Abeona Therapeutics Inc (ABEO) 2023 Q2 法說會逐字稿

Good day, and welcome to the Abeona Therapeutics Second Quarter 2023 Update Conference Call. (Operator Instructions) As a reminder, today's conference is being recorded. I'll now introduce your host for today's conference, Greg Gin, Vice President of Investor Relations and Corporate Communications at Abeona. Sir, please go ahead.

美好的一天，歡迎參加 Abeona Therapeutics 2023 年第二季度更新電話會議。 （操作員指示）提醒一下，今天的會議正在錄製中。現在我將介紹今天會議的主持人，Abeona 投資者關係和企業傳播副總裁 Greg Gin。先生，請繼續。

Thank you, operator, and good morning, everyone. I would like to welcome and thank everyone for joining us on our second quarter 2023 update conference call. The press release announcing the results is available on our website at www.abeonatherapeutics.com. Before we start, 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.

謝謝接線員，大家早上好。我謹歡迎並感謝大家加入我們的 2023 年第二季度更新電話會議。宣布結果的新聞稿可在我們的網站 www.abeonatherapeutics.com 上獲取。在我們開始之前，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。這些前瞻性陳述基於當前的預期，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存在重大差異。

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. Vish Seshadri, Chief Executive Officer; and Joe Vazzano, Chief Financial Officer. Joining us for the Q&A session will be Dr. Madhav Vasanthavada, Vice President, Business Development; and Dr. Brian Kevany, Chief Technical Officer. With that, I will now turn the call over to Vish Seshadri to lead us off. Vish?

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 提交的定期報告中的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 上獲取。首席執行官 Vish Seshadri 博士在今天的電話會議上發表了事先準備好的講話；和首席財務官喬·瓦扎諾。業務開發副總裁 Madhav Vasanthavada 博士將參加我們的問答環節。和首席技術官 Brian Kevany 博士。現在，我將把電話轉給 Vish Seshadri 來帶領我們出發。維什？

Thank you, Greg. Hello, everybody, and thank you for joining us this morning. We are happy to update you on our continued progress, in particular with our lead program, EB-101, our investigational autologous engineered cell therapy had to deliver years of sustained clinical benefit in patients with recessive dystrophic epidermolysis bullosa, or RDEB, in short. Remember that all our clinical data pertain to treatment of large chronic booms, which are the hardest to treat and have remained open for years because they cannot sell to you and inflict the great (inaudible) burden to these patients. The durability of both investigator-assessed [hemin] and patient-reported pain following a single administration of serving toughest RDEB wounds paints a very compelling and unique value proposition for EB-101 for patients.

謝謝你，格雷格。大家好，感謝您今天早上加入我們。我們很高興向您通報我們的持續進展，特別是我們的主導項目EB-101，我們的研究性自體工程細胞療法必須為隱性營養不良性大皰性表皮鬆解症(RDEB) 患者帶來多年的持續臨床益處。請記住，我們所有的臨床數據都與大規模慢性熱潮的治療有關，這是最難治療的，並且多年來一直保持開放狀態，因為它們無法出售給您並給這些患者造成巨大（聽不清）的負擔。在最嚴重的 RDEB 傷口單次給藥後，研究者評估的 [血紅素] 和患者報告的疼痛的持久性為 EB-101 為患者描繪了一個非常引人注目和獨特的價值主張。

We have made significant progress during the last quarter towards planned EB-101 BLA submission. We have completed process performance qualification, or PPQ manufacturing runs for both retroviral vector and EB-101 drug product to demonstrate our validated process and readiness for commercial production. In June, we announced that we had gained alignment with the FDA on the data required to establish retroviral vector or RVV comparability, which is a critical CMC component for the BLA. The FDA has requested additional assay data to establish comparability between RVV sourced from Indiana University and RVV manufactured in-house at Abeona, both of which have been used in the EB-101 clinical study, we have since generated the additional stat that we believe establishes comparability between the 2 vector sources and have included in the briefing package that we submitted to the FDA in July for a pre-BLA meeting later this month.

上個季度我們在計劃的 EB-101 BLA 提交方面取得了重大進展。我們已經完成了逆轉錄病毒載體和 EB-101 藥品的工藝性能鑑定或 PPQ 生產運行，以證明我們經過驗證的工藝和商業生產的準備情況。 6 月，我們宣布我們已與 FDA 就建立逆轉錄病毒載體或 RVV 可比性所需的數據達成一致，這是 BLA 的關鍵 CMC 組成部分。 FDA 已要求提供額外的測定數據，以確定源自印第安納大學的RVV 和Abeona 內部製造的RVV 之間的可比性，兩者均已用於EB-101 臨床研究，此後我們生成了我們認為可以確定的額外統計數據兩種載體來源之間的可比性，並已包含在我們於 7 月份向 FDA 提交的簡報包中，以供本月晚些時候舉行的 BLA 前會議使用。

In that meeting, we will align with the agency on the format content and overall acceptability of the anticipated BLA for EB-101 based on discussion of critical components of the BLA package. Following the conclusion of the meeting and subject to supportive FDA feedback, we anticipate submitting the EB-101 on BLA in the third quarter of 2023. As we expect a priority review based on the anticipated timing of the BLA submission, we expect potential BLA approval in the second quarter of 2024. If the BLA is approved, we anticipate being granted a priority in review voucher, which is worth approximately $100 million based on recent PRV transactions. In parallel, with our BLA-related activities, we're looking ahead to EB-101's potential FDA approval and launch next year.

在那次會議上，我們將根據對 BLA 包關鍵組成部分的討論，就 EB-101 預期 BLA 的格式內容和整體可接受性與該機構保持一致。會議結束後，並根據 FDA 的支持反饋，我們預計於 2023 年第三季度提交關於 BLA 的 EB-101。由於我們預計將根據 BLA 提交的預期時間進行優先審查，因此我們預計 BLA 可能會獲得批准2024年第二季度。如果BLA 獲得批准，我們預計將獲得優先審核憑證，根據最近的PRV 交易，該憑證價值約為1 億美元。與此同時，在開展 BLA 相關活動的同時，我們期待 EB-101 明年可能獲得 FDA 批准並上市。

In anticipation of approval, we raised $25 million in a registered direct offering priced at the market with select existing investors in July with the proceeds from the offering, we're ready to invest in pre-commercialization activities and the planned launch of EB-101 in the U.S. by being thoughtful about what we need to spend now versus later after day approval. We have already initiated dialogue with top EB treatment centers in the U.S. about onboarding EB-101 as a treatment option, and we're encouraged by the level of interest. Physicians indicate there is a high unmet need in debt and particularly for a treatment like EB-101 that has a compelling value proposition. The positive feedback we hear from KOLs is consistent with what you've heard from payers, hospital administrators and patient advocacy groups. We plan to continue to engage with payers, both commercial and Medicaid to ensure broad market actions after launch.

為了獲得批准，我們在 7 月份通過註冊直接發行籌集了 2500 萬美元，該發行的價格按市場價格與部分現有投資者進行，我們已準備好投資預商業化活動和計劃推出的 EB-101在美國，我們要考慮現在需要花什麼，而不是稍後批准。我們已經開始與美國頂級 EB 治療中心就將 EB-101 作為一種治療選擇進行對話，我們對大家的興趣感到鼓舞。醫生表示，債務方面的需求尚未得到滿足，尤其是像 EB-101 這樣具有令人信服的價值主張的治療方法。我們從 KOL 那裡聽到的積極反饋與您從付款人、醫院管理人員和患者權益團體那裡聽到的一致。我們計劃繼續與商業和醫療補助付款人合作，以確保推出後廣泛的市場行動。

In addition to preparing the market for EB-101, we're also preparing our organization for commercialization. We have mapped out key commercial and medical roles that we plan to fill in the second half of this year and the capabilities we will build to support a successful launch. During the second quarter, additional efficacy and safety data from the EB-101 pivotal Phase III VIITAL study was reported at the International Society for Investigative Dermatology, or ISID and Society for Pediatric Dermatology SPD meetings. The results presented at ISID and SPD showed that EB-101 improved wound healing and pain reduction at 6, 12 and 24 weeks compared to control wounds following a onetime application of EB-101.

除了為 EB-101 準備市場外，我們還在為我們的組織商業化做準備。我們已經規劃了計劃在今年下半年填補的關鍵商業和醫療角色，以及我們將為支持成功發布而建立的能力。第二季度，國際皮膚病研究學會 (ISID) 和兒科皮膚病學會 SPD 會議上報告了 EB-101 關鍵 III 期 VIITAL 研究的額外療效和安全性數據。在 ISID 和 SPD 上公佈的結果表明，與一次性使用 EB-101 後的對照傷口相比，EB-101 在 6、12 和 24 週時改善了傷口癒合並減輕了疼痛。

Furthermore, EB-101 demonstrated improvement in patient-reported and caregiver reported outcomes for itch and blistering severity. Let's turn briefly to our preclinical ophthalmology programs. We're excited by the broad potential for treating serious eye diseases with new AAV-based therapies using novel AAV capsids from our in-licensed AIM capsid library and internal research. At ASGCT, we presented 3 posters highlighting encouraging findings from animal proof-of-concept experiments for investigative AAV-based gene therapies for Stargardt disease, X-linked retinitis and autosomal dominant optic atrophy -- the preclinical proof-of-concept data provides early evidence of the potential of our proprietary AAV capsid and gene constructs to express the recombinant protein and target tissues and rescue mutant phenotypes in mouse disease models.

此外，EB-101 證明患者報告和護理人員報告的瘙癢和水皰嚴重程度的結果有所改善。讓我們簡單回顧一下我們的臨床前眼科項目。我們對使用來自我們許可的 AIM 衣殼庫和內部研究的新型 AAV 衣殼進行基於 AAV 的新型療法治療嚴重眼部疾病的廣泛潛力感到興奮。在ASGCT 上，我們展示了3 張海報，重點介紹了針對Stargardt 病、X 連鎖視網膜炎和常染色體顯性視神經萎縮的基於AAV 的基因療法的動物概念驗證實驗的令人鼓舞的發現——臨床前概念驗證數據提供了早期證據表明我們專有的 AAV 衣殼和基因構建體在表達重組蛋白和靶組織並挽救小鼠疾病模型中的突變表型方面具有潛力。

We completed pre-IND meetings with the FDA regarding preclinical development plans and regulatory requirements to support first-in-human trials for 2 preclinical gene therapy product candidates from our AAV ophthalmology program. We're gathering additional proof-of-concept data before committing to IND-enabling tox studies, which we anticipate initiating in the second half of 2023. I'd now like to turn the call over to our Chief Financial Officer, Joe Vazzano, who will review the second quarter financial results and recent capital rates. Joe?

我們與 FDA 完成了關於臨床前開發計劃和監管要求的 IND 前會議，以支持我們 AAV 眼科項目的 2 種臨床前基因治療產品候選者的首次人體試驗。在致力於支持 IND 的毒物研究之前，我們正在收集更多的概念驗證數據，預計該研究將於 2023 年下半年啟動。我現在想將電話轉給我們的首席財務官 Joe Vazzano，誰將審查第二季度的財務業績和最近的資本利率。喬？

Thanks, Vish. I would like to remind everyone that the Form 10-Q is available on our website, which is where you can get additional details on our financial results for the 3 and 6 months ended June 30, 2023. Starting with the financial resources on our balance sheet. We had cash, cash equivalents, restricted cash and short-term investments of $37.1 million as of June 30, 2023, prior to the $25 million registered direct offering in July of 2023 as compared to $40.7 million as of March 31, 2023. Based on our current operating plan and assumptions, our estimated current financial resources, including the proceeds from the $25 million registered direct offering in July of 2023, are sufficient to fund our commercial launch preparations for EB-101 and our business operations into the fourth quarter of 2024. In other words, our cash runway extends beyond the potential commercial launch of EB-101 and receipt of a priority review voucher.

謝謝，維什。我想提醒大家，我們的網站上提供了10-Q 表格，您可以在其中獲取截至2023 年6 月30 日的3 個月和6 個月財務業績的更多詳細信息。首先從我們餘額中的財務資源開始床單。截至2023 年6 月30 日，我們的現金、現金等價物、限制性現金和短期投資為3,710 萬美元，在2023 年7 月註冊直接發行為2,500 萬美元之前，截至2023 年3 月31 日為4,070 萬美元。我們當前的運營計劃和假設、我們估計的當前財務資源，包括2023 年7 月註冊的2500 萬美元直接發行的收益，足以為我們EB-101 的商業啟動準備工作以及我們到2024 年第四季度的業務運營提供資金換句話說，我們的現金跑道超出了 EB-101 的潛在商業推出和獲得優先審查憑證的範圍。

License and other revenues for the second quarter of 2023 were $3.5 million compared to $1 million in the second quarter of 2022. The revenue in the second quarter of 2023 resulted from a clinical milestone achieved under a sub (inaudible) agreement with taysha Gene Therapies related to an investigational AAV-based gene therapy for RETT syndrome. Turning to research and development activities. We spent $8.5 million for the 3 months ended June 30, 2023, compared to $6.7 million in the 3 months ended June 30, 2022. Our spend on general and administrative activities was $5.0 million for the 3 months ended June 30, 2023, compared to $3.5 million in the same period of 2022. Net loss attributable to common shareholders was $16.7 million for the second quarter of 2023 or $0.92 loss per common share as compared to a net loss attributable to common shareholders of $7.9 million or $1.36 loss per common share in the second quarter of 2022.

2023 年第二季度的許可和其他收入為350 萬美元，而2022 年第二季度為100 萬美元。2023 年第二季度的收入源於與taysha Gene Therapies 相關的子（聽不清）協議下實現的臨床里程碑一種針對 RETT 綜合徵的基於 AAV 的研究性基因療法。轉向研究和開發活動。截至2023年6月30日止的三個月，我們花費了850萬美元，而截至2022年6月30日的三個月為670萬美元。截至2023年6月30日的三個月，我們的一般和行政活動支出為500萬美元，而截至2023年6月30日止的三個月，我們的一般和行政活動支出為500萬美元。 2022 年同期為350 萬美元。2023 年第二季度普通股股東應占淨虧損為1670 萬美元，每股普通股虧損0.92 美元，而2023 年第二季度普通股股東應占淨虧損為790 萬美元，每股普通股虧損1.36 美元。 2022 年第二季度。

With that, I'll turn the call back over to the operator for the Q&A session. Operator?

這樣，我會將電話轉回給接線員進行問答環節。操作員？

(Operator Instructions). Thank you. Our first question is coming from Maury Raycroft with Jefferies.

（操作員說明）。謝謝。我們的第一個問題來自杰弗里斯的莫里·雷克羅夫特。

Congrats on the progress. Just wondering, based on the briefing package and additional RVV data that you submitted to FDA. Have you gotten any recent feedback yet based on those -- that submission? And also as a follow-up, are you providing any more granularity on the pre-BLA meeting agenda topics where you need to align and also just on timing of the meeting.

祝賀取得的進展。只是想知道，根據您向 FDA 提交的簡報包和其他 RVV 數據。您最近收到過基於這些提交的反饋嗎？另外，作為後續行動，您是否提供了需要協調的 BLA 前會議議程主題以及會議時間的更多詳細信息。

Thank you for your question, Maury. I'll start with your first question, which is have we had any further discussion about the briefing package for the pre-BLA meeting itself? And our focus so far, just as a reminder, we've been having a lot of collaborative interactions with the FDA since December of last year after we had our pivotal data out there. And over those 6, 7 months, we've had a lot of communication, made a lot of progress on various aspects of the BLA submission. And to remind you, the big -- 2 big items on the CMC side were the PPQ run completion as well as establishing the RVV comparability. What we had agreed an informal meeting before even making our pre-BLA briefing book submission was the outstanding work that had to be done with assays to establish the RVV comparability.

謝謝你的問題，莫里。我將從你的第一個問題開始，即我們是否對 BLA 前會議本身的簡報包進行了進一步的討論？到目前為止，我們的重點是，提醒一下，自去年 12 月獲得關鍵數據後，我們一直與 FDA 進行了大量的合作互動。在這 6、7 個月裡，我們進行了很多溝通，在 BLA 提交的各個方面都取得了很多進展。提醒您，CMC 方面的兩大重點是 PPQ 運行完成以及建立 RVV 可比性。在提交 BLA 前簡報之前，我們就召開了一次非正式會議商定，必須通過分析來完成確定 RVV 可比性的出色工作。

And we believe we've done that to the satisfaction of the agency because we had agreed on what exactly needed to be done and what the data outcome needed to be. like. So based on the results that we have seen, we're fairly confident that the comparability of RVV from the 2 vector sources is adequately supportive. So the big focus areas of discussion between the FDA and Abeona has been covered with that data that we submitted in the pre-BLA -- as we speak, they are still reviewing. We haven't had any interactions since the submission itself. Now the second part of the question, Maury, in terms of the specifics of the pre-BLA meeting itself, our goals are twofold.

我們相信我們已經做到了令該機構滿意的事情，因為我們已經就到底需要做什麼以及數據結果需要是什麼達成了一致。喜歡。因此，根據我們所看到的結果，我們相當有信心來自 2 個矢量源的 RVV 的可比性具有充分的支持性。因此，FDA 和 Abeona 之間討論的重點領域已涵蓋在我們在 BLA 預審中提交的數據中——就在我們發言時，他們仍在審查。自提交以來我們沒有進行任何互動。現在是問題的第二部分，莫里，就 BLA 會議本身的具體細節而言，我們的目標是雙重的。

One is the format content and overall acceptability of the BLA package is that appropriate? And two, we want a confirmatory answer that the CMC data that we've put together as well as the clinical data that we have put together are sufficient to submit a BLA. Those are the 2 answers that we need to hear from the FDA formally. And we're fairly confident we're making -- we're on the right track. Based on all the last 7 months of discussion we've had on various topics that relate to both CMC and clinical. The only piece on the clinical side that I would add is the FDA has seen the top line data already and gave us some feedback -- preliminary feedback on how we would pool the data from the 2 different studies. We have done that, and this is the first time that they are reviewing the combined consolidated data as part of the briefing book. So we haven't had interactions since we submitted the briefing book. I hope that answered your question.

一是BLA包的格式內容和整體可接受性是否合適？第二，我們想要一個確認性的答案，即我們匯總的 CMC 數據以及我們匯總的臨床數據足以提交 BLA。這是我們需要從 FDA 正式聽到的兩個答案。我們對我們正在做的事情充滿信心——我們走在正確的軌道上。基於過去 7 個月的所有討論，我們就與 CMC 和臨床相關的各種主題進行了討論。在臨床方面，我要補充的唯一一點是，FDA 已經看到了最重要的數據，並給了我們一些反饋——關於我們如何匯總兩項不同研究的數據的初步反饋。我們已經做到了這一點，這是他們第一次將合併後的數據作為簡報的一部分進行審查。所以自從我們提交簡報以來我們就沒有進行過互動。我希望這回答了你的問題。

Yes. Yes, that's definitely a helpful perspective. And maybe one other question, if I may. You said that you will be commercializing 101 by yourself in the United States. Can you just talk a little bit more about where you're at with the commercial -- or if you could talk more about what the commercial launch rollout will look like and where you're at with CMC scale up for the launch? And if that's something that you can do before getting approved? Or would you wait for that approval to scale up.

是的。是的，這絕對是一個有用的觀點。如果可以的話，也許還有一個問題。你說你將在美國自己將101商業化。您能否多談談您在商業廣告方面的進展情況，或者您是否可以更多地談談商業發布的情況以及您在 CMC 擴大發布方面的進展情況？如果這是你在獲得批准之前可以做的事情嗎？或者你會等待批准擴大規模。

Great. So your 2-part question. One was about our general commercial launch preparation itself. I'm going to let Dr. Madhav Vasanthavada comment on that because he's been spearheading a lot of that planning. But I'll first address your second question, which is the CMC manufacturing buildup. As we have previously communicated, we are currently set up to manufacture up to about 120 manufacturing cycles in a year, which translates to 120 patients, we only consider onetime treatment for each patient.

偉大的。所以你的問題分為兩部分。其中之一是關於我們的一般商業發布準備本身。我將讓 Madhav Vasanthavada 博士對此發表評論，因為他一直是許多此類計劃的帶頭人。但我首先要解決你的第二個問題，即 CMC 製造建設。正如我們之前所傳達的，我們目前每年的生產量約為 120 個製造週期，即 120 名患者，我們只考慮為每位患者提供一次治療。

Now we are not -- we're going to be very careful and choiceful in how and when we trigger undertaking capacity expansion because as a reminder, we have the PRV proceeds that we anticipate upon approval that could further fund our capacity expansion. And so we believe that the current capacity is sufficient, and we will definitely hire appropriately to have that operating scale and full operation there. But in terms of how we are preparing for the commercial launch itself, I'll let Madhav add some comments there, Madhav.

現在我們不是——我們將非常謹慎和選擇如何以及何時觸發進行產能擴張，因為提醒一下，我們預計在批准後可以獲得 PRV 收益，這可以進一步為我們的產能擴張提供資金。所以我們認為目前的產能是足夠的，我們一定會適當的招聘，以達到這樣的運營規模和全面運營。但就我們如何準備商業發布本身而言，我將讓 Madhav 在那裡添加一些評論，Madhav。

Yes. Thanks, Vish. And thanks, Maury, for that question. So as Vish mentioned, we've initiated pre-commercial activities, essentially with the goal to prioritize the sequence of events that we will trigger as we approach towards BLA approval. But just to give a flavor, we are looking to understand and sort of prepare the market on the one hand for EB-101 launch in the U.S. as well as prepare the organization. And the fact that VYJUVEK has already now gotten approved and patients are beginning to get identified is actually an excellent news for us because this is, like we mentioned earlier, is helping raise the awareness, identify these patients, and that's going to be a good cursor for us.

是的。謝謝，維什。謝謝莫里提出這個問題。正如 Vish 提到的，我們已經啟動了預商業活動，主要目標是確定在接近 BLA 批准時觸發的事件順序的優先順序。但為了讓大家了解一下，我們一方面希望了解並為 EB-101 在美國的推出做好市場準備，另一方面也為組織做好準備。 VYJUVEK 現在已經獲得批准並且患者開始得到識別這一事實對我們來說實際上是一個好消息，因為正如我們之前提到的，這有助於提高認識，識別這些患者，這將是一個好消息我們的光標。

Meanwhile, while we're waiting for the approval, we are already going ahead and engaging with certain top EB centers, treatment centers to discuss EB-101 as a potential treatment option in their centers because this is a hospital and patient procedure. And we are seeing a lot of interest from them to engage because these centers, they both -- they are treating both pediatric patients as well as adult patients, and they are expressing a high unmet need in depth. And as we all know, the EB-101 clinical profile is extremely compelling, given the onetime procedural nature and the long-term durability. So these providers, the feedback that we are getting from them is helpful.

與此同時，在我們等待批准的同時，我們已經開始與某些頂級 EB 中心、治療中心合作，討論 EB-101 作為他們中心的潛在治療選擇，因為這是一個醫院和患者的程序。我們看到他們有很大的興趣參與，因為這些中心，他們都在治療兒科患者和成人患者，並且他們深入表達了高度未滿足的需求。眾所周知，鑑於一次性程序性質和長期耐久性，EB-101 的臨床概況非常引人注目。因此，我們從這些提供商那裡得到的反饋很有幫助。

But besides providers, Maury, we're also engaging with the payer groups, as you may recall, the initial engagement we've had with Insurance companies was very helpful for us, and we want to continue this engagement as we go towards the launch so that we are fully equipped to ensure a broad market access and a rapid market access immediately post approval in the U.S. So we talked about the payers. We talked about providers. And then final piece, I would also say is from a CMS standpoint, we have already initiated activities to ensure there is a new technology add-on payments because this is a hospital procedure. Securing this NTAP is going to be extremely helpful because it provides additional payments to hospitals for Medicare-covered patients.

但是，除了提供商之外，莫里，我們還與付款人團體進行接觸，您可能還記得，我們與保險公司的最初接觸對我們非常有幫助，我們希望在推出產品時繼續這種接觸這樣我們就完全有能力確保在美國獲得批准後立即獲得廣泛的市場准入和快速的市場准入。所以我們談到了付款人。我們討論了提供商。最後一點，我還要說的是，從 CMS 的角度來看，我們已經啟動了一些活動，以確保有新技術附加付款，因為這是醫院程序。獲得此 NTAP 將非常有幫助，因為它為醫療保險覆蓋的患者向醫院提供額外付款。

So these are all the activities and many more that we have ongoing and planned to help prepare the market. And from an organizational standpoint, we have already mapped out, as Vish mentioned earlier, some of the key commercial roles, medical roles that we plan to fill in this year as well as next year and some additional capabilities that we're going to be putting in place for a successful launch. And the fact that we are coming with an autologous cell therapy launch experience. Having done that recently, we know really a strong network of people and the talent that are there and just eager to get them on board. Hope that addresses Maury.

這些是我們正在進行和計劃幫助準備市場的所有活動以及更多活動。從組織的角度來看，正如 Vish 之前提到的，我們已經規劃好了我們計劃今年和明年填補的一些關鍵商業角色、醫療角色以及我們將要具備的一些額外能力。為成功發射做好準備。事實上，我們即將推出自體細胞療法。最近這樣做後，我們確實了解了一個強大的人員網絡和人才，並且渴望讓他們加入。希望這能解決莫里的問題。

Yes, very helpful.

是的，非常有幫助。

Our next question is coming from Kristen Kluska with Cantor Fitzgerald.

我們的下一個問題來自克里斯汀·克魯斯卡和坎托·菲茨杰拉德。

First question, just given that the RDEB community is pretty tightened it and aware of the clinical landscape. What's your expectation on how much awareness there will be moving closer to an approval decision? I guess I understand that you're going to be building a sales force and getting the word out there. But do you also anticipate this to be one of the situations where you can get people coming inbound to you?

第一個問題，鑑於 RDEB 社區相當嚴格並且了解臨床情況。您對接近批准決定的認識程度有何期望？我想我知道您將建立一支銷售隊伍並進行宣傳。但您是否也預計這會成為吸引人們來找您的情況之一？

Kristen, and thank you for your question. Again, I think Madhav is the appropriate person to address this matter.

克里斯汀，謝謝你的提問。再說一次，我認為馬達夫是解決這個問題的合適人選。

Absolutely. I think at the time of launch, we do expect there will be self referrals and the awareness that we will have created. As you rightly mentioned, patient advocacy groups are really powerful in this setting, and they've been doing a lot of reports. Debra and PeDRA in particular, have been fantastic partners. We are engaging with them. And as we go closer to launch, we expect greater awareness, especially in these centers of excellence. We know that these patients travel, they travel distances to come to get their treatments done and our approach in the outer years or in outer months post launch will be to take advantage of the sales team in the non-sort of centers of excellence and raising awareness with the community-based physicians and making sure that patients understand where these treatments are available. So for sure, more to come, but it's a great starting point, tighten patient community and some amount of awareness that will already have been generated.

絕對地。我認為在發佈時，我們確實希望會有自我推薦和我們所創造的意識。正如您正確地提到的，患者權益團體在這種情況下確實非常強大，他們已經做了很多報告。尤其是 Debra 和 PeDRA，它們一直是出色的合作夥伴。我們正在與他們接觸。隨著我們距離發布越來越近，我們預計會有更多的認識，尤其是在這些卓越中心。我們知道這些患者會旅行，他們長途跋涉來完成治療，我們在推出後的外部幾年或外部幾個月的方法將是利用非卓越中心的銷售團隊，並提高提高社區醫生的認識，並確保患者了解哪裡可以提供這些治療。因此，可以肯定的是，還會有更多的事情發生，但這是一個很好的起點，可以加強患者社區和已經產生的一定程度的意識。

Okay. And then I wanted to ask you on epidemiology. I know that this is a rare disease, but not that rare to the point where you have a good sense of how many patients are available. So first, I wanted to ask how you think, given the landscape with 2 potential commercial therapies could help impact or perhaps drive awareness on epidemiology. And then given this impressive data set that you recently had in Japan, I'm wondering if you've had people approach you to perhaps look at markets besides the usual U.S. and Europe that we would think about.

好的。然後我想問你流行病學的問題。我知道這是一種罕見的疾病，但也沒有那麼罕見，以至於你可以很好地了解有多少患者。首先，我想問您如何看待，因為兩種潛在的商業療法可能有助於影響或提高對流行病學的認識。然後考慮到您最近在日本獲得的令人印象深刻的數據集，我想知道您是否有人聯繫過您，以考察我們通常會考慮的美國和歐洲以外的市場。

Yes. I think the way we are looking at this opportunity is by wounds, right? And that's what we are hearing from physicians that it's not just about the number of patients, but it's the different types of wounds that each patient has got. And we are hearing from physicians that both these therapies are for different types of wounds. And so if you look at the current base of bolus patients and/or prevalent patients, we have estimates of up to 350, which is what are the reported numbers. But as we know that about 1,100 is what has been identified by Crystal, who have already gone to the centers of excellence. So from our standpoint at the time of launch and given our manufacturing capacity, we think these hundreds of thousands of patients which are out there.

是的。我認為我們看待這個機會的方式是通過傷口，對嗎？這就是我們從醫生那裡聽到的，這不僅與患者的數量有關，還與每個患者的傷口類型不同有關。我們從醫生那裡得知，這兩種療法適用於不同類型的傷口。因此，如果您查看當前推注患者和/或患病患者的基數，我們估計最多有 350 名患者，這就是報告的數字。但據我們所知，Crystal 已經確定了大約 1,100 名員工，他們已經前往卓越中心。因此，從我們推出時的角度來看，考慮到我們的製造能力，我們認為現有的患者有數十萬。

For us, we are going to be well satiated with the patient numbers out there in terms of the way we will expand our capacity ramp up. And so that's what is very encouraging at least from our perspective of supplying these patients. And as we have more therapies and awareness, the diagnosis also we expect to increase. And many a times, these patients under ICD code 81.2 is what is used for RDEB, but several patients are also misdiagnosed or put in other classifications. So I think having the right diagnosis and awareness is going to help identify RDEB patients in particular more so. With regards to other than U.S. markets, absolutely, we do have interest that is coming in from pharma companies in Europe, as well as actually in Japanese markets where there is interest and those discussions are ongoing to see as to when is the right time to look into expansion.

對於我們來說，就我們擴大產能的方式而言，我們將對現有的患者數量感到滿意。因此，至少從我們為這些患者提供服務的角度來看，這是非常令人鼓舞的。隨著我們有更多的治療方法和認識，我們預計診斷也會增加。很多時候，這些 ICD 代碼 81.2 下的患者就是用於 RDEB 的患者，但也有一些患者被誤診或被歸入其他分類。因此，我認為正確的診斷和認識將有助於識別 RDEB 患者，尤其如此。至於除美國市場以外的市場，我們確實對歐洲的製藥公司感興趣，實際上對日本市場也有興趣，這些討論正在進行中，以確定何時是合適的時機考慮擴張。

Yes. And just to add to that, Kristen, we will be looking for partnerships to go ex U.S. because right now, we're so laser-focused on getting the U.S. launch to BLA and preparing for the U.S. launch and for a company with finite resources like us, we have to depend on creative partnerships to get this to many patients. But in terms of interest and demand, we are already seeing that happening, right? So we're excited by that. We believe that the important catalysts that will take those dialogues to the next level is when we reach a milestone in the U.S., like a BLA acceptance or a BLA approval that's going to trigger a lot more conversation along those lines and the ex U.S. conversation will become prime time at that point in time. So right now, we're just putting our heads down to see how to best maximize the U.S. opportunity and address that unmet need first.

是的。除此之外，Kristen，我們將尋找走出美國的合作夥伴，因為現在，我們非常專注於將美國的發布推向BLA，並為美國的發布做好準備，並為一家資源有限的公司做好準備。像我們一樣，我們必須依靠創造性的合作夥伴關係才能將其帶給許多患者。但就興趣和需求而言，我們已經看到這種情況發生了，對吧？所以我們對此感到興奮。我們相信，將這些對話提升到新水平的重要催化劑是當我們在美國達到一個里程碑時，例如 BLA 的接受或 BLA 的批准，這將引發更多類似的對話，而前美國的對話將成為該時間點的黃金時段。因此，現在我們只是埋頭思考如何最大限度地利用美國的機會並首先解決未滿足的需求。

Great. And wishing you all the best this quarter.

偉大的。祝您本季度一切順利。

Thank you, Chris.

謝謝你，克里斯。

Thank you. Our next question is coming from David Bautz with Zacks Small Capital Research.

謝謝。我們的下一個問題來自 Zacks Small Capital Research 的 David Bautz。

Good morning, everyone. Crystal Biotech recently announced that they had received 121 patient start forms since launching BYJUVEC about 6 weeks ago. So I'm curious if you could just comment on what your thoughts are on that number, maybe in regards to potential market size and patient access? And then how we should think about that in terms of the launch of EB-101.

大家，早安。 Crystal Biotech 最近宣布，自大約 6 週前推出 BYJUVEC 以來，他們已收到 121 份患者啟動表格。所以我很好奇您是否可以評論一下您對這個數字的看法，也許是關於潛在的市場規模和患者的獲取機會？然後我們應該如何考慮 EB-101 的推出。

Yes, thank you for that question. It's very early for us to speculate translating 120 patient forms into what the full market opportunity is. It's still early in their launch, and we're also learning. But I'll turn it over to Madhav to speak about his experience because he's really leading our launch preparations here.

是的，謝謝你提出這個問題。對於我們來說，推測將 120 份患者表格轉化為完整的市場機會還為時過早。他們的推出還處於早期階段，我們也在學習。但我會把它交給 Madhav 來談談他的經歷，因為他確實在這裡領導了我們的發射準備工作。

Yes. So thanks, David. I think it's for sure, encouraging. I know that they had both the dominant and recessive form, and I think this split was roughly 30 some patients having the dominant version. And it's in line with where our expectations are also and as the -- I don't want to talk much about their launch preparations. But from what I'm seeing on the public domain as insurance companies open up greater access and more centers having understanding this product, there's going to be a greater adoption curve. So I think this is all very encouraging for us. And given that we have another several more months to go before EB-101 gets launched, I think it's a good setup and we are encouraged to hearing what this feedback are going to be from patients as they continue to get these therapies and better understand ensuring that they understand the value proposition of EB-101, which is a onetime therapy that can offer several years of durable benefit for their larger set wounds. Does that help, David? Or...

是的。謝謝，大衛。我認為這是肯定的，令人鼓舞的。我知道他們同時具有顯性和隱性形式，而且我認為這一比例大約有 30 名患者俱有顯性形式。這也符合我們的期望，而且我不想過多談論他們的發射準備工作。但從我在公共領域看到的情況來看，隨著保險公司開放更多的渠道和更多的中心了解該產品，採用曲線將會更大。所以我認為這對我們來說非常令人鼓舞。考慮到我們在 EB-101 上市之前還有幾個月的時間，我認為這是一個很好的設置，我們很高興聽到患者的反饋，因為他們繼續接受這些療法並更好地了解確保他們了解EB-101 的價值主張，這是一種一次性療法，可為較大的固定傷口提供數年的持久益處。這有幫助嗎，大衛？或者...

Yes, that's very helpful. Now you had also mentioned that you completed the process performance qualification manufacturing run as part of the CMC package. So I'm curious, were there a certain number of runs that you needed to complete there as per the FDA what they would require? And then are there any additional CMC activities that need to occur prior to filing the BLA?

是的，這非常有幫助。現在您還提到您已完成工藝性能鑑定製造運行，作為 CMC 包的一部分。所以我很好奇，根據 FDA 的要求，您是否需要完成一定數量的運行？那麼在提交 BLA 之前是否需要進行任何其他 CMC 活動？

Sure. There is an agreement on the number of process performance qualification runs that have to be completed, which is 10 manufacturing runs, but 3 of those runs had to be done prior to BLA approval in a consecutive 3 run fashion. So we have completed, in fact, 4 PPQ runs. So we have what we need for a BLA submission per se. And then the remaining runs to make up to the 10 can continue even post approval or between now and approval even. So we have multiple different time points to complete that obligation. In terms of your other question, which is, are there other CMC activities that need to be completed.

當然。對於必須完成的工藝性能鑑定運行次數達成了一致，即 10 次製造運行，但其中 3 次運行必須在 BLA 批准之前以連續 3 次運行的方式完成。事實上，我們已經完成了 4 次 PPQ 運行。這樣我們就擁有了提交 BLA 所需的一切。然後，剩餘的運行甚至可以在批准後或從現在到批准之間繼續進行，以達到 10。因此，我們有多個不同的時間點來完成該義務。至於你的另一個問題，即是否還有其他 CMC 活動需要完成。

We have completed the last set of CMC activities that we had agreed with the agency, especially because the retroviral vector comparability package that we had discussed. That and the PPQ runs where the biggest items on the list. We've had multiple discussions on many -- very many other topics. One example I can give you is transportation and stability associated. So when we manufacture for a patient, we run a batch fly it to Florida or distant parts of the country and bring it back and after various standpoints, we look for stability of the drug product, and we're happy with what we're seeing that's all being written up as part of the BLA package in Module 3. So we have completed the work that needs to be done. We're almost complete with writing it up with all the data that's necessary. So we feel fairly confident that things are coming together for a Q3 submission here. I hope that gives you an idea.

我們已經完成了與該機構商定的最後一組 CMC 活動，特別是因為我們已經討論過逆轉錄病毒載體可比性包。 PPQ 運行在列表中最大的項目上。我們就許多其他主題進行了多次討論。我可以舉的一個例子是交通和穩定性的相關性。因此，當我們為患者生產時，我們會批量將其空運到佛羅里達州或該國的偏遠地區，然後將其帶回，經過各種立場後，我們尋求藥品的穩定性，我們對我們的產品感到滿意看到這一切都被寫成模塊 3 中 BLA 包的一部分。所以我們已經完成了需要完成的工作。我們幾乎已經完成了所有必要數據的編寫。因此，我們對第三季度的提交工作進展順利充滿信心。我希望這能給你一個想法。

Yes, sounds great.

是的，聽起來很棒。

Thank you -- our next question is coming from James Molloy with Alliance Global Partners.

謝謝——我們的下一個問題來自聯盟全球合作夥伴的詹姆斯·莫洛伊。

So this is (inaudible) filing in for James Molloy. So for EB-101, are you looking into publishing the additional RVV compatibility data that was requested by the FDA? Or will it fully remain in the briefing package and the upcoming BLA submission?

這是（聽不清）詹姆斯·莫洛伊的申請。那麼對於 EB-101，您是否考慮發布 FDA 要求的額外 RVV 兼容性數據？或者它會完全保留在簡報包和即將提交的 BLA 中嗎？

Thank you. We are not planning per se to Publish this as a peace meal, but definitely, it's going to be part of our module 3, which goes into the BLA. Without getting too technical about it, I would just like to mention that we have preset goals on what comparability means with the FDA. And we believe we're in a good position to establish that comparability based on the results that we've generated. So that being said, in terms of scientific advancement, this is one piece that is important. There are so many other pieces that speak to retroviral vectors. We're talking about potency, identity, stability, drug product level potency as well. So there are numerous different parameters. Holistically, the data can potentially make it to the public domain eventually, but we haven't yet made a decision on that.

謝謝。我們本身並不打算將其作為和平餐發布，但毫無疑問，它將成為我們模塊 3 的一部分，該模塊包含在 BLA 中。在不涉及太多技術性的情況下，我只想提一下，我們對與 FDA 的可比性意味著什麼有預設的目標。我們相信，我們能夠根據我們生成的結果建立這種可比性。話雖如此，就科學進步而言，這是一件很重要的事情。還有很多其他與逆轉錄病毒載體相關的作品。我們還討論效力、同一性、穩定性、藥品水平的效力。所以有很多不同的參數。總體而言，這些數據最終有可能進入公共領域，但我們尚未就此做出決定。

Got it. And then also with the study starts to be initiated by the end of the year for your other -- for your AAV gene therapy program. What might be the study protocols here? And then what indications might you be looking at what ophthalmology indications are you looking on to targeting for (inaudible).

知道了。然後，這項研究也將在今年年底開始啟動，用於您的其他 AAV 基因治療計劃。這裡的研究方案可能是什麼？然後您可能會考慮哪些適應症，以哪些眼科適應症為目標（聽不清）。

Yes. The preclinical data that we have, as I mentioned, are for Stargardt disease, we have X-linked retinitis and autosomal dominant optic atrophy -- for all the 3 disease areas, there is what we've shown so far is proof of expression of the transgene in the right compartment of (inaudible). So we need to first -- the first step that we need to sales is in a larger sample set of animals confirm the results that we've seen. And we're being very up-gated and in how we're going to commit our resources because our focus is laser-focused on EB-101. Having said that, our target is still to have first-in-human patients in the second half of next year, so approximately a year from now. The feedback we just received from the FDA on what it takes to have an IND approved definitely has given us an operating plan of what we need to do in steps.

是的。正如我所提到的，我們擁有的臨床前數據是關於 Stargardt 病、X 連鎖視網膜炎和常染色體顯性視神經萎縮——對於所有 3 個疾病領域，我們迄今為止所展示的是表達的證據右側隔室中的轉基因（聽不清）。所以我們需要首先——我們需要銷售的第一步是在更大的動物樣本中確認我們所看到的結果。我們對如何投入資源非常重視，因為我們的重點是 EB-101。話雖如此，我們的目標仍然是在明年下半年，也就是從現在開始大約一年後，首次出現在人類身上。我們剛剛從 FDA 收到的有關 IND 批准所需條件的反饋肯定為我們提供了需要分步驟進行的操作計劃。

And as I had -- just along the lines of the previous comments we made, we first have additional proof-of-concept experiments in animals before we trigger GLP, IND-enabling tox, which still should start at least for 1 or 2 indications in the second half of this year. And we're going to be opportunistic on when we trigger it for all our programs because we have numerous different funding sources that we could tap into to further these programs, and that's something that we should be ready to talk about in the last quarter of this year.

正如我所言，正如我們之前所做的評論一樣，我們首先在動物身上進行額外的概念驗證實驗，然後再觸發 GLP、IND 啟用的毒素，這仍然應該至少針對 1 或 2 個適應症開始今年下半年。當我們為所有計劃觸發它時，我們將抓住機會，因為我們有許多不同的資金來源，我們可以利用它們來推進這些計劃，這是我們應該準備在最後一個季度討論的事情。今年。

Thank you. (Operator Instructions). As we have no further questions, I will hand it back to Mr. Seshadri for any closing comments you may have.

謝謝。 （操作員說明）。由於我們沒有其他問題，我會將其交還給 Seshadri 先生，以供您提出任何結束意見。

Thank you, Ali. In closing, I want to thank our shareholders and all our other stakeholders who listened to this call, and we'll talk to you again soon.

謝謝你，阿里。最後，我要感謝我們的股東和所有其他利益相關者聽取了這次電話會議，我們很快就會再次與您交談。

This does conclude today's conference, and you may disconnect your lines at this time, and we thank you for your participation.

今天的會議到此結束，此時您可以掛斷電話，我們感謝您的參與。