Abeona Therapeutics Inc (ABEO) 2024 Q2 法說會逐字稿

Good morning, everyone, and welcome to the Abeona Therapeutics second quarter 2024 conference call. (Operator Instructions) Please note this conference is being recorded. I will now turn the conference over to your host, Greg Gin, Vice President of Investor Relations and Corporate Communications. Greg, the floor is yours.

大家早安，歡迎參加 Abeona Therapeutics 2024 年第二季電話會議。（操作員說明）請注意本次會議正在錄製中。現在我將會議交給東道主投資者關係和企業傳播副總裁格雷格金 (Greg Gin)。格雷格，地板是你的。

Thank you, Jenny. Good morning and thank you for joining us on our second quarter 2024 conference call. During this call, we will refer to the press release issued this morning announcing the second quarter results, which is available on our corporate website at www.abeonatherapeutics.com.

謝謝你，珍妮。早安，感謝您參加我們的 2024 年第二季電話會議。在本次電話會議中，我們將參考今天上午發布的第二季業績新聞稿，該新聞稿可在我們的公司網站 www.abeonatherapeutics.com 上取得。

I would like to note that remarks made during today's call may contain projections and forward-looking statements. Forward-looking statements are made pursuant to the safe harbor provisions of the federal securities laws. These forward-looking statements are based on current expectations and are subject to change, and actual results may differ materially from those expressed or implied in the forward-looking statements.

我想指出，今天的電話會議中發表的言論可能包含預測和前瞻性陳述。前瞻性陳述是根據聯邦證券法的安全港條款做出的。這些前瞻性陳述是基於目前的預期，可能會發生變化，實際結果可能與前瞻性陳述中明示或暗示的結果有重大差異。

Various factors that could cause actual results to differ include, but are not limited to those identified under the Risk Factors section in our Form 10-K and periodic reports filed with the SEC. These documents are available on our website at www.abeonatherapeutics.com. On the call today with prepared remarks are Dr. Vishwas Seshadri, Chief Executive Officer; Dr. Madhav Vasanthavada, Chief Commercial Officer and Head of Business Development; and Joseph Vazzano, Chief Financial Officer. Also joining us for the Q&A session will be Dr. Brian Kevany, Chief Technical Officer.

可能導致實際結果有所不同的各種因素包括但不限於我們的 10-K 表格和向 SEC 提交的定期報告中風險因素部分中確定的因素。這些文件可在我們的網站 www.abeonatherapeutics.com 上取得。執行長 Vishwas Seshadri 博士在今天的電話會議上發表了事先準備好的演講； Madhav Vasanthavada 博士，首席商務長兼業務開發主管；財務長約瑟夫‧瓦札諾 (Joseph Vazzano)。技術長 Brian Kevany 博士也將參加我們的問答環節。

And with that, I will now turn the call over to Vish Seshadri, to lead us off. Vish?

現在，我將把電話轉給 Vish Seshadri，讓我們出發。維什？

Thank you, Greg. Good morning, everyone. We appreciate everybody joining this call. We're excited to provide our second quarter update, and I'll start with the status of the BLA resubmission process. As a reminder, the complete response letter highlighted the need for additional CMC information pertaining to in general validation requirements for certain manufacturing and release testing methods. The CRO that we received in April did not identify any deficiencies related to the clinical efficacy or clinical safety data in the BLA and the FDA did not request any new clinical trials or clinical data to support the approval of pz-cel.

謝謝你，格雷格。大家早安。我們感謝大家加入這次電話會議。我們很高興提供第二季的更新，我將從 BLA 重新提交流程的狀態開始。作為提醒，完整的回覆信強調需要與某些製造和發布測試方法的一般驗證要求相關的額外 CMC 資訊。我們四月收到的 CRO 沒有發現 BLA 中與臨床療效或臨床安全性數據相關的任何缺陷，FDA 也沒有要求任何新的臨床試驗或臨床數據來支持 pz-cel 的批准。

Since our last quarterly call, we've continued to make tremendous progress and have now generated the necessary data and reports to address nearly all of the CRO items with work ongoing for two outstanding items. Specifically, we've completed the work necessary to address deficiencies pertaining to RCR or replication competent retrovirus assay, retrovirus validation, container closure integrity testing, whole-cell DNA, virtual contract testing validation and importantly, data that support the extension of product shelf life.

自從上次季度電話會議以來，我們繼續取得巨大進展，現在已經產生了必要的數據和報告來解決幾乎所有 CRO 項目，其中兩個未完成項目的工作正在進行中。具體來說，我們已經完成了必要的工作，以解決與RCR 或複製能力逆轉錄病毒測定、逆轉錄病毒驗證、容器封閉完整性測試、全細胞DNA、虛擬合約測試驗證以及重要的是支援延長產品保質期的數據相關的缺陷。

For the two outstanding items, namely our rapid sterility assay and cell-based identity assay, feasibility data that informed the design of our validation protocols have been generated and we have finalized the validation protocols and have initiated the validation runs. I'm pleased to say we successfully completed a Type A meeting with the FDA last week wherein we gained preliminary alignment on the acceptability of the data that addressed several of the CMC deficiencies noted in the CRO.

對於兩個突出的項目，即我們的快速無菌測定和基於細胞的身份測定，已經產生了為我們的驗證方案設計提供資訊的可行性數據，我們最終確定了驗證方案並啟動了驗證運行。我很高興地說，我們上週成功完成了與 FDA 的 A 類會議，其中我們就數據的可接受性達成了初步一致，解決了 CRO 中指出的幾個 CMC 缺陷。

In addition, we also gained the FDA input on validation protocols for the rapid sterility and cell-based identity assays, and that has been incorporated into the validation procedures that are now ongoing. Based on the FDA's written responses to our questions in premeeting materials and our minutes from this meeting, we believe we have clarity and alignment with the FDA on the content of our BLA resubmission for pz-cel.

此外，我們還獲得了 FDA 對快速無菌和基於細胞的身份測定驗證方案的意見，並且該意見已納入目前正在進行的驗證程序中。根據 FDA 對我們在會前資料中提出的問題的書面答覆以及我們本次會議的記錄，我們相信我們對 pz-cel 重新提交的 BLA 的內容與 FDA 保持一致。

We are, therefore, on track to resubmit the BLA for pz-cel this year. If the BLA resubmission is accepted for review, we expect the FDA to set a PDUFA date six months from the date of resubmission. Beyond the progress on pz-cel, we announced in July a nonexclusive agreement with Beacon Therapeutics, allowing Beacon to evaluate our patented AAV204 capsid for the development and commercialization of potential gene therapies in select ophthalmology indications with an option to evaluate up to nine targets.

因此，我們預計今年重新提交 pz-cel 的 BLA。如果 BLA 重新提交被接受審查，我們預計 FDA 將設定 PDUFA 日期，自重新提交之日起六個月。除了pz-cel 的進展之外，我們在7 月宣布與Beacon Therapeutics 達成一項非排他性協議，允許Beacon 評估我們的專利AAV204 衣殼，用於特定眼科適應症的潛在基因療法的開發和商業化，並可選擇評估最多9 個標靶。

We look forward to collaborating with Beacon and believe this agreement underscore AAV204's potential to enable efficient targeting in the eye of novel AAV-based gene therapies for ophthalmic diseases with high unmet need.

我們期待與 Beacon 合作，並相信該協議強調了 AAV204 在基於 AAV 的新型基因療法中高效靶向眼部的潛力，用於治療需求未滿足的眼科疾病。

Finally, I'll mention that we completed $175 million underwritten offering with institutional investors in May, strengthening our balance sheet and extending our cash runway well beyond anticipated significant regulatory milestone and commercial launch of pz-cel.

最後，我要提到的是，我們在5 月與機構投資者完成了1.75 億美元的承銷發行，增強了我們的資產負債表，並擴展了我們的現金跑道，遠遠超出了預期的重大監管里程碑和pz-cel 的商業推出。

I'll now turn the call over to our Chief Commercial Officer, Dr. Madhav Vasanthavada, to provide an update on our commercialization readiness activities. Madhav?

我現在將把電話轉給我們的商務長 Madhav Vasanthavada 博士，以提供我們商業化準備活動的最新資訊。馬達夫？

Thanks, Vish, and good morning, everyone. We are building a solid foundation to prepare for a successful launch of pz-cel following its potential approval. And we are very excited by the feedback we've been hearing from multiple stakeholders. I'll start with our support for medical education activities at various conferences where we have had great conversations with EB physicians and patients.

謝謝，維什，大家早安。我們正在為 pz-cel 在獲得批准後的成功推出奠定堅實的基礎。我們對從多個利害關係人那裡聽到的回饋感到非常興奮。我將從我們在各種會議上對醫學教育活動的支持開始，在這些會議上我們與 EB 醫生和患者進行了精彩的對話。

As a Society for Investigative Dermatology, SID Annual Meeting in May, we presented our new long-term safety data of pz-cel in 18 RDEB patients with the longest follow-up period of 11 years. As key takeaway, in addition to the generally well-tolerated safety profile, no squamous cell carcinoma was reported in any of the 128 pz-cel-treated sites during the follow-up.

作為皮膚病研究學會 5 月的 SID 年會，我們展示了 pz-cel 在 18 名 RDEB 患者中的新長期安全性數據，最長追蹤期為 11 年。關鍵要點是，除了普遍耐受性良好的安全性外，在追蹤期間，128 個 pz-cel 治療部位均未報告鱗狀細胞癌。

Squamous cell carcinomas have been reported in nontreated sites unrelated to pz-cel. Then in July, at the Society of Pediatric Dermatology, SPD Annual Meeting we presented data illustrating pz-cel's ability to cover wounds of various sizes, including large areas and on different anatomical locations. Based on our interactions with health care professionals, whether they plan to treat with pz-cel themselves or refer to qualify treatment centers, they are very enthusiastic about pz-cel's potential to make a difference in addressing the persistent unmet needs of our patients.

據報道，在與 pz-cel 無關的未治療部位會出現鱗狀細胞癌。然後在 7 月份，在兒科皮膚病學會 SPD 年會上，我們展示了數據，說明 pz-cel 能夠覆蓋各種大小的傷口，包括大面積和不同解剖位置的傷口。根據我們與醫療保健專業人員的互動，無論他們是計劃自己使用pz-cel 進行治療還是轉介合格的治療中心，他們都非常熱衷於pz-cel 在解決患者持續未得到滿足的需求方面發揮作用的潛力。

We recently attended the debra Care Patient Conference in Atlanta, where we noticed so many RDEB patients almost all of them having significant wounds on their bodies wrapped in bandages. Pz-cel has the potential to see large body surface areas, including the toughest to treat wounds while demonstrating meaningful pay and edge reduction based on clinical data from two trials, a Phase 1, 2a study with up to eight years of follow-up and the intra-patient randomized Phase 3 VITAL study.

我們最近參加了在亞特蘭大舉行的 debra Care 患者會議，在那裡我們注意到有很多 RDEB 患者，幾乎所有人身上都有用繃帶包裹的嚴重傷口。Pz-cel 有潛力看到大的身體表面積，包括最難治療的傷口，同時根據兩項試驗的臨床數據顯示出有意義的報酬和邊緣減少，這是一項1、2a 期研究，隨訪時間長達八年，病患內部隨機 3 期 VITAL 研究。

Pz-cel is the only product in the RDEB space with more than a decade of clinical experience to show not only a clean long-term safety profile but also durable wound healing after only a single application. These aspects of pz-cel make it a highly differentiated and clinically meaningful potential treatment for RDEB patients, caregivers and physicians.

Pz-cel 是 RDEB 領域唯一一款擁有十多年臨床經驗的產品，不僅具有良好的長期安全性，而且僅在一次使用後即可實現持久的傷口癒合。pz-cel 的這些方面使其成為 RDEB 患者、照護者和醫生的高度差異化且具有臨床意義的潛在治療方法。

At the debra Care Conference, we also shared a clinical research update and data on wound healing following the pz-cel treatment process. The interest from patients, caregivers and providers was palpable when they saw the before and after pz-cel wound healing pictures for several large and chronic wounds. We also heard from patients who have received pz-cel, including a few who had recently completed their second pz-cel treatment, they shared -- they reached out to share with us the life-changing impact that pz-cel has had on their lives and the lives of their loved ones. Such testimonials highlight the transformation potential of pz-cel therapy.

在 debra Care 會議上，我們也分享了 pz-cel 治療過程後傷口癒合的臨床研究更新和數據。當患者、照護者和提供者看到幾個大的慢性傷口的 pz-cel 傷口癒合前後的圖片時，他們的興趣顯而易見。我們也聽取了接受過pz-cel 治療的患者的來信，其中包括一些最近完成第二次pz-cel 治療的患者，他們分享道，他們主動與我們分享pz-cel 對他們的生活產生了改變的影響。這些評估凸顯了 pz-cel 療法的變革潛力。

Taking our efforts with the payer community, we are excited to announce that our ongoing discussions with major commercial payers are generating positive results. Payers are recognizing the clinical value of pz-cel and its potential to address significant unmet needs in the current treatment landscape, enhancing our optimism for favorable coverage and broad and timely patient access post approval.

透過與付款人社群的努力，我們很高興地宣布，我們與主要商業付款人正在進行的討論正在產生積極的成果。付款人正在認識到 pz-cel 的臨床價值及其解決當前治療領域中未滿足的重大需求的潛力，這增強了我們對有利的覆蓋範圍以及批准後廣泛而及時的患者訪問的樂觀態度。

Lastly, from a site onboarding standpoint, each interaction with targeted experienced EB centers is allowing us to deepen relationships and increase our readiness towards site actuation. Massive service agreement negotiations are ongoing, and we remain on track for potential launch approximately three months after pz-cel approval. All of these sites remain highly engaged, and we plan to accelerate our onboarding activities soon after BLA resubmission is complete.

最後，從網站啟動的角度來看，與經驗豐富的目標 EB 中心的每次互動都使我們能夠加深關係並提高我們對網站啟動的準備程度。大規模服務協議談判正在進行中，我們仍有望在 pz-cel 獲得批准後大約三個月內推出。所有這些網站都保持高度活躍，我們計劃在 BLA 重新提交完成後儘快加快我們的入職活動。

With that, I will now hand the call over to our Chief Financial Officer, Joe Vazzano, to discuss our financial results. Joe?

現在，我將把電話轉交給我們的財務長喬·瓦扎諾 (Joe Vazzano)，討論我們的財務表現。喬？

Thanks, Madhav. I would like to remind everyone that you can find additional details on our financial results for the three and six months ended June 30, 2024, and our most recent Form 10-Q, which is available on our website. Starting with the financial resources on our balance sheet, we had cash, cash equivalents, short-term investments and restricted cash of $123 million as of June 30, 2024. This compares to $62.7 million as of March 31, 2024.

謝謝，馬達夫。我想提醒大家，您可以在我們的網站上找到有關我們截至 2024 年 6 月 30 日的三個月和六個月的財務業績以及最新的 10-Q 表格的更多詳細資訊。從資產負債表上的財務資源開始，截至 2024 年 6 月 30 日，我們的現金、現金等價物、短期投資和限制性現金為 1.23 億美元。相比之下，截至 2024 年 3 月 31 日，這一數字為 6,270 萬美元。

Net cash used in operating activities was $12.7 million for the three months ended June 30, 2024. Based on our current operating plan and assumptions, with our existing cash resources, also including the credit facility, we estimate we have sufficient financial resources to fund our operations into 2026. Our cash runway assumptions do not account for any potential revenue from commercial sales or pz-cel or proceeds from the sale of a Priority Review Voucher, or PRV, if awarded by the FDA.

截至 2024 年 6 月 30 日的三個月，經營活動使用的現金淨額為 1,270 萬美元。根據我們目前的營運計劃和假設，利用我們現有的現金資源（包括信貸額度），我們估計我們有足夠的財務資源為我們的營運提供資金到 2026 年。我們的現金跑道假設不考慮商業銷售或 pz-cel 的任何潛在收入或優先審查券 (PRV) 銷售收益（如果 FDA 授予）。

I'll remind you that pz-cel has been granted rare pediatric disease designation by the FDA. So upon its potential approval, we believe that we are eligible to receive a PRV. Research and development expenses were $9.2 million for the three months ended June 30, 2024, compared to $8.5 million for the three months ended June 30, 2023.

我要提醒您的是，pz-cel 已被 FDA 授予罕見兒科疾病資格。因此，一旦獲得批准，我們相信我們有資格獲得 PRV。截至2024年6月30日止三個月的研發費用為920萬美元，截至2023年6月30日止三個月的研發費用為850萬美元。

Our spend in general and administrative activities was $8.6 million for the three months ended June 30, 2024, compared to $5 million for the three months ended June 30, 2023. The increase in general and administrative expenses is primarily due to commercial and launch preparation costs.

截至2024年6月30日的三個月，我們的一般和行政活動支出為860萬美元，而截至2023年6月30日的三個月為500萬美元。一般和管理費用的增加主要是由於商業和發射準備成本。

Net income was $7.4 million for the second quarter of 2024. It's important to note that the net income in the second quarter of 2024 included a $24.9 million gain resulting from the quarterly remeasurement of the fair value of warrant liability. These warrants are required to be classified as a liability and remeasured at fair market value each reporting period. Net loss in the second quarter of 2023 was $16.7 million, including an $8.6 million loss resulting from the quarterly remeasurement of the fair value of warrant liabilities.

2024 年第二季淨利為 740 萬美元。值得注意的是，2024 年第二季的淨利潤包括因季度重新計量認股權證負債公允價值而產生的 2,490 萬美元收益。這些認股權證需要歸類為負債，並在每個報告期間以公平市價重新計量。2023年第二季淨虧損為1,670萬美元，其中包括因季度重新計量認股權證負債公允價值而產生的860萬美元虧損。

And with that, I'll hand the call back over to Vish, for brief closing remarks before opening the call for Q&A.

接下來，我將把電話轉回給 Vish，在開始問答電話之前進行簡短的結束語。

Thanks, Joe. In closing, we have made significant progress in less than four months since receiving the complete response letter. We are in a much better position than we could have hoped for, and we are on track for the BLA resubmission in the second half of 2024. We remain committed to bringing pz-cel to patients with RDEB as quickly as possible. I firmly believe we will get there.

謝謝，喬。最後，自從收到完整的回信以來，我們在不到四個月的時間內取得了重大進展。我們的處境比我們希望的要好得多，我們預計在 2024 年下半年重新提交 BLA。我們仍然致力於盡快將 pz-cel 帶給 RDEB 患者。我堅信我們會到達那裡。

Operator, please open the Q&A session.

接線員，請打開問答環節。

(Operator Instructions)

（操作員說明）

Maury Raycroft, Jefferies.

莫里‧雷克羅夫特，傑弗里斯。

This is Farzin on for Maury. For the two remaining outstanding items related to sterility assays and identity assays, can you say more about what the FDA feedback was? Like I mean, are they asking for you to replicate something for a new iteration or are they asking for a new experiment there?

這是法爾津 (Farzin) 替補莫里 (Maury) 的比賽。對於與無菌測定和身分測定相關的剩餘兩個未決項目，您能否詳細介紹一下 FDA 的回饋？就像我的意思是，他們是要求你為新的迭代複製一些東西，還是要求在那裡進行新的實驗？

Thank you for that question. So let me take the first outstanding item, which is the rapid sterility test. As a reminder, this is a method that was suggested by the FDA themselves because of prior experience with this approach. The suggestions were primarily to the statistical approach that we're using to establish the comparability between the current gold standard USB 71 method of looking at sterility and what we have developed and it is not a development of a new experimental method per se.

謝謝你提出這個問題。那麼，讓我來談談第一個突出的項目，快速無菌檢測。提醒一下，這是 FDA 自己建議的方法，因為之前有這種方法的經驗。這些建議主要針對我們用來建立目前檢查無菌性的黃金標準 USB 71 方法與我們開發的方法之間的可比性的統計方法，它本身並不是新實驗方法的開發。

So I just wanted to clarify what that input was to the validation procedure. We don't see this as even a major amendment to how we were validating. This was out of abundance of caution, we wanted the FDA to take a look at our validation protocol and approach and suggest if they had a preference for one statistical method versus other. So that's really what we had, and that's why we wanted to hold off in starting the validation experiment until we got that feedback. So that's regarding the sterility assay.

所以我只是想澄清驗證過程的輸入是什麼。我們認為這甚至不是對我們驗證方式的重大修改。這是出於非常謹慎的考慮，我們希望 FDA 查看我們的驗證方案和方法，並建議他們是否更傾向於一種統計方法。這就是我們所擁有的，這就是為什麼我們想要推遲開始驗證實驗，直到我們得到回饋。這就是關於無菌檢測的情況。

Regarding the identity assay, we've actually started the validation work, and we have pretty good alignment from the FDA on our approach in how we look at the cell composition of our sheet, the discussion was more around what kind of characterization data to be included and how we put justifications in place that the way we've developed these identity assays. So I hope that addresses the two questions. Happy to talk further about it if you are interested.

關於身份測定，我們實際上已經開始了驗證工作，並且我們在如何看待我們的表的細胞組成方面與 FDA 取得了很好的一致性，討論更多地圍繞著什麼樣的表徵數據包括以及我們如何為我們發展這些身分分析的方式提供理由。所以我希望這能解決這兩個問題。如果您有興趣，很樂意進一步討論。

Got it. And then did the FDA provide feedback on the retroviral replication assay? Is that one good to go? Or there is no more feedback from FDA?

知道了。FDA 是否對逆轉錄病毒複製檢測提供了回饋？那是一件好事嗎？還是FDA沒有更多的回饋？

Yes, that one is good to go. Even before we had the Type A meeting, we had written back and forth with the FDA and shared the data that we've generated and wanted the confirmation that this really addresses the need for the RCR assay, and we have that behind us now.

是的，那一個就可以了。甚至在我們召開 A 類會議之前，我們就已經與 FDA 來回寫信並分享了我們產生的數據，並希望確認這確實滿足了 RCR 檢測的需求，現在我們已經做到了這一點。

And then one quick one is that will you need to have another formal meeting with the FDA? Or is there any more granularity on the -- when in second half (inaudible)

然後一個快速的問題是，您是否需要與 FDA 舉行另一次正式會議？或者在下半場時是否有更多的粒度（聽不清楚）

So we do not plan to have any more formal meetings with the FDA like a Type A or Type B meeting per se, but we are not guiding exactly when the second half of this year we are planning the resubmission. Purely because the validation runs are ongoing as we speak, and it's just a matter of when they get completed. So it's tricky to predict when exactly these types of experiences will be completed. Their reports generated all the I's dotted and T's crossed. So we will guide as previously we had done, we remain on track for a second half of the year submission.

因此，我們不打算與 FDA 舉行任何更正式的會議，例如 A 類或 B 類會議本身，但我們並沒有準確指導今年下半年我們計劃重新提交的時間。純粹是因為正如我們所說，驗證運行正在進行中，這只是驗證何時完成的問題。因此，很難準確預測這些類型的體驗何時會完成。他們的報告產生了所有 I 點和 T 交叉點。因此，我們將像以前一樣進行指導，我們仍將在今年下半年提交。

Thank you so much.

太感謝了。

Dae Gon Ha, Stifel.

大貢河，斯蒂菲爾。

Hey, good morning, guys. Thanks for taking our call (technical difficulty) the second half question a little bit more. Was there any direct feedback on additional data or additional assay? I mean it seems like the last time we spoke, everything was on track to be sort of at the end of the Type A meeting, that seemed almost like the rate limiting step before you resubmit.

嘿，早上好，夥計們。感謝您接聽我們的電話（技術難度），後半部的問題多了一點。是否有關於額外數據或額外測定的任何直接回饋？我的意思是，看起來我們上次談話時，一切都在 A 類會議結束時按計劃進行，這似乎幾乎是您重新提交之前的速率限制步驟。

So I'm just wondering if there was anything additional that you need to run or anything additional that needs to be completed to kind of fulfill the dotting of the T's and I guess, dotting on the I's and crossing the T's, if you will.

所以我只是想知道是否有任何額外的需要運行或需要完成的任何額外的東西來完成 T 的點，我想，如果你願意的話，在 I 上點並交叉 T 。

And then one question for Madhav. In your prepared remarks, you were talking about on track for launching about three months after pz-cel approval. Did I hear that correct? And if so, just wondering what additional work needs to get done to get the sites on board. It seems like six months is quite a bit of time, but now you're estimating about three more months beyond that. So any update on that would be great. Thank you so much.

然後問馬達夫一個問題。在您準備好的演講中，您談到了在 pz-cel 獲得批准後大約三個月後啟動的計劃。我沒聽錯吧？如果是這樣，只是想知道需要完成哪些額外工作才能讓網站加入。看起來六個月的時間相當長，但現在您估計還需要大約三個月的時間。所以任何關於這方面的更新都會很棒。太感謝了。

Great. Dae Gon, let me address your first question regarding any additional data. We have generated a lot of feasibility data for those two outstanding topics that I spoke about. And if you really look at the laundry list of the number of things that we had to do, you're looking at the tip of the iceberg here. I mean these are just the two aspects. But they are also the more complex assays and before conducting validation, we have generated the feasibility data that informed the design and having done that, we wanted to make sure that we put the protocol in front of the FDA and make sure that they're aligned with how we've approached it.

偉大的。Dae Gon，讓我回答您關於任何其他數據的第一個問題。我們已經為我談到的這兩個突出主題產生了大量可行性數據。如果你仔細查看我們必須做的事情的清單，你看到的只是冰山一角。我的意思是這只是兩個方面。但它們也是更複雜的測定，在進行驗證之前，我們已經產生了為設計提供資訊的可行性數據，完成後，我們希望確保將協議提交給 FDA 並確保它們與我們的處理方式一致。

So it's really input on that. It is not additional work that they've suggested. It's essentially a statistical approach as to how you establish comparability. In fact, I'm only talking about what is that little piece within the complex question of sterility that we needed to tweak based on the FDA's feedback. But there are many, many different aspects of it that we've already aligned on. So we're good to go on that.

所以這確實是一個投入。他們建議的並不是額外的工作。它本質上是一種關於如何建立可比性的統計方法。事實上，我只是在談論複雜的無菌問題中我們需要根據 FDA 的回饋進行調整的那一小部分。但我們已經在很多很多不同的方面達成了一致。所以我們很高興繼續下去。

So even having just that piece in the validation protocol that they advise to follow a slightly different approach is a pretty big win for us because it could have come multiple different ways. And having known this clarity, we feel pretty confident based on all the data we've generated using the system in-house. As a reminder, both of these assays are done in-house. We're not getting this done through a vendor.

因此，即使驗證協議中只有他們建議遵循稍微不同的方法的一部分，對我們來說也是一個相當大的勝利，因為它可能有多種不同的方式。在了解了這種清晰度後，我們對使用內部系統產生的所有數據感到非常有信心。提醒一下，這兩種檢測都是在內部完成的。我們不會透過供應商來完成這項工作。

So that's something that's assuring it's in our control. So it's just a matter of generating that data. And I know that previously we had not guided exactly when in the second half and we leave it at that because it's all the pins fall in place exactly the way we wanted, maybe end of September, early October. We don't want to go into the guessing speculating game right now, is it quarter three or is it quarter four, but we do feel quite confident that it will get done this year.

所以這可以確保它在我們的控制之下。所以這只是生成數據的問題。我知道之前我們並沒有準確地指導下半年的具體時間，所以我們就這樣留著，因為所有的細節都按照我們想要的方式就位，也許是九月底，十月初。我們現在不想進入猜測遊戲，是第三季度還是第四季度，但我們確實非常有信心今年會完成。

And to your other question on what kind of site onboarding activities would be completed by the time we get approval and what has to be triggered after I'll let Madhav address that.

至於你的另一個問題，即在我們獲得批准時將完成什麼樣的網站登入活動，以及在我將讓 Madhav 解決這個問題後必須觸發什麼。

Thanks, Dae Gon, for the question. So we are very much engaged with the sites in training them, but there are certain aspects that can only be done after a product insert or prescribing information is given, which is beyond past approval. So training the sites on the actual product information. Once we have the price point, we have to note that as part of the charge master in the hospital institutions the P&T committee and the agenda item that is placed on there in certain institutions.

謝謝大坤的提問。因此，我們非常積極地與網站合作，對他們進行培訓，但有些方面只能在提供產品說明書或處方資訊後才能完成，這超出了過去的批准範圍。因此，請根據實際產品資訊對網站進行培訓。一旦我們確定了價格點，我們就必須注意到，作為醫院機構收費主控的一部分，P&T 委員會以及某些機構中放置在那裡的議程項目。

So things of that nature that can only be done after we have the label very similar to autologous CAR T cell therapies as a model. And having launched those therapies, we typically guide two to three months is usually the time frame that's needed for post-approval activations. We'll, of course, be working as soon as we can in getting the centers ready so that first patient can be treated because that's really our intent there. Hopefully, that gives some context.

因此，這種性質的事情只有在我們擁有與自體 CAR T 細胞療法非常相似的標籤作為模型後才能完成。在推出這些療法後，我們通常會指導兩到三個月是批准後啟動所需的時間範圍。當然，我們將盡快讓這些中心做好準備，以便第一個病人能夠得到治療，因為這確實是我們的意圖。希望這能提供一些背景資訊。

Yeah. No, that's very helpful. I guess on that point, on the pricing and reimbursement, you also talked about some payer discussions being fruitful. I was wondering if you could comment on sort of the poly, I guess, combo therapy type of discussions? Have you had that? What are the sort of feedback on payer side about funding both Vyjuvek as well as pz-cel?

是的。不，這非常有幫助。我想在這一點上，在定價和報銷方面，您也談到了一些付款人的討論是富有成效的。我想知道您是否可以對某種聚合療法（我想是組合療法類型的討論）發表評論？你有過這樣的經驗嗎？付款方對於資助 Vyjuvek 和 pz-cel 有何回饋？

Generally, when you look at the pz-cel's profile, it's resonating extremely well. I think payers understand why Vyjuvek is on the market, Filsuvez is on the market. And the profile for pz-cel is distinct and differentiated in that for large and chronic wounds, especially where there's a very heavy bare burden. We haven't heard any major direct objections of blocking one versus the other because the modalities are distinct.

一般來說，當您查看 pz-cel 的配置時，您會發現它的共鳴非常好。我認為付款人明白為什麼 Vyjuvek 會出現在市場上，Filsuvez 出現在市場上。pz-cel 的特性在大面積和慢性傷口方面是獨特且有區別的，特別是在裸露負擔非常重的情況下。我們還沒有聽到任何針對阻止其中一種的重大直接反對意見，因為方式不同。

So far, so good. And we are, of course, also working with payers to making sure that the access policies that we eventually come out with are favorable because these patients require multiple treatment options. And the fact that pz-cel is one -- single application for durable years of wound coverage versus Vyjuvek where -- or Filsuvez where there is a -- you can pause continue and that kind of thing. It also works favorably from that perspective.

到目前為止，一切都很好。當然，我們也在與付款人合作，以確保我們最終制定的准入政策是有利的，因為這些患者需要多種治療選擇。事實上，pz-cel 是一種單次應用，可持久覆蓋多年的傷口，而 Vyjuvek 或 Filsuvez 則可以暫停，繼續進行此類操作。從這個角度來看，它也起到了有利的作用。

Great. Thanks for taking my questions.

偉大的。感謝您回答我的問題。

Ram Selvaraju, H.C. Wainwright.

塞爾瓦拉朱 (Ram Selvaraju)，H.C.溫賴特。

Thank you so much for taking my questions and congratulations on all the progress. I just wanted to ask one quick clarificatory point regarding the process via which the FDA will consider the resubmission. What is the statutory timing with which the FDA would need to respond to the BLA resubmission once it is filed and to sign a PDUFA date? Can you just remind us what that time frame is, please?

非常感謝您提出我的問題並祝賀所有進展。我只是想問一個關於 FDA 考慮重新提交的流程的快速澄清點。一旦提交 BLA 重新提交並簽署 PDUFA 日期，FDA 需要在什麼法定時間上回應？您能提醒我們那個時間範圍嗎？

Yeah. It is understanding, Ram, great to hear from you. Thank you for the question. Our understanding is that upon resubmission in a two week time frame, the FDA indicates their acceptance of the resubmission and determines also at that point in time, whether it's a Type 1 or a Type 2 variation of the resubmission. We anticipate this may be Type 2 kind of a variation, but that's to be determined. In terms of timing, if it is a Type 2, then it is six months from the time of resubmission that the FDA would likely set a PDUFA date. I hope that answers the question.

是的。拉姆，很理解你，很高興收到你的來信。謝謝你的提問。我們的理解是，在兩週時間範圍內重新提交後，FDA 表示他們接受重新提交，並在該時間點確定重新提交的類型是 1 型還是 2 型變體。我們預計這可能是 Type 2 的變體，但這還有待確定。就時間而言，如果是 2 類，那麼 FDA 可能會在重新提交後六個月內設定 PDUFA 日期。我希望這能回答這個問題。

Yeah, very much. So and just for a quick other follow-up is, if we just, for a moment, think about the hypothetical scenario in which you receive a PRV and elect to monetize it. In such a context, can you give us a sense of whether strategically you would look to broaden your product offering, specifically in the dermatology space or if you would think strategically about potentially broadening your reach into other rare diseases as you think about the optimal commercial strategy for the company.

是的，非常喜歡。因此，作為一個快速的後續行動，我們暫時考慮假設的場景，在這種情況下，您收到 PRV 並選擇將其貨幣化。在這種情況下，您能否讓我們了解您是否會從策略上考慮擴大您的產品範圍，特別是在皮膚科領域，或者您是否會在考慮最佳商業機會時策略性地考慮將業務範圍擴大到其他罕見疾病公司的策略。

Great question, Ram. So you're talking about pipeline and other assets here, not just the pz-cel life cycle management. So pz-cel life cycle management itself is one avenue, we haven't really discussed very much, whether it's ex-US expansion or whether we're talking about other types of applications because we're hearing from a lot of patients that they want pz-cel to be applied for hand surgeries and things like that, which we haven't evaluated clinically. So that's one aspect of it.

很好的問題，拉姆。因此，您在這裡討論的是管道和其他資產，而不僅僅是 pz-cel 生命週期管理。因此，pz-cel 生命週期管理本身就是一種途徑，我們還沒有真正討論太多，無論是在美國以外的擴張，還是我們正在談論其他類型的應用，因為我們從許多患者那裡聽到，他們希望將pz-cel 應用於手部手術等，我們還沒有進行臨床評估。這是一方面。

The other aspect of it is we have core competencies in engineered cell therapy with the pz-cel experience. So that is very applicable, as you can see in multiple engineered cell therapy avenues in a disease-agnostic way. And when I say disease-agnostic way, the entire commercial infrastructure that we set up for autologous cell therapy itself is -- can have a translational effect in multiple other therapeutic areas just by the nature of how -- whether it's skin-to-skin or a vein-to-vein process and the patient experience that is involved here.

另一方面是我們憑藉 pz-cel 經驗在工程細胞治療方面擁有核心能力。所以這是非常適用的，正如您在多種工程細胞治療途徑中以與疾病無關的方式所看到的那樣。當我說與疾病無關的方式時，我們為自體細胞療法本身建立的整個商業基礎設施可以在多個其他治療領域產生轉化效果，無論是皮膚接觸還是皮膚接觸或靜脈到靜脈的過程以及此處涉及的患者體驗。

So -- and do not forget, we also have our ophthalmology platform where we have AAV -- novel AAV capsids with tropism and transaction efficiencies in select eye compartments. And we haven't forgotten those. In the background, there's work that is going on with especially our retinal thesis program. And so in what all different directions, I think it's going to be sowing the seeds in a few different multiple avenues and then letting those saplings grow and see where we can actually best marry both the R&D and commercial infrastructure we're building with a pz-cel launch and which -- where the areas of promise are showing up.

因此，不要忘記，我們也有眼科平台，其中有 AAV——新型 AAV 衣殼，在選定的眼室中具有趨向性和交易效率。我們沒有忘記那些。在後台，我們正在進行視網膜論文專案的工作。因此，在所有不同的方向上，我認為它將在幾個不同的多種途徑中播下種子，然後讓這些樹苗生長，看看我們在哪裡可以真正最好地將我們正在建造的研發和商業基礎設施結合起來-cel 發射以及其中 - 有希望的領域正在顯現。

So I think it's really going to be a discovery process. We'll talk more and more about it as we get closer to pz-cel launch. But just to avoid being schizophrenic right now, we're so focused on the pz-cel launch itself because that is really the most critical deliverable for the company.

所以我認為這確實是一個發現的過程。隨著 pz-cel 發布的臨近，我們將越來越多地討論它。但為了避免現在精神分裂，我們非常關注 pz-cel 的發布本身，因為這確實是公司最關鍵的交付成果。

Thank you so much for that thoughtful response and congrats again on all the progress.

非常感謝您深思熟慮的回复，並再次恭喜所有進展。

Thank you.

謝謝。

(Operator Instructions)

（操作員說明）

Kristen Kluska, Cantor Fitzgerald.

克里斯汀·克魯斯卡，坎托·菲茨杰拉德。

Hi, this is Rick Miller on for Christian. Thanks for taking our questions. On the SaaS approach that the FDA suggested, are you able to characterize whether this is a more stringent approach? Or what was the motivation do you think for suggesting this specific approach?

大家好，我是克里斯蒂安的里克·米勒。感謝您回答我們的問題。關於 FDA 建議的 SaaS 方法，您是否能夠描述這是否是一種更嚴格的方法？或者您認為提出這種具體方法的動機是什麼？

Yeah. Thank you, Rick. Can you repeat that question? I just want to make sure I understand it correctly before I respond.

是的。謝謝你，瑞克。你能重複一下這個問題嗎？我只是想在回復之前確保我正確理解。

Yes. So I believe you mentioned a statistical approach that the FDA suggested related to one of the two remaining outstanding items. So just kind of if you're able to characterize whether this approach is a more stringent approach? Or what do you think the motivation there was for suggesting this specific approach?

是的。因此，我相信您提到了 FDA 建議的與剩下的兩個未決項目之一相關的統計方法。那麼，您是否能夠描述這種方法是否是一種更嚴格的方法？或者您認為提出這種具體方法的動機是什麼？

Yeah. The motivation is purely precedented. The FDA has a preference for a certain type of approach. Guidance do not always exactly specify how to interpret when it comes to actual experimentation. For the given context of how we conduct the experiment, we have a very sensitive rapid detection assay that we've developed for sterility, which is actually not the first time. I think the bioluminescence methods exist for other companies and therapeutic products.

是的。動機純粹是先例。FDA 偏愛某種類型的方法。當涉及到實際實驗時，指南並不總是準確地指定如何解釋。對於我們如何進行實驗的給定背景，我們開發了一種非常靈敏的快速無菌檢測方法，這實際上並不是第一次。我認為其他公司和治療產品也存在生物發光方法。

And historically, we've used USB 71 approaches to how we look at sterility. And I think when we show that method A and method B are equivalent, there are multiple different approaches of showing that. And here what the FDA has given clear instructions to us is that when you're looking at either a noninferiority or a comparability in terms of how you quantitate any microorganisms you spike in our matrix and show that you're able to detect with equal level of sensitivity that required a certain way of statistical handling. So that's really what the feedback was about. So I hope that gives a little bit more color.

從歷史上看，我們一直使用 USB 71 方法來看待無菌性。我認為當我們證明方法 A 和方法 B 是等效的時，有多種不同的方法可以證明這一點。FDA 向我們發出的明確指示是，當您在定量添加到我們的基質中的任何微生物方面考慮非劣效性或可比性時，並表明您能夠以同等水平進行檢測需要某種統計處理方式的敏感性。這就是回饋的真正意義。所以我希望能帶給大家更多的色彩。

Yeah, that helps a lot. Thank you, that's all for us. Thanks.

是的，這很有幫助。謝謝你，這就是我們的全部了。謝謝。

James Molloy, Alliance Global Partners.

詹姆斯·莫洛伊，聯盟全球合作夥伴。

Hey. guys. Good morning. Thank you very much for taking my questions. I had a quick question on -- in the PRV market, I know that Biogen recently reported they sold their last one for $89 million, down a little from $100 million to sort of have been going. Have you guys seen any softening in the PRV market or anything further -- anything going on there that you guys are noting assuming again, you get the approval and get a PRV and look to monetize it?

嘿。夥計們。早安.非常感謝您回答我的問題。我有一個簡單的問題——在 PRV 市場上，我知道 Biogen 最近報告他們以 8900 萬美元的價格出售了最後一款產品，比實際價格 1 億美元略有下降。你們是否看到 PRV 市場出現任何疲軟或進一步的情況 - 你們注意到那裡發生的任何事情再次假設，你們獲得批准並獲得 PRV 並希望將其貨幣化？

Thank you for that question, Jim. I'll let Joe address the question here.

謝謝你提出這個問題，吉姆。我讓喬在這裡回答這個問題。

Yeah. Thanks for that, Jim. Yeah, other than that, for example that you mentioned, I have not seen any softening. I'm still seeing deals north of $100 million -- $103 million or so. I think the going rate is normally about $100 million bill.

是的。謝謝你，吉姆。是的，除此之外，例如你提到的，我沒有看到任何軟化。我仍然看到超過 1 億美元的交易 - 1.03 億美元左右。我認為現行匯率通常約為 1 億美元。

(multiple speakers) Yeah. And Jim, usually it's an optimization between speed and pricing. And in our case, the advantage of being funded so well beyond the approval time frame allows us to optimize our pricing rather than for speed. I think that's what is important. But overall, if you look at the last several months or years even, the price of the PRV has been rather steady at around that $100 million mark. So I will leave it at that, but we're confident that we're not doing a fire escape here.

（多個發言者）是的。吉姆，通常是速度和價格之間的最佳化。就我們而言，獲得遠遠超出批准時間範圍的資助的優勢使我們能夠優化定價而不是速度。我認為這才是重要的。但總的來說，如果你看看過去幾個月甚至幾年，PRV 的價格一直相當穩定在 1 億美元左右。所以我就到此為止，但我們確信我們不會在這裡進行火災逃生。

Excellent. Absolutely not, no. And then maybe given the competitive -- Vyjuvek selling pretty well, getting off to a nice jump has that impacted? Or has there any change to potential looking at a potential partnership rather than a self-launch or some sort of combination thereof on your end? Should -- again, should you get approval?

出色的。絕對不是，不是。然後，也許考慮到競爭激烈——Vyjuvek 賣得相當好，取得了不錯的跳躍，這有影響嗎？或者您在尋找潛在合作夥伴關係而不是自行啟動或兩者的某種組合方面是否有任何變化？應該——再說一次，你應該獲得批准嗎？

Jim, we are very much committed for self-launch, actually, even more so now that Vyjuvek's performance. I mean I think Vyjuvek's performance the first gene therapy what it clearly shows is the willingness for patients and the community to try gene therapies for a genetic disorder like this. So that I think is a class. This is a good sign and the fact that pz-cel, all things that you are aware of in terms of clinical differentiation and everything what we communicated is a very strong value proposition. These patients for their large surface areas require a solution, which pz-cel can offer and the willingness to pay from payer communities is so high for an ultra-rare disease. So it's a profitable business model from that perspective.

吉姆，我們非常致力於自主推出，事實上，鑑於 Vyjuvek 的表現，我們更是如此。我的意思是，我認為 Vyjuvek 的表現是第一個基因療法，它清楚地表明患者和社區願意嘗試針對此類遺傳性疾病的基因療法。所以我認為這是一個類別。這是一個好兆頭，事實上，pz-cel、您在臨床分化方面所了解的所有事情以及我們傳達的所有內容都是一個非常強大的價值主張。這些患者因其表面積大而需要一種解決方案，而 pz-cel 可以提供這種解決方案，而且支付者社區對於這種超罕見疾病的支付意願非常高。所以從這個角度來看，這是一個有利可圖的商業模式。

And so I think this is the right step for Abeona at this time, launching it ourselves and with a finite centers of excellence model, and we have a great team in place. All the people here from -- on commercial team with prior cell therapy and launches and experience and even from market access, having launched Zolgensma and gene therapy experience. So I think we have put together a really good team focused on the launch. And then, of course, we can scale it to other opportunities, as Ram was asking earlier.

因此，我認為這對 Abeona 來說是正確的一步，我們自己推出了它，並建立了有限的卓越中心模型，而且我們擁有一支優秀的團隊。這裡的所有人都來自商業團隊，擁有細胞療法和上市經驗，甚至來自市場准入，擁有推出 Zolgensma 和基因療法的經驗。所以我認為我們已經組建了一支非常優秀的團隊專注於發布。然後，當然，我們可以將其擴展到其他機會，正如拉姆之前所問的那樣。

And then maybe last question here. Any updates on sort of the earlier state -- early state pipeline? Obviously, you guys hit your hands full and the game is getting it through. But any thoughts on your earlier stage and sort of the next key catalysts we should keep an eye on for?

也許還有最後一個問題。關於早期狀態的任何更新—早期狀態管道？顯然，你們已經全力以赴，比賽正在順利進行中。但對於您的早期階段以及我們應該關注的下一個關鍵催化劑有什麼想法嗎？

Yes. Thanks, Jim. Earlier-stage assets, the AAV-based ophthalmology assets that we have. We've continued to generate further numbers in our preclinical data, which are encouraging, especially with our RS1 program and hopefully, in a future scientific conference, we will be able to provide an R&D update with more data there.

是的。謝謝，吉姆。早期資產，我們擁有的基於 AAV 的眼科資產。我們繼續在臨床前數據中產生更多數據，這是令人鼓舞的，特別是在我們的 RS1 計劃中，希望在未來的科學會議上，我們能夠提供包含更多數據的研發更新。

But in terms of any committed development into clinical trials, it's something that's TBD. We'll communicate more about these programs as we get closer to the pz-cel launch, and as I mentioned, just so our organization, which is thin and being is very focused on a successful PV-cell launch. That's really the reason why we haven't spoken so much about our ophthalmology program.

但就任何致力於臨床試驗的開發而言，這都是待定的事情。隨著 pz-cel 發布的臨近，我們將更多地交流這些計劃，正如我所提到的，我們的組織規模較小，非常專注於光伏電池的成功發布。這確實是我們沒有過多談論我們的眼科計畫的原因。

Great. Thank you for taking the questions.

偉大的。感謝您提出問題。

Thank you.

謝謝。

Thank you very much. Well, that appears to be the end of our question and answer session. I will now turn the call back over to Vish for closing remarks.

非常感謝。好吧，我們的問答環節似乎就結束了。現在，我將把電話轉回給 Vish，讓他發表結束語。

Thank you, Jenny. Thank you, everyone, for joining us for today's business update. With that, we'll talk to you again soon.

謝謝你，珍妮。感謝大家接受我們今天的業務更新。這樣，我們很快就會再次與您交談。

Thank you very much. This does conclude today's conference call. You may now disconnect your phone lines and have a wonderful day. Thank you for your participation.

非常感謝。今天的電話會議到此結束。您現在可以斷開電話線並度過美好的一天。感謝您的參與。