assets of the company in December 2024, which resulted in proceeds of $8.5 million.

除了發行優先股所獲得的資金外，我們還於 2024 年 12 月以非稀釋的方式將公司非核心資產相關的某些智慧財產權和特許權使用費貨幣化，獲得了 850 萬美元的收益。

The preferred issuance plus the aforementioned sale of intellectual property rights and royalty rights helped shore up our balance sheet and we finished the year with cash, cash equivalents, and investments of $97.9 million. We're confident that this balance will support us well beyond our anticipated launch date and well into 2026. This runway is based on current projections, which includes anticipated revenue from the commercialization of PRGN-2012.

優先股發行加上前面提到的智慧財產權和特許權使用費的出售幫助我們鞏固了資產負債表，我們在年底的現金、現金等價物和投資總額為 9,790 萬美元。我們相信，這種平衡將支持我們超越預期的發布日期並順利進入 2026 年。該跑道基於當前預測，其中包括 PRGN-2012 商業化的預期收入。

As Helen mentioned earlier, our BLA was accepted by the FDA in February under a priority review with a PDUFA target action date set for August 27 of 2025. This target action date plus the preparedness of our commercial team as you heard from Phil earlier, provides me with a level of comfort to include revenue in my projections. Although from an accounting perspective, this anticipated revenue is considered outside of our direct control as that revenue is dependent upon the successful FDA approval of the PRGN-2012 BLA.

正如海倫之前提到的，我們的 BLA 於 2 月被 FDA 接受，並接受優先審查，PDUFA 目標行動日期定為 2025 年 8 月 27 日。正如您之前從菲爾那裡聽到的，這個目標行動日期加上我們商業團隊的準備情況，讓我有一定程度的信心將收入納入我的預測中。但從會計角度來看，這項預期收入被認為超出了我們的直接控制範圍，因為該收入取決於 FDA 是否成功批准 PRGN-2012 BLA。

In closing, I want to reiterate that Precigen has always shown a strong financial discipline and we will continue to do so in the future. Our focus has and always will be to utilize our resources to garner the highest value for our shareholders.

最後，我想重申，Precigen 一直表現出強大的財務紀律，我們將來也將繼續這樣做。我們的重點一直是、並且永遠是利用我們的資源為股東創造最高價值。

With that, I'd like to open up the call for Q&A and turn it over to Alan, the operator.

現在，我想開始問答環節，並將時間交給接線員艾倫 (Alan)。

(Operator Instructions) Jason Butler of Citizens.

（操作員指示）公民 Jason Butler。

congrats on the progress. Two for me. One, just at a high level, can you give us an update on the interactions with FDA and just how would you characterize the ongoing review for 2012? And then just on the commercial side, can you speak to what your current thoughts are on the size of the field force and the number of centers that you'll be targeting during the initial launch? Thank you.

祝賀你取得進展。對我來說是兩個。首先，您能否從高層次向我們介紹與 FDA 互動的最新情況，以及您如何描述 2012 年正在進行的審查？然後從商業方面來說，您能否談談您目前對實地人員規模以及首次發布期間所針對的中心數量的看法？謝謝。

Hi, Jason. Thank you for the questions. So, in regard to our BLA submission and actually receiving a priority review, we have been having, as we have mentioned throughout there are submissions as well as even before that, very, very close interaction and alignments with the FDA.

你好，傑森。謝謝您的提問。因此，關於我們的 BLA 提交和實際獲得優先審查，正如我們一直提到的那樣，我們一直在與 FDA 進行提交，甚至在此之前，我們也在與 FDA 進行非常密切的互動和協調。

And we are very thankful to the FDA for a close interaction and the guidances and continue to do so. We are, I think, in a very good position in our submissions of the BLA and discussions and we continue to do so as you can imagine throughout the review cycle. So currently, of course, we stay very close in alignment with the FDA. And in regard to the commercial, I think maybe Phil can address this question.

我們非常感謝 FDA 的密切互動和指導，並將繼續這樣做。我認為，我們在提交 BLA 和討論方面處於非常有利的地位，正如您所想像的那樣，在整個審查週期中，我們將繼續保持這種狀態。因此，目前我們當然與 FDA 保持著密切的聯繫。關於商業廣告，我想菲爾也許可以回答這個問題。

Yeah, sure. There were a couple of paths to that. So, regarding the size of the field force, we've been fairly consistent in our thinking that we believe it's somewhere in the range of 15 to 20 representatives in the field and that hasn't changed in terms of what we're thinking.

是的，當然。有幾條路可以實現這一目標。因此，關於實地部隊的規模，我們的想法一直相當一致，我們認為實地代表人數在 15 到 20 人之間，而且我們的想法沒有改變。

So, a fairly modest sales team. We believe there are around 500 fellowship trained otolaryngologists out there who will be responsible for treating the bulk of the patients and those will largely be concentrated in the urban academic centers and the large IDNs and that will be an important part of our targeting.

所以，這是一個相當溫和的銷售團隊。我們相信，大約有 500 名接受過專科培訓的耳鼻喉科醫生將負責治療大部分患者，這些醫生主要集中在城市學術中心和大型 IDN，這將是我們目標的重要組成部分。

Great. Thank you very much.

偉大的。非常感謝。

Ben Burnett of Stifel.

Stifel 的 Ben Burnett。

Hello, and this is Carolina Ibanez Ventoso on for Ben Burnett. Thank you for taking our questions. And congratulations on for all the progress. Do you have any line of sight at this point on the timing of any additional meetings with the FDA, pre-licensing inspections and labeling discussions during the review process? I have a follow-up as well.

大家好，我是 Carolina Ibanez Ventoso，為 Ben Burnett 播報。感謝您回答我們的問題。恭喜您所取得的所有進步。目前，您是否了解在審查過程中與 FDA 進行任何額外會議、許可前檢查和標籤討論的時間表？我也有後續行動。

Thank you for the questions. Of course, as part of the regulatory process, there are inspections for facilities and because of that, we will not be making any comment, but this is typical, especially for GMP facilities, as I mentioned.

謝謝您的提問。當然，作為監管流程的一部分，我們會對設施進行檢查，因此我們不會發表任何評論，但這是典型的情況，特別是對於 GMP 設施，正如我所提到的。

We have been always in very close alignment and continue to be with the FDA and we look forward to further communication as obviously we get closer to our PDUFA date.

我們一直與 FDA 保持著密切的聯繫，並將繼續保持這種聯繫，隨著我們距離 PDUFA 日期越來越近，我們期待進一步的溝通。

Okay. Thank you. And Tom, if PRGN-2012 gets approved, do you anticipate there will be a bolus of patients? And how do you plan to position yourself to address it?

好的。謝謝。湯姆，如果 PRGN-2012 獲得批准，您是否預計會有大量患者接受該療法？您打算如何解決這個問題？

Yeah, great question. I mean, we absolutely do think there's pent-up demand here. These patients have known have had no other options really are on label at all. It's only surgery and some off-label treatments, which don't under don't address the underlying infection.

是的，很好的問題。我的意思是，我們確實認為這裡有被壓抑的需求。這些患者已經知道他們沒有其他選擇，實際上根本沒有標籤上的選擇。這只是手術和一些非說明書規定的治療方法，並不能解決潛在的感染。

So, absolutely, we believe that there will be a concentration of patients who are in desperate need of this type of medication when we get to market. And so we will be deploying our team and our efforts accordingly to make sure that as many of those patients appropriately get our treatment as soon as possible.

因此，我們絕對相信，當我們的產品進入市場時，將會有大量患者迫切需要這種藥物。因此，我們將相應地部署我們的團隊和努力，以確保盡可能多的患者盡快得到適當的治療。

Understood. Thank you very much.

明白了。非常感謝。

Thank you, Carol.

謝謝你，卡羅爾。

Swayampakula Ramakanth of H.C. Wainright.

H.C. 的斯瓦安帕庫拉·拉瑪坎特 (Swayampakula Ramakanth)溫賴特。

Thank you. This is RK from H.C. Wainwright. Good afternoon, Helen, Phil, Rutul, and Steven. So in terms of the indication itself, Phil, you are saying that based on you're looking at the claims, the number seems to be higher and in the order of 27,000 in the United States and probably around 125,000 outside US.

謝謝。這是來自 H.C. Wainwright 的 RK。下午好，海倫、菲爾、魯圖爾和史蒂文。因此，就跡象本身而言，菲爾，您說根據您所看到的索賠情況，這個數字似乎更高，在美國約為 27,000 人，在美國以外可能約為 125,000 人。

But that is with no therapy no approved therapy, as you said, and only patients who are brave enough to go under the knife. So, with an approved drug, how easy two things. How easy is it for patients to get diagnosed that they do have RRP?

但正如您所說，這是沒有治療方法，沒有批准的治療方法，只有勇敢接受手術的患者才能接受治療。因此，有了批准的藥物，兩件事就變得容易了。患者是否容易被診斷出患有 RRP？

And number two, how are you thinking of trying to get those patients or approach those patients who have not yet been diagnosed or is there a mechanism by which you can actually get these patients diagnosed correctly?

第二，您打算如何嘗試取得這些患者或接觸那些尚未確診的患者，或者是否有一種機制可以讓這些患者得到正確的診斷？

Yeah, great. Great. Let me take the first part, RK. So, the patients are very visible. They're having surgeries. They have many of them have frequent surgeries on an annual basis. So we believe a lot of those patients are going to be readily identifiable at launch.

是的，很棒。偉大的。讓我來談談第一部分，RK。因此，病人非常顯眼。他們正在做手術。他們中的許多人每年都要接受多次手術。因此我們相信，很多患者在推出時就很容易被辨識。

And obviously, that's a key area for us to focus on. Like all good launches, we will have personal and non-personal promotion, we will have remote promotion available. And so where we can't reach everyone with a face to face interaction, we will be able to provide information and direct people to the appropriate information for their patients.

顯然，這是我們關注的重點領域。與所有成功的產品發布一樣，我們將進行個人和非個人推廣，並提供遠端推廣。因此，即使我們無法與每個人進行面對面的交流，我們也能夠提供資訊並指導人們獲取適合其患者的資訊。

But the focus is definitely on those who are already visible because we think that will be the bulk of the patients actually that are out there at the moment. The other phenomenon I would say that we have seen in other rare diseases is when you start to get a new treatment in a rare disease area, the amount of diagnosed patients or presenting patients actually increases because of the enhanced awareness, understanding and education out there. We've seen that in a number of other rare diseases and we don't think this will be any different.

但重點肯定是那些已經可見的患者，因為我們認為他們實際上是目前大部分患者。我想說的是，我們在其他罕見疾病中看到的另一種現像是，當你開始在罕見疾病領域接受新的治療時，由於人們的認識、理解和教育的增強，確診患者或就診患者的數量實際上會增加。我們已經在許多其他罕見疾病中看到過這種情況，我們認為這也不會有什麼不同。

Okay. And maybe RK, this is Helen. I can also add to what Phil mentioned. In regard to the patient, obviously, identifications, currently the adult patients that they have and they live with the RRP, this is a patient population that is at the target and these patients are highly themselves are involved in regard to the finding a new innovative treatment because as you know, for almost the past 50 years, this disease has been around and unfortunately, there has been no treatment for this disease except the continuous surgery, which is just a band-aid in a sense.

好的。也許是 RK，這是海倫。我還可以補充 Phil 提到的內容。關於患者，顯然，目前他們所治療的成年患者與 RRP 一起生活，這是目標患者群體，這些患者高度參與尋找新的創新治療方法，因為如你所知，近 50 年來，這種疾病一直存在，不幸的是，除了持續手術外，沒有其他治療方法，而從某種意義上說，這只是一個創可貼。

And from that perspective, not only the patients themselves but also we have been obviously through a whole educational system with our patient the groups that, for instance, we had last year the RRP Day and for patient awareness and RRP awareness.

從這個角度來看，不僅患者本身，而且我們顯然也對我們的患者群體實施了一整套教育體系，例如，去年我們舉辦了 RRP 日，以提高患者意識和 RRP 意識。

And these basically campaigns will continue as we go through our PDUFA and beyond to ensure that not only all the patients are aware of the innovative treatment but also that our investigators, laryngologists, and the KOLs, they are fully familiar with the various aspects of the hopefully the new soon-to-be a standard of treatment upon obviously FDA approval for RRP, which is we are thrilled to have played a part of when this hopefully becomes approved for these patients in this century.

隨著我們履行 PDUFA 及後續程序，這些宣傳活動將繼續進行，以確保不僅所有患者都了解這種創新治療方法，而且我們的研究人員、喉科醫生和關鍵意見領袖也完全熟悉這種新治療方法的各個方面，希望這種治療方法在獲得 FDA 批准 RRP 後很快會成為一種標準治療，我們很高興能夠批准這種治療方法在本世紀時為這些患者。

Perfect. Thanks for all that color. And let me try another two-part question. The as far as the confirmatory trial is if for the PRGN-2012, what's the current status? And also do you think you will be close to completion by the time you get we get to the PDUFA date and the last the second part or the second question actually is on the PRGN-3006, should we expect any of that data from the Phase 1 study to be published either in the first half or later in the year?

完美的。感謝所有這些色彩。讓我再嘗試一下由兩個部分組成的另一個問題。就 PRGN-2012 的確認試驗而言，目前的狀態如何？而且，您是否認為當我們到達 PDUFA 日期時，您將接近完成，最後的第二部分或第二個問題實際上是關於 PRGN-3006，我們是否應該預期第一階段研究的任何數據將在上半年或稍後發布？

Great questions. In regard to the confirmatory for PRGN-2012, as mentioned, it's part of the BLA submission is that you have initiated the trial already and we did that last year actually well ahead of the submission of the BLA. And currently, the patients, not only they are being enrolled, the finishing date for the confirmatory trial, as you know, our endpoints.

很好的問題。關於 PRGN-2012 的確認，如上所述，它是 BLA 提交的一部分，您已經啟動了試驗，我們去年就已經開始試驗，實際上早在提交 BLA 之前就已經開始試驗了。目前，不僅正在招募患者，而且正在確定確認性試驗的完成日期，正如您所知，這是我們的終點。

The confirmatory trial design, which again in full alignment and very close agreement with the FDA. FDA has given us the exact same design as we had for our pivotal, which was basically a single-arm trial, 35 patients, no placebo requirement as a second basically arm and it's complete responses, which takes 12 months, minimum of 12 months.

確認性試驗設計再次與 FDA 完全一致且非常接近。FDA 為我們提供了與我們的關鍵試驗完全相同的設計，這基本上是一個單臂試驗，有 35 名患者，不需要安慰劑作為第二組，並且完全緩解需要 12 個月，至少 12 個月。

As I mentioned, we currently have been following, of course, our patients in a prospective manner well over 12 months and the median is at 24 months and our Phase 1 patient, which are the oldest patients that they have been are obviously, those they have now all of those complete responders are still in a complete response three years later.

正如我所提到的，我們目前正在以前瞻性的方式追蹤我們的患者超過 12 個月，中位數為 24 個月，而我們的第 1 階段患者，顯然是年齡最大的患者，現在所有這些完全緩解者在三年後仍然處於完全緩解狀態。

So, by the definition of the complete response endpoint, we will not be having the data by close to the PDUFA date. We anticipate that, of course, in 2026, 2027, we will be reporting on those data. So but as you can imagine, there is a high level of excitement from the patients for this trial, especially in a view of the safety that we have shown the efficacy that we have shown, the ease of administration, the route of administration, which is a subcu, like you are getting a flu shot basically and obviously, the durability of a response that is being seen by the patients.

因此，根據完整回應端點的定義，我們將不會在接近 PDUFA 日期時獲得資料。當然，我們預計在 2026 年、2027 年我們將報告這些數據。因此，正如您所想像的，患者對這次試驗感到非常興奮，特別是考慮到我們已經證明的安全性、我們已經證明的有效性、給藥的簡便性、給藥途徑（皮下給藥），就像您接種流感疫苗一樣，而且顯然患者可以看到反應的持久性。

So I think this further has added to the enthusiasm of the patients and investigators and also really the physicians for this, right?

所以我認為這進一步增加了患者、研究人員以及醫生的熱情，對嗎？

Thank you.

謝謝。

Yeah. And in regard to my apologies, in regard to the UltraCAR and AML, as we will be having the discussions with the FDA, we will be giving an update. And also we are looking at the proper venue to show the data and we will be translating those information in the near future.

是的。關於我的道歉，關於 UltraCAR 和 AML，由於我們將與 FDA 進行討論，我們將提供最新消息。我們也正在尋找合適的場所來展示數據，我們將在不久的將來翻譯這些資訊。

Thank you, Helen. Thanks for all that.

謝謝你，海倫。謝謝你們。

Thank you.

謝謝。

Jennifer Kim of Cantor Fitzgerald LP.

Cantor Fitzgerald LP 的 Jennifer Kim。

Maybe to start off to follow-up on one of the previous questions. I just want to clarify, will the FDA's manufacturing facility inspection come ahead of the mid-cycle review? And I assume that's happening in late May, but do you think you'll have a sense by the mid-cycle review how the FDA is thinking? Maybe we can start there.

也許可以開始跟進之前的問題。我只是想澄清一下，FDA 的生產設施檢查是否會先於中期審查進行？我估計這會在五月底發生，但您認為到中期審查時您能了解 FDA 的想法嗎？也許我們可以從那裡開始。

Thank you, Jennifer. Rutul here. I think yes, we do anticipate a pre-approval inspection or PAI before the approval, but in it's as Helen had mentioned in response to the prior question that it's part of the regulatory process. So we're not able to comment on the timing at this point. But as we have mentioned, we look-forward to hosting them and we anticipate that inspection before the approval,

謝謝你，詹妮弗。這裡是魯圖爾。我認為是的，我們確實預計在批准之前會進行預先批准檢查或 PAI，但正如海倫在回答先前的問題時提到的那樣，這是監管流程的一部分。因此我們目前無法對時間發表評論。但正如我們所提到的，我們期待接待他們，我們預計在批准之前會進行檢查，

Okay. And maybe a question for Phil. The 500 doctors that you're initially targeting, what percent of market does that capture? And could you give some color on how the internal sales force is coordinating with Arizona partnership?

好的。也許還有一個問題想問菲爾。您最初瞄準的 500 名醫生佔了多少市場份額？您能否介紹內部銷售人員如何與亞利桑那州的合作夥伴協調？

Right. Yeah. I mean those 500 ENTs that I mentioned, I mean, they'll be responsible for the vast majority of the patients that we believe are out there. There obviously a community presence as well for patients. So that's something that we're not ignoring.

正確的。是的。我提到的 500 位耳鼻喉科醫生，將負責我們認為的絕大多數病人。顯然，社區也為患者提供服務。所以我們不會忽視這一點。

But again, we feel that the concentration is in the urban centers and that's how we all be thinking about our field force deployment to a large extent. We all the field teams are EVERSANA are through EVERSANA.

但我們再次感覺到，重點還是在城市中心，而我們在很大程度上也是這樣考慮我們的實地部隊部署的。我們所有的現場團隊都是EVERSANA，都是透過EVERSANA。

So we're not having any Precigen employees that are in the field as such. They will all be through EVERSANA but obviously, they will act like Precigen employees and through that strong partnership with EVERSANA. And the other important thing sorry, Jennifer, the other thing important to stress, I did mention it in my prepared notes was that they're dedicated to PRGN-2012.

因此，我們沒有任何 Precigen 員工在該領域工作。他們都將透過 EVERSANA 開展工作，但顯然，他們將像 Precigen 員工一樣行事，並透過與 EVERSANA 的牢固合作關係行事。另一件重要的事情，抱歉，詹妮弗，另一件需要強調的重要的事情，我在準備好的筆記中確實提到過，它們是專門為 PRGN-2012 準備的。

Okay. That's helpful. If I could squeeze one more question in for Phil. In your discussions with payers, is the expectation still that, I guess the access to the treatment if approved would depend mostly on treat-to-trial criteria and label versus a medical exception, or have those discussions evolved?

好的。這很有幫助。我可以再問菲爾一個問題嗎？在與付款人的討論中，是否仍然期望，我猜如果獲得批准，獲得治療的機會將主要取決於治療試驗標準和標籤與醫療例外，或者這些討論已經發展了？

Yeah, we continue to support it's a great question. We continue to speak to many payers. We've actually now spoken to payers that represent collectively over 300 million lives in the US and we're getting a consistent response that you know that the price range that we've been talking with them about that we're likely to see a certain degree of utilization management, some prior authorizations.

是的，我們繼續支持，這是一個很好的問題。我們繼續與許多付款人交談。實際上，我們現在已經與代表美國 3 億多人生命的付款人進行了交談，我們得到的一致回應是，您知道我們與他們討論的價格範圍，我們可能會看到一定程度的利用率管理和一些事先授權。

We hope that that is PA to label. That's what we are trying to achieve, but there are some payers who would actually include inclusion and exclusion criteria. We feel that we have a strong case. We're building strong value proposition to make sure that in either of those scenarios, we're in a great position.

我們希望這是 PA 所標記的。這就是我們努力想要實現的目標，但有些付款人實際上會包含納入和排除標準。我們覺得我們有充分的理由。我們正在建立強大的價值主張，以確保在任何一種情況下，我們都處於有利地位。

Medical exception is really the last resort in terms of where we think some of these payers will go based upon the discussions that we have. So we're preparing ourselves with a strong value proposition to complement the clinical value proposition that we have.

根據我們的討論，我們認為醫療例外確實是這些付款人將採取的最後手段。因此，我們正在準備一個強而有力的價值主張來補充我們現有的臨床價值主張。

And I think, Jennifer, it's from the perspective of obviously that this would be for all RRP patients and the as recent data have shown, when these patients even receive when they have received up to five surgeries.

珍妮佛，我認為，從這個角度來看，這顯然適用於所有 RRP 患者，而且最近的數據顯示，當這些患者接受過多達五次手術時，他們也會接受治療。

Then the damage to the vocal cords and trachea, it becomes irreversible. So, you can imagine that it becomes very important in a setting that treat patients prior to getting to these levels. And obviously, the payers also recognize that in the basically the effect of the surgery on patients that on their life, on the irreversible damage, but also on the losses that it's occurred as a result of all of that. And I think this is one of the very important part of the recognition by the payers here.

那麼聲帶和氣管的損傷就變得不可逆了。因此，你可以想像，在達到這些水平之前對患者進行治療變得非常重要。顯然，付款人也認識到手術對患者的影響，對他們的生活造成不可逆轉的損害，以及由此造成的損失。我認為這是獲得付款人認可的非常重要的部分之一。

That's helpful. Thanks, guys.

這很有幫助。謝謝大家。

Brian Cheng of JPMorgan.

摩根大通的 Brian Cheng。

Thanks for taking our questions this afternoon. Maybe just first, can you give us an update on, the latest on where you are in preparing the sites to administer 2012?

感謝您今天下午回答我們的問題。首先，您能否向我們介紹 2012 年管理場地的準備工作的最新進展？

And you talk about there are 500 initial doctors. Can you talk about how the initial 500 doctors overlap with those initial sites that are ready to go to administer 2012? And then we have a follow-up. Thank you.

您說最初有 500 位醫生。您能否談談最初的 500 名醫生與準備在 2012 年開展工作的初始站點有何重疊？然後我們進行後續跟進。謝謝。

Thanks, Brian. I mean, there's nothing overly complex about administering the drug at the site or special preparation that really needed. We know we have cold chain requirement but we know we're for subcutaneous injections.

謝謝，布萊恩。我的意思是，在現場給藥並不需要太複雜或進行任何特殊準備。我們知道我們有冷鏈要求，但我們知道我們用於皮下注射。

Nothing in the research that we've done and the sites that we're speaking to suggest that there's anything in terms of special preparation that is required other than that to think about. So, we at the academic institutions are large IDNs, they're well-equipped.

我們所做的研究和我們所談論的網站都沒有表明除了考慮這一點之外還需要做任何特殊準備。所以，我們的學術機構都是大型 IDN，設備齊全。

We're obviously understanding exactly what that last 100 yards for the drug journey looks like and we're speaking to them regarding that so that basically we deliver a seamless distribution service as possible from ordering the drug to the patient and that's been the focus. And there's nothing particularly complex or special that we need to call out in terms of preparation.

我們顯然非常清楚藥品旅程的最後 100 碼是什麼樣的，我們正在與他們討論這個問題，以便我們基本上能夠提供從訂購藥品到患者整個過程中盡可能無縫的配送服務，而這一直是我們的重點。在準備方面，沒有什麼特別複雜或特別的事情需要我們指出。

Okay. And then maybe just one on your thoughts on pricing and payer access. And maybe just one if what's your latest take on pricing for 2012? And then just on the pricing standpoint, can you talk about how we should think about access here when it comes to your agreement with payers?

好的。然後也許只是想談談您對定價和付款人訪問的看法。也許還有一個問題，您對 2012 年的價格有何最新看法？然後，僅從定價的角度來看，您能否談談在與付款人達成協議時我們應該如何考慮訪問問題？

Do you think that the value-based agreements will be expected here? Any initial thoughts just based on your ongoing engagement with payers will be very helpful.

您認為這裡會期待基於價值的協議嗎？任何基於您與付款人的持續接觸的初步想法都將非常有幫助。

Yeah, sure. As I said earlier, I mean we continue to speak to payers and we continue to get a fairly consistent response in terms of the value proposition, economic and clinical that we're making. So nothing's really changed in that regard. Value-based pricing, I mean, I know it's out there. It's something that's being considered.

是的，當然。正如我之前所說，我的意思是我們繼續與付款人交談，並且我們繼續就我們所提出的價值主張、經濟和臨床得到相當一致的回應。因此，在這方面實際上沒有任何改變。我的意思是，基於價值的定價，我知道它存在。這是正在考慮的事情。

The payers tend to have a view that it's attractive in theory, difficult in practice sometimes. So we're exploring that a little bit more. But again, we'll we believe that the price point in the way that we'll bring this value to the market is going to be relatively straightforward and something that is within the wheelhouse of these institutions and not something they haven't seen before.

付款人往往認為，這在理論上很有吸引力，但在實踐中有時卻很困難。因此我們正在對此進行進一步探索。但再次強調，我們相信，我們將這種價值帶入市場的價格點將相對簡單，並且屬於這些機構的掌控範圍之內，而不是他們以前從未見過的東西。

Yeah. And also, Brian, maybe I can add. This is Helen. Obviously, as we have communicated before, this is a rare disease with no standard of care for the past for whole century to be honest and payers recognize that and they recognize also the losses to these individuals, not only the financial, but the emotional, and also from a life perspective.

是的。另外，布萊恩，也許我可以補充一下。這是海倫。顯然，正如我們之前所傳達的，這是一種罕見疾病，說實話，在過去的整個世紀裡都沒有標準的治療方法，付款人認識到了這一點，他們也認識到這些人的損失，不僅是經濟上的，還有情感上的，以及生活上的。

And then on top of that, one of the things that becomes very, very important and this the payers appreciate and in discussions that we have had is very clear the safety that this drug delivers, the efficacy, which is unseen prior by any treatment, 51% complete responders.

除此之外，還有一件非常非常重要的事情，付款人非常欣賞這一點，而且在我們討論的過程中，非常清楚的是這種藥物的安全性和療效，這是任何治療方法都無法比擬的，完全緩解率為 51%。

And as I mentioned today and updated actually for the first time, we have now the median standing at 24 months, but also our Phase 1, all the complete responders from a Phase 1 study that we started in 2021, and they are still in a complete response.

正如我今天提到的，實際上也是第一次更新，我們現在的中位數是 24 個月，而且我們的第 1 階段，以及我們 2021 年開始的第 1 階段研究中所有完全緩解者，他們仍然處於完全緩解狀態。

And this is another important part of the discussions that payers are very much appreciative of in regard to the efficacy, durability of response, the ease of administration, and also the effect that it has not only on stopping the disease for majority of these patients as a complete responders and reducing the requirement for surgeries in 86% of the patient but also from the fact that once they get in and the earlier that they get in with our PRGN-2012, it can really affect and reverse the situation, not to get to the situation that now there is irreversible damage to the vocal cords or the trachea of these patients.

這是討論的另一個重要部分，付款人非常欣賞它的功效、反應的持久性、給藥的便利性，以及它不僅能使大多數完全緩解的患者停止患病，並減少 86% 患者的手術需求，而且一旦他們開始使用我們的 PRGN-2012，越早開始使用，就能真正影響和扭轉局面，而不會導致這些患者的聲帶或氣管受到不可逆轉的損害。

And as a result of that, there is a permanent and lifetime damage. And I think that's another extremely important value out there that they do pay attention to.

結果是，造成永久性和終生的損害。我認為這是他們關注的另一個極為重要的價值。

Great. Thank you so much for taking our questions.

偉大的。非常感謝您回答我們的問題。

Thank you.

謝謝。

There are no further questions at this time. I would hand over the call to Dr. Helen Sabzevari for closing remarks. Please go ahead.

目前沒有其他問題。我將把發言權交給 Helen Sabzevari 博士，請她做最後發言。請繼續。

Thank you. As we move one step closer to commercialization, I want to take this opportunity to thank our team at Precigen for their tireless work and a steadfast dedication to bringing PRGN-2012 to market. Our team understands that patients with RRP are burdened significantly by this disease and that with surgery, there are innumerable risks to their health and for long-term irreversible injury.

謝謝。隨著我們向商業化邁進了一步，我想藉此機會感謝 Precigen 團隊的不懈努力和堅定不移的奉獻精神，將 PRGN-2012 推向市場。我們的團隊了解，患有 RRP 的患者承受著這種疾病的沉重負擔，手術會對他們的健康和長期不可逆轉的傷害造成無數風險。

Our team is actually and acutely focused on delivering the first and only FDA approved therapy to the RRP community as quickly as possible. With that, I wish you all a very good evening and thank you for participating in our call.

我們的團隊實際上正專注於盡快向 RRP 社群提供第一個也是唯一一個經 FDA 批准的治療方法。最後，我祝大家有個愉快的夜晚，並感謝你們參加我們的電話會議。

Ladies and gentlemen, this concludes today's conference call. Thank you for your participation and you may now disconnect.

女士們、先生們，今天的電話會議到此結束。感謝您的參與，您現在可以斷開連接。