Abeona Therapeutics Inc (ABEO) 2024 Q3 法說會逐字稿

Good morning, and welcome to the Abeona Therapeutics third quarter 2024 conference call. (Operator Instructions) Please note, this conference is being recorded.

早安，歡迎參加 Abeona Therapeutics 2024 年第三季電話會議。（操作員說明）請注意，本次會議正在錄製中。

I will now turn the conference over to your host, Greg Gin, VP of Investor Relations. Greg, the floor is yours.

現在我將會議交給東道主投資者關係副總裁格雷格金 (Greg Gin)。格雷格，地板是你的。

Thank you, Jenny. Good morning, everyone, and thank you for joining us on our third-quarter 2024 conference call.

謝謝你，珍妮。大家早安，感謝您參加我們的 2024 年第三季電話會議。

During this call, we'll refer to the press release issued this morning announcing the third quarter results, which is available on our corporate website at www.abeonathapeutics.com. I would like to note that remarks made during today's call may contain projections and forward-looking statements.

在本次電話會議中，我們將參考今天上午發布的第三季業績新聞稿，該新聞稿可在我們的公司網站 www.abeonathapeutics.com 上查看。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。

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.

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 sections in our Form 10-K and periodic reports filed with the SEC. These documents are available on our website at www.abeonathapeutics.com.

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.abeonathapeutics.com 上取得。

On the call today with prepared remarks are Dr. Vish Sesadri, Chief Executive Officer; Dr. Madhav Vasanthavada, Chief Commercial Officer and Head of Business Development; and Joe Vazzano, Chief Financial Officer. Also joining us for the Q&A session will be Dr. Brian Kevany, Chief Technical Officer.

執行長 Vish Sesadri 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；和財務長喬·瓦扎諾。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 Sesadri to lead us off. Vish?

現在，我將把電話轉給 Vish Sesadri 來帶領我們出發。維什？

Thank you, Greg. We appreciate everybody joining this call this morning.

謝謝你，格雷格。我們感謝大家今天早上參加這次電話會議。

We have continued to be productive since our last call and have made progress on multiple fronts toward bringing our lead product candidate, prademagene zamikeracel or pz-cel to patients with recessive dystrophic epidermolysis bullosa or RDEB. These patients continue to seek durable treatment options for their wounds. I'll start with the regulatory progress first.

自上次電話會議以來，我們繼續保持高效，並在多個方面取得了進展，將我們的主要候選產品prademagene zamikeracel 或pz-cel 帶給患有隱性營養不良性大皰性表皮鬆解症或RDEB 的患者。這些患者繼續為其傷口尋求持久的治療方案。我先從監管進展開始。

Since receiving the complete response letter, CRL, in April, we have worked closely with the FDA in preparing the resubmission of our pz-cel biologics license application and getting it back in the review process. As a reminder, the CRL highlighted the need for additional CMC data pertaining to the validation of certain manufacturing and release testing methods.

自從四月收到完整的回信 CRL 以來，我們一直與 FDA 密切合作，準備重新提交我們的 pz-cel 生物製劑許可申請，​​並將其重新納入審查流程。作為提醒，CRL 強調需要與某些製造和發布測試方法的驗證相關的額外 CMC 數據。

The CRL 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. In August, we had a Type A meeting where we aligned with the FDA on the content of the resubmission, including additional information to address all the deficiencies noted in the CRL.

CRL 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。8 月，我們召開了一次 A 類會議，在會議上我們與 FDA 就重新提交的內容達成一致，包括解決 CRL 中指出的所有缺陷的附加資訊。

I'm pleased to report that we completed the resubmission of our BLA for pz-cel at the end of October. Just last week, the FDA accepted the resubmission for review and set a PDUFA date of April 29, 2025. Meeting this regulatory milestone in Q4 as per our guidance was made possible by the tireless efforts and determination of our team, especially technical operations, quality, regulatory and clinical, and I thank them for their dedication.

我很高興地報告，我們已於 10 月底完成了 pz-cel 的 BLA 重新提交。就在上週，FDA 接受了重新提交的審查，並將 PDUFA 日期定為 2025 年 4 月 29 日。根據我們的指導，在第四季度實現這一監管里程碑是由於我們團隊的不懈努力和決心，特別是技術操作、品質、監管和臨床，我感謝他們的奉獻。

The FDA's acceptance of our resubmission indicates that our package includes all the necessary data components, including those items identified in the CRL and observations from the pre-license inspection to support the review of the updated dossier. The FDA has previously informed us that they do not intend to host an advisory committee or AdCom meeting.

FDA 接受我們的重新提交表明我們的資料包包含所有必要的資料組件，包括 CRL 中確定的項目以及許可前檢查的觀察結果，以支持更新檔案的審查。FDA 先前已通知我們，他們不打算主辦諮詢委員會或 AdCom 會議。

In addition, pre-license inspections for all clinical sites and our manufacturing facility were completed earlier this year. Therefore, we do not anticipate additional site inspections as part of the resubmission review process.

此外，所有臨床場所和我們的生產設施的預許可檢查已於今年稍早完成。因此，我們預計不會在重新提交審核過程中進行額外的現場檢查。

We will continue to work with the FDA to facilitate their timely review. We remain confident in our prospects for approval given the unmet medical need in RDEB to treat large chronic wounds, our strong clinical data, our comprehensiveness in addressing every CRL item and PLI observation, and our collaborative interactions with and feedback to date from the FDA.

我們將繼續與 FDA 合作，以促進其及時審查。鑑於RDEB 治療大面積慢性傷口的醫療需求尚未得到滿足、我們強大的臨床數據、我們在處理每個CRL 項目和PLI 觀察方面的全面性，以及我們與FDA 的合作互動和迄今為止的反饋，我們對批准的前景仍然充滿信心。

So now the focus for the rest of this year and into 2025 is obviously to drive through the regulatory process as we continue to ramp up our commercial readiness activities for a potential launch next year, which Madhav will elaborate on in a moment. But before that, I'm excited to share that we have recently entered into a lease agreement for additional building space in Cleveland, Ohio, adjacent to our existing facility to enable capacity expansion beyond our current manufacturing footprint.

因此，今年剩餘時間和 2025 年的重點顯然是推動監管流程，因為我們將繼續加強商業準備活動，為明年可能推出的產品做好準備，Madhav 稍後將對此進行詳細闡述。但在此之前，我很高興地告訴大家，我們最近在俄亥俄州克利夫蘭簽署了一項額外建築空間的租賃協議，該建築空間毗鄰我們現有的工廠，以便能夠將產能擴展到我們當前的製造足跡之外。

I also want to briefly mention the recent issuance of a new US patent for pz-cel. The patent is entitled Gene Therapy for Recessive Dystrophic Epidermolysis Bullosa Using Genetically Corrected Autologous Keratinocytes and includes claims that cover the use of pz-cel for the treatment of RDEB. The patent has an expiration date of Jan 3, 2037, subject to any applicable patent term extension.

我還想簡單提一下最近頒發的 pz-cel 美國新專利。該專利名為“使用基因校正的自體角質形成細胞治療隱性營養不良性大皰性表皮鬆解症的基因療法”，並包括涵蓋使用 pz-cel 治療 RDEB 的權利要求。該專利的有效期限為 2037 年 1 月 3 日，如有任何適用的專利期限延期。

Another US patent will issue on November 19, 2024, relating to the packaging and transport system for pz-cel sheets. Absent any patent term adjustment or extension, that patent would expire in July 2040. The issuance of these patents by the USPTO acknowledges the groundbreaking nature of the technology underlying pz-cel.

另一項美國專利將於 2024 年 11 月 19 日頒發，涉及 pz-cel 片材的包裝和運輸系統。如果沒有任何專利期限調整或延期，該專利將於 2040 年 7 月到期。美國專利商標局頒發這些專利承認了 pz-cel 基礎技術的突破性本質。

If approved, pz-cel will be the first product to use gene-corrected keratinocyte sheets to treat RDEB. Based on pz-cel's clinical trial experience, we are optimistic that the underlying technology for making pz-cel and using it to treat a human patient that is claimed in this patent, which allows stable insertion of this functional collagen VII gene into the genome of each patient's own skin cells could uniquely position pz-cel to achieve wound healing and pain reduction in RDEB wounds following a single administration.

如果獲得批准，pz-cel 將成為第一個使用基因校正角質形成細胞片來治療 RDEB 的產品。基於pz-cel 的臨床試驗經驗，我們對本專利中所聲稱的製造pz-cel 並使用它來治療人類患者的基礎技術感到樂觀，該技術可以將這種功能性VII 型膠原基因穩定插入到人類的基因組中。

I'll now turn the call over to our Chief Commercial Officer, Dr. Madhav Vasanthavada, to highlight our commercial launch preparations. Madhav?

現在我將把電話轉給我們的商務長 Madhav Vasanthavada 博士，重點介紹我們的商業發布準備。馬達夫？

Thanks, Vish, and hello, everyone. We are excited about the progress we are making as we continue to prepare for the potential launch of pz-cel in the US next year. Let me start off with our progress on building the commercialization team and say that we have attracted a strong and passionate group of people with significant launch experience in the cell and gene therapy space and with deep expertise in market access, clinical site onboarding, cell therapy, patient operations, supply chain, patient access, marketing, and medical affairs.

謝謝，維什，大家好。我們對所取得的進展感到興奮，我們將繼續為明年可能在美國推出的 pz-cel 做準備。讓我先介紹一下我們在建立商業化團隊方面的進展，並說我們吸引了一群強大而充滿激情的人員，他們在細胞和基因治療領域擁有豐富的啟動經驗，並且在市場准入、臨床現場入職、細胞治療方面擁有深厚的專業知識、病患營運、供應鏈、病患訪問、行銷和醫療事務。

At launch, we expect to have approximately 20 members on the pz-cel commercialization team, and we feel very confident that this team will deliver a successful pz-cel launch. Before going into other aspects of launch preparations, for those new to the pz-cel story, let me remind you that pz-cel is being developed for the treatment of RDEB, a recessive form of the genetic condition dystrophic epidermolysis bullosa.

在發佈時，我們預計 pz-cel 商業化團隊將有大約 20 名成員，我們非常有信心團隊將成功推出 pz-cel。在進入發射準備的其他方面之前，對於那些剛接觸pz-cel 故事的人，我要提醒您，pz-cel 正在開髮用於治療RDEB，這是一種隱性形式的遺傳性疾病營養不良性大皰性表皮鬆解症。

RDEB is a severe debilitating disease in which nearly a third of the patient's body can be covered in wounds. These wounds can be chronic, they can become chronic, and do not heal by themselves, and are at a high risk for several infections, including cancer called squamous cell carcinoma. Patients rarely live beyond 40 years of age.

RDEB 是一種嚴重的使人衰弱的疾病，患者身體的近三分之一都被傷口覆蓋。這些傷口可以是慢性的，也可以變成慢性的，並且不能自行癒合，並且極有可能發生多種感染，包括稱為鱗狀細胞癌的癌症。患者很少活超過 40 歲。

There are no treatments on the market today that can heal RDEB wounds durably with a single treatment application. Pz-cel, if approved, and we are optimistic of its approval, will be the first-in-class autologous gene therapy to have shown game-changing benefits to RDEB patients and their families. In clinical trials, pz-cel has shown wound healing and pain reduction in large and chronic wounds over multiple years of follow-up after only a single surgical application and with a clean safety profile.

目前市面上沒有任何治療方法可以透過單次治療持久治癒 RDEB 傷口。Pz-cel 如果獲得批准，我們對其批准持樂觀態度，它將成為首個同類自體基因療法，為 RDEB 患者及其家人帶來改變遊戲規則的益處。在臨床試驗中，經過多年的隨訪，pz-cel 僅需一次手術即可實現大面積和慢性傷口的傷口癒合和疼痛減輕，並且具有良好的安全性。

We are, therefore, excited about the impact pz-cel will have on RDEB patients, and we continue to hear positive feedback about pz-cel from physicians, payers, patients, and caregivers we have spoken with. Now in terms of commercialization strategy, our go-to-market approach is simple.

因此，我們對 pz-cel 對 RDEB 患者產生的影響感到興奮，並且我們不斷從與我們交談過的醫生、付款人、患者和護理人員那裡聽到有關 pz-cel 的積極反饋。現在就商業化策略而言，我們的上市方法很簡單。

Upon launch, we intend to have onboarded and trained five EB centers that are very well recognized and are geographically dispersed across the US to create the best possible pz-cel patient experience. Over time, our focus will be to onboard additional qualified treatment centers, QTCs, while driving patient referrals into the QTCs from other EB treatment centers and community settings.

啟動後，我們打算入職並培訓五個 EB 中心，這些中心得到了廣泛認可，並且分佈在美國各地，以創造盡可能最佳的 pz-cel 患者體驗。隨著時間的推移，我們的重點將是建立更多合格的治療中心（QTC），同時推動患者從其他 EB 治療中心和社區機構轉診至 QTC。

Based on our recently completed claims analysis, we estimate there are about 1,300 dystrophic EB patients being treated in the US. Of these patients, we can confirm there are about 750 pz-cel-eligible US RDEB patients, and we have found that nearly 30% of these 750 patients are concentrated at a handful of EB centers and most of these centers we are targeting at launch.

根據我們最近完成的理賠分析，我們估計美國約有 1,300 名營養不良 EB 患者正在接受治療。在這些患者中，我們可以確認大約有750 名符合pz-cel 資格的美國RDEB 患者，並且我們發現這750 名患者中近30% 集中在少數EB 中心，而我們啟動時的目標是這些中心中的大多數。

This gives us confidence that we are working with the right treatment centers and are confident in expecting a high demand at launch. We've also learned from claims data that patients travel from far-off distances into EB centers of excellence or COEs. Roughly 40% of patients currently being treated at the COEs come from out of state.

這讓我們相信我們正在與合適的治療中心合作，並有信心在推出時預計會有很高的需求。我們也從索賠數據中了解到，患者從很遠的地方來到 EB 卓越中心或 COE。目前在 COE 接受治療的患者中約有 40% 來自州外。

Patients travel for specialized care with catchment areas spanning 300 to 400 miles from the COEs and an average referral distance of 250 to 300 miles. These data points continue to strengthen our confidence in pz-cel demand and our commercialization strategy.

患者前往距離 COE 300 至 400 英里的服務區域接受專門護理，平均轉診距離為 250 至 300 英里。這些數據點繼續增強我們對 pz-cel 需求和商業化策略的信心。

With regard to progress on treatment site onboarding, we have conducted multiple interdisciplinary meetings between Abeona teams and the potential treatment sites, including executing formal in-person meetings with members of the senior leadership at the hospitals.

關於治療地點入職的進展，我們在 Abeona 團隊和潛在治療地點之間舉行了多次跨學科會議，包括與醫院高層領導成員舉行正式的面對面會議。

These meetings are often large group meetings with members from different functions of the hospitals, including contracting, medical that includes lead EB physicians and surgeons, cell therapy coordinators, pharmacy directors, and revenue cycle managers. These sessions are helping us to align on an execution path toward site onboarding in connection with a potential pz-cel approval.

這些會議通常是大型小組會議，參與者來自醫院不同職能部門，包括合約、醫療部門，其中包括首席 EB 醫生和外科醫生、細胞治療協調員、藥房總監和收入週期經理。這些會議正在幫助我們調整執行路徑，以實現與潛在的 pz-cel 批准相關的現場啟動。

The commitment of time and resources by these centers is a testimony to the significant value that pz-cel may bring to RDEB patients if approved. In the coming months, we will continue with pre-approval onboarding steps and training to ensure the multidisciplinary teams at each center are educated in all aspects of pz-cel treatment and with processes to begin treating patients pending FDA approval.

這些中心投入的時間和資源證明瞭如果獲得批准，pz-cel 可能會為 RDEB 患者帶來巨大的價值。在接下來的幾個月中，我們將繼續進行預先批准的入職步驟和培訓，以確保每個中心的多學科團隊接受pz-cel 治療各個方面的教育，並了解在FDA 批准之前開始治療患者的流程。

Similar to other cell therapy launches, we would expect sites to be ready to start treating patients in about two to three months post-approval. Now turning to market access. We are focused on ensuring timely and appropriate patient access upon approval.

與其他細胞療法的推出類似，我們預計站點將在批准後大約兩到三個月內準備好開始治療患者。現在轉向市場准入。我們致力於確保患者在獲得批准後及時、適當地就診。

As a reminder, on payer mix, we believe approximately 60% of RDEB patients are covered by commercial plans, about 30% by Medicaid, and the remaining 10% by Medicare. Our market access team continues to engage with the key national and regional commercial payers that represent more than 80% of RDEB commercial lives with a goal to remove barriers to access and ensure appropriate reimbursement for pz-cel.

提醒一下，就付款人結構而言，我們認為大約 60% 的 RDEB 患者由商業計劃承保，約 30% 由醫療補助承保，其餘 10% 由醫療保險承保。我們的市場准入團隊繼續與佔 RDEB 商業生活 80% 以上的主要國家和地區商業付款人合作，目標是消除准入障礙並確保 pz-cel 得到適當的報銷。

In the coming months, we will also engage with the state Medicaid programs to support patient access and broaden reimbursement for pz-cel. Earlier this August, the Centers for Medicare and Medicaid Services, CMS, granted our request for a product-specific procedure code, ICD-10-PCS, which is now in effect for accurate hospital billing and timely reimbursement for pz-cel procedures between healthcare facilities and all types of insurance providers, commercial and government.

在接下來的幾個月中，我們還將參與州醫療補助計劃，以支持患者獲得醫療服務並擴大 pz-cel 的報銷範圍。今年8 月初，醫療保險和醫療補助服務中心(CMS) 批准了我們對產品特定程序代碼ICD-10-PCS 的請求，該代碼現已生效，用於準確的醫院賬單和及時報銷醫療保健之間的pz-cel 程序設施和所有類型的保險提供者、商業和政府。

In addition, Medicare has mapped pz-cel to a favorable MS-DRG 018 assignment, recognizing the breakthrough nature of pz-cel's technology for RDEB patients and supporting reimbursement and patient access for the relatively smaller pool of Medicare RDEB patients. MS-DRG 018 is among the highest available inpatient hospital reimbursement levels for cell and gene therapies, and we are thrilled by CMS' favorable consideration.

此外，Medicare 已將 pz-cel 映射到有利的 MS-DRG 018 分配，認識到 pz-cel 技術對 RDEB 患者的突破性本質，並支持相對較小的 Medicare RDEB 患者的報銷和患者訪問。MS-DRG 018 是細胞和基因療法住院報銷水平最高的項目之一，我們對 CMS 的有利考慮感到非常興奮。

Commercial payers are also recognizing the clinical value of a treatment like pz-cel as they review the durability of our clinical trials following a one-time treatment application. They also recognize patients may need to come back for additional treatments to cover their previously untreated wound areas based in part on our ongoing pz-cel Phase 3b study, where six out of seven patients we have treated so far have received pz-cel at least twice, including one patient that has received pz-cel three times.

商業付款人也認識到 pz-cel 等治療方法的臨床價值，因為他們在一次性治療申請後審查我們的臨床試驗的持久性。他們也意識到，患者可能需要回來接受額外的治療，以涵蓋先前未治療的傷口區域，部分原因是我們正在進行的pz-cel 3b 期研究，迄今為止，我們治療過的7 名患者中，有6 人至少接受了pz-cel兩次，其中一名患者接受了 3 次 pz-cel。

The willingness of returning patients to participate in the pz-cel clinical trials is a testament to the impact on patients from these pz-cel trials. One pediatric patient in the Phase 3b study received 12 pz-cel sheets in a single application, providing substantial coverage spanning 480 centimeters square on their back wounds. These data points are all exciting and give us increasing confidence about the role pz-cel could play in treating RDEB wounds upon its potential approval.

返回患者參與 pz-cel 臨床試驗的意願證明了這些 pz-cel 試驗對患者的影響。3b 期研究中的一名兒科患者一次使用了 12 張 pz-cel 片材，為他們的背部傷口提供了 480 平方厘米的大面積覆蓋。這些數據點都令人興奮，讓我們對 pz-cel 在獲得批准後在治療 RDEB 傷口方面所發揮的作用越來越有信心。

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

現在，我想將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 months ended September 30, 2024, in our most recent Form 10-Q, which is available on our website.

謝謝，馬達夫。我想提醒大家，您可以在我們最新的 10-Q 表格（可在我們的網站上找到）中找到有關我們截至 2024 年 9 月 30 日的三個月財務業績的更多詳細資訊。

Starting with the financial resources on our balance sheet, we had cash, cash equivalents, short-term investments, and restricted cash of $110 million as of September 30, 2024. This compares to $123 million as of June 30, 2024. Based on our current operating plan and assumptions, with our existing cash resources, we estimate we have sufficient financial resources to fund operations into 2026.

從資產負債表上的財務資源開始，截至 2024 年 9 月 30 日，我們擁有現金、現金等價物、短期投資和限制性現金 1.1 億美元。相比之下，截至 2024 年 6 月 30 日，這一數字為 1.23 億美元。根據我們目前的營運計劃和假設，憑藉現有的現金資源，我們估計我們有足夠的財務資源為 2026 年的營運提供資金。

Our cash runway assumption do not account for any potential revenue from commercial sales of pz-cel or proceeds from the sale of a priority review voucher, or PRV, if awarded by the FDA. I'll remind you that pz-cel has been granted rare pediatric disease designation by the FDA.

我們的現金跑道假設沒有考慮 pz-cel 商業銷售的任何潛在收入或優先審查憑證或 PRV（如果 FDA 授予）銷售收益。我要提醒您的是，pz-cel 已被 FDA 授予罕見兒科疾病資格。

So upon its potential approval, we believe that we are eligible to receive a PRV. The last two reported PRV sales were each completed at a value of above $150 million, indicating that PRVs could be increasing in value relative to the long-time steady average of about $100 million.

因此，一旦獲得批准，我們相信我們有資格獲得 PRV。最近兩次報告的 PRV 銷售均以超過 1.5 億美元的價值完成，這表明 PRV 的價值相對於約 1 億美元的長期穩定平​​均價值可能會增加。

Research and development expenses were $8.9 million for the three months ended September 30, 2024, compared to $7.1 million for the three months ended September 30, 2023. Our spend on general and administrative activities was $6.4 million for the three months ended September 30, 2024, compared to $4.2 million for the three months ended September 30, 2023.

截至2024年9月30日止三個月的研發費用為890萬美元，而截至2023年9月30日止三個月的研發費用為710萬美元。截至2024年9月30日的三個月，我們的一般和行政活動支出為640萬美元，而截至2023年9月30日的三個月為420萬美元。

The increase in general and administrative expenses is primarily due to commercial and launch preparation costs. Net loss was $30.3 million for the third quarter of 2024. It is important to note that the net loss in the third quarter of 2024 included a $15.2 million loss resulting from the quarterly remeasurement of the fair value of warrant and derivative liabilities.

一般和管理費用的增加主要是由於商業和發射準備成本。2024 年第三季淨虧損為 3,030 萬美元。值得注意的是，2024年第三季的淨虧損包括因季度重新計量認股權證和衍生負債的公允價值而產生的1,520萬美元損失。

The warrants are required to be classified as a liability and remeasured at a fair market value each reporting period. Net loss in the third quarter of 2023 was $11.8 million, including a $1.1 million loss resulting from the quarterly remeasurement of the fair value of warrant liabilities.

認股權證須歸類為負債，並在每個報告期間以公平市價重新計量。2023年第三季淨虧損為1,180萬美元，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110萬美元虧損。

With that, I will open the call for Q&A. Operator, can you please open the Q&A session?

接下來，我將開始問答環節。接線生，可以打開問答環節嗎？

(Operator Instructions) Dae Gon Ha, Stifel.

（操作員指示）Dae Gon Ha，Stifel。

Congrats on all the progress. It's remarkable. Two questions from me. One is for Vish. As we think about the resubmission and the acceptance, given that the CRL was primarily CMC related, I guess, which aspect at this point since it is more of a review issue, which aspect do you think keeps you up at night? Or do you find to be the most question-bearing going forward?

祝賀所有的進展。這很了不起。我有兩個問題。一張是給維什的。當我們考慮重新提交和接受時，考慮到 CRL 主要與 CMC 相關，我想，在這一點上，哪一方面（因為它更多的是審查問題），您認為哪一方面讓您夜不能寐？或者您認為未來最有疑問？

And second, for Madhav, as we think about the commercial rollout, thanks for the insights on your market research there. How should we think about or how are you guys thinking about the rollout strategy as we think Vyjuvek as well as Filsuvez that's already available.

其次，對於 Madhav，當我們考慮商業推廣時，感謝您對市場研究的見解。我們應該如何考慮或你們如何考慮推出策略，因為我們認為 Vyjuvek 和 Filsuvez 已經可用。

Polypharmacy-wise, what's been your respective comment that you've received from payer discussions? And how are you strategizing around getting patients back into the centers?

就多重用藥而言，您從付款人討論中收到的各自評論是什麼？您如何制定讓患者返回中心的策略？

I'll first address your CMC question, and then I'll have Madhav talk about the Vyjuvek and Filsuvez. So in terms of the -- as we had previously reported, the Type A meeting, we were quite comprehensive in discussing pretty much all outstanding CMC issues noted in the CRL as well as the PLI.

我將首先解決您的 CMC 問題，然後我將讓 Madhav 談論 Vyjuvek 和 Filsuvez。因此，就我們先前報導的 A 類會議而言，我們相當全面地討論了 CRL 和 PLI 中指出的幾乎所有未決的 CMC 問題。

And the only ones we didn't discuss were probably because during the informal meetings leading up to the Type A meeting, we already got those pre-reviewed, if you may, and got a sense of we are in the right direction there.

我們唯一沒有討論的可能是因為在 A 類會議之前的非正式會議上，我們已經對這些內容進行了預先審查（如果可以的話），並且感覺到我們正朝著正確的方向前進。

So in terms of tests or results that the FDA has not yet seen but agreed on, let's say, a protocol for validation, the only outstanding ones were identity testing and Stargardt, right? Those are the two topics. And even there, we had quite deep conversations on exactly what the expectation is.

因此，就 FDA 尚未看到但已達成一致的測試或結果而言，比如說驗證協議，唯一突出的是身份測試和 Stargardt，對吧？這是兩個話題。即使在那裡，我們也就具體的期望進行了深入的對話。

And so for identity testing, we actually had an informal meeting post the Type A meeting and shared a little bit of our preliminary data, and we have the confidence based on that discussion that we have what we need to put that package together.

因此，對於身份測試，我們實際上在 A 類會議後舉行了一次非正式會議，並分享了一些我們的初步數據，基於該討論，我們有信心我們擁有將這個包組合在一起所需的東西。

I think Stargardt is the new data that they're going to see. But in terms of what they would expect, the statistical considerations, and all the types of organisms that we have to demonstrate using our new method.

我認為 Stargardt 是他們將看到的新數據。但就他們的期望、統計考慮以及我們必須使用我們的新方法展示的所有類型的生物體而言。

These are all things that we had a pretty good agreement about, which is why I feel comfortable about how strong our package is. What I'm going to do is Brian Kevany, our Chief Technical Officer, is actually online. So Brian, if you would like to add any more color on those two aspects, please do so.

這些都是我們達成的很好的共識，這就是為什麼我對我們的方案有多強大感到滿意。我要做的是我們的首席技術長 Brian Kevany 實際上在線。所以布萊恩，如果你想在這兩個方面添加更多的色彩，請這樣做。

Yes. Thanks, Vish, and thanks for the question. Yes, I think you covered pretty much everything that we are aligning on internally. I think stargardt was the biggest topic as part of the CRL. So I think as Vish mentioned, that's one area that we're interested in having them look at our additional data, especially considering that Stargardt seems to be like a topic that the agency is paying particularly close attention to.

是的。謝謝維什，也謝謝你的提問。是的，我認為您幾乎涵蓋了我們內部調整的所有內容。我認為 stargardt 是 CRL 中最大的主題。因此，我認為正如 Vish 所提到的，這是我們有興趣讓他們查看我們的附加數據的一個領域，特別是考慮到 Stargardt 似乎是該機構特別密切關注的主題。

But I can't speak more highly of the data that the team has collected and the package that they put together for the resubmission. So I would echo Vish's sentiments around our confidence in the package that we put into the agency.

但我無法對團隊收集的數據以及他們為重新提交而整理的數據包給予更高的評價。因此，我同意維什的觀點，即我們對向該機構提供的一攬子計劃充滿信心。

Thank you, Brian. Dae Gon, I hope that addressed your first question. I'll turn it over to Madhav to talk a little bit about how Vyjuvek Filsuvez's existing treatment options and how our therapies will play there.

謝謝你，布萊恩。Dae Gon，我希望這能解決你的第一個問題。我將把它交給 Madhav，談談 Vyjuvek Filsuvez 現有的治療方案以及我們的療法將如何發揮作用。

Yes. No, thanks, Dae Gon, for the question. Yes. So certainly, there are existing treatment options. And yes, patients are receiving treatment options in their home setting. When we present the market -- based on our market research that we have done, not just with physicians but also with patients and caregivers, many of the respondents of our market research are on existing treatment options.

是的。不，謝謝大坤的提問。是的。當然，現有的治療選擇是存在的。是的，患者正在家中接受治療。當我們介紹市場時—根據我們所做的市場研究，不僅包括醫生，還包括患者和照護人員，我們市場研究的許多受訪者都選擇了現有的治療方案。

And they look at the profile of pz-cel, especially the before and after wound images. And the fact that you're able to show how the wounds look like in a finite six-month period of time has been really moving for them, emotionally moving and they are motivated to consider a treatment like pz-cel just given the extent of wound burden that they have.

他們查看了 pz-cel 的輪廓，特別是傷口前後的圖像。事實上，你能夠在有限的六個月時間內展示傷口的樣子，這對他們來說真的很感動，在情感上令人感動，他們有動力考慮像pz-cel 這樣的治療方法，因為考慮到他們所承受的傷口負擔。

So when a treatment like pz-cel were to get approved and come to be offered to these patients, we are hearing testimonies that they will consider this treatment option given the effects that at least clinical trials have shown.

因此，當像 pz-cel 這樣的治療方法獲得批准並提供給這些患者時，我們聽到的證詞表明，鑑於至少臨床試驗已經顯示的效果，他們會考慮這種治療選擇。

Now as we talk about rollout, because we are targeting high-volume EB centers that are not just EB centers, but also clinical researchers from these centers, the fact that we are seeing this engagement, we know that there are patients, at least in our preliminary onboarding conversations as we are beginning to have those discussions.

現在，當我們談論推出時，因為我們的目標是大容量 EB 中心，這些中心不僅是 EB 中心，而且還包括來自這些中心的臨床研究人員，事實上，我們看到這種參與，我們知道至少在當我們開始進行這些討論時，我們進行了初步的入職對話。

And as patients begin to come to these treatment centers and get their treatments, the referral strategy will kick in as well as other patient to patient and we're raising awareness through patient advocacy groups and multiple other channels. I think the clinical benefit of this product is going to speak there.

隨著患者開始來到這些治療中心並接受治療，轉診策略以及其他患者之間的轉診策略將開始生效，我們正在透過患者倡導團體和多個其他管道提高人們的認識。我認為該產品的臨床益處將在那裡說明。

So that's how we feel with regard to keeping the demand going. With regards to the payers and the conversations, we have had no pushback with regards to the payer engagement so far with about pz-cel vis-a-vis other treatment options.

這就是我們對保持需求的看法。關於付款人和對話，到目前為止，我們在付款人參與 pz-cel 相對於其他治療方案方面沒有遇到任何阻力。

And again, that goes to -- firstly, it's a rare disease part of it, but also goes to the fact that when you can cover large areas of the body with a single surgical application and multiple years of healing, that data again resonates very strongly with them. So we'll certainly keep you all posted. But so far, we feel very confident about the uptake.

再說一遍，首先，這是一種罕見疾病，但也涉及到這樣一個事實：當你可以透過一次手術應用和多年的治癒來覆蓋身體的大面積區域時，這些數據再次產生了非常大的共鳴。強烈地與他們在一起。因此，我們一定會隨時向您通報情況。但到目前為止，我們對採用率非常有信心。

Maurice Raycroft, Jefferies.

莫里斯·雷克羅夫特，杰弗里斯。

Congrats on the progress. I was wondering if the favorable Medicare reimbursement designation, how that impacts conversations with payers related to commercial reimbursement plans.

祝賀取得的進展。我想知道是否有利的醫療保險報銷指定，這如何影響與商業報銷計劃相關的付款人的對話。

And can you talk more about payer conversations and what pricing strategy could look like? Maybe remind me if the first treatment will be a flat price and then subsequent treatments would also be a flat price or variable price per number of sheets? Or how are you thinking about that?

您能否多談談付款人對話以及定價策略可能會是什麼樣子？也許提醒我，如果第一次處理是統一價格，然後後續處理也是統一價格還是每張數的可變價格？還是你怎麼想的？

Yes. Thanks, Maury. I think the Medicare question on the reimbursement. So the reimbursement, the MS-DRG 018 is going to be applied only to the Medicare pool of patients, right? So the actual nature of reimbursement is not going to spill into the commercial side because the commercially insured patients we expect we'll have a single or a case rate arrangement often between the provider side and the payer side.

是的。謝謝，莫里。我認為醫療保險報銷的問題。那麼 MS-DRG 018 報銷將僅適用於 Medicare 患者池，對吧？因此，報銷的實際性質不會滲透到商業方面，因為我們預計商業保險患者通常會在提供者和付款方之間進行單一或個案費率安排。

But the fact that we have a DRG 018 is reflected positively by commercial payers because they recognize that this is a sophisticated technology and not any other burn procedure a graft-related cost. So the value of the technology, the genetic nature of it has been established with this assignment. So that is one.

但我們擁有 DRG 018 的事實得到了商業付款人的積極反映，因為他們認識到這是一項複雜的技術，而不是任何其他燒傷手術的移植相關費用。因此，這項技術的價值及其遺傳本質是透過這項任務而確立的。這就是其中之一。

The ICD-10-PCS code is what is really positive because that procedure code, which is a product-specific procedure code, will make the hospital billing even for commercially insured patients seamless because now they don't have to rely on multiple miscellaneous codes. So I think that ICD-10-PCS code is going to help even with commercially insured patients.

ICD-10-PCS 代碼是真正積極的，因為該程序代碼是特定於產品的程序代碼，即使對於商業保險患者，醫院也可以無縫計費，因為現在他們不必依賴多個雜項代碼。因此，我認為 ICD-10-PCS 代碼即使對於有商業保險的患者也會有所幫助。

With regards to the pricing strategy, our pricing, the way we are thinking about it is a flat price per treatment for the patients. We expect for the NDC that we have, all available pz-cel sheets will be supplied and that will all have flat price per kit. So up to 12 sheets, flat price.

關於定價策略，我們的定價方式是為病患的每次治療提供固定價格。我們預計，對於我們擁有的 NDC，將提供所有可用的 pz-cel 片材，並且每個套件均採用統一價格。因此最多 12 張，統一價格。

If the patient were to come back, let's say, later down the line in their lifetime, that will be a separate its own stand-alone price point, flat price. We are still discussing with payers with regards to any -- there won't be any WAC price changes between the first and the second treatment cycles.

如果病人回來，比方說，在他們有生之年晚些時候回來，那將是一個單獨的獨立價格點，統一價格。我們仍在與付款人討論第一和第二個治療週期之間的 WAC 價格不會發生任何變化。

And we also have not had any pushback with regards to any subsequent treatment cycles from the payers. Does that answer, Maury, or any follow-up?

對於付款人的任何後續治療週期，我們也沒有遇到任何阻力。莫里，這有答案嗎？

Yes, that's really helpful. And maybe one other question. Just wondering if you have a line of sight into how many patients you could treat commercially potentially by the end of 2025, assuming approval and then the two to three months launching after the approval?

是的，這確實很有幫助。也許還有另一個問題。只是想知道，如果獲得批准，然後在批准後兩到三個月內啟動，到 2025 年底，您是否可以商業化治療多少患者？

And then can you talk more about what needs to be done to make the new facility operational that you're leasing and when that could happen as well?

然後您能否更多地談談需要做什麼才能使您租賃的新設施投入運作以及何時可以實現？

Yes. In terms of how many patients we can expect to treat, I think that is something certainly we will continue to monitor and probably guide as we get closer. What our strategy is to have a centralized location to be able to funnel in all of the patients and get them queued up from a payer coverage and insurance benefit standpoint because that's going to be a process that we will start soon after we get approval alongside which we will get the centers of excellence, qualified treatment centers activated and make them patient ready, right?

是的。就我們預計可以治療的患者數量而言，我認為我們肯定會繼續監測，並可能隨著時間的推移提供指導。我們的策略是建立一個集中的地點，能夠匯集所有患者，並從付款人承保範圍和保險福利的角度讓他們排隊，因為這將是我們在獲得批准後不久就會開始的一個過程。卓越中心、合格的治療中心，並讓他們為病人做好準備，對嗎？

So part of it is going to depend on how quickly we can bring the qualified treatment centers and make them patient-ready to treat after we get the approval. So that is one.

因此，部分取決於我們能以多快的速度引進合格的治療中心，並在獲得批准後讓它們做好接受治療的準備。這就是其中之一。

And then the second is, of course, on the manufacturing side, we are going to ramp up that capacity. We have said and we continue to believe that the demand at the time of launch is going to exceed the supply, and that is certainly our focus.

第二個當然是在製造業方面，我們將提高產能。我們已經說過並且仍然相信推出時的需求將超過供應，這當然是我們的重點。

As Vish alluded on the call, we have already started thinking about expanding our manufacturing footprint in the building where we are and how to go about that. So Vish, if you have anything else to add on that?

正如 Vish 在電話會議中提到的那樣，我們已經開始考慮擴大我們所在大樓的製造足跡以及如何實現這一目標。Vish，您還有什麼要補充的嗎？

Yes. Thanks, Madhav, and thanks for that question, Maury. I think it's probably a good idea to remind people that at launch, we're going to have manufacturing capacity. The current manufacturing footprint can take us up to about 10 patient runs or 10 manufacturing runs in a month, but even that will have a certain ramp-up speed, not because of the space per se, but because of hiring and training personnel.

是的。謝謝馬達夫，也謝謝莫里提出的問題。我認為提醒人們在產品發佈時我們將擁有製造能力可能是個好主意。目前的製造足跡可以讓我們在一個月內進行大約 10 次患者運行或 10 次製造運行，但即便如此，也會有一定的加速速度，不是因為空間本身，而是因為僱用和培訓人員。

We're already doing some of that preapproval to train more manufacturing people. However, I think the ramp-up will continue post-approval as well just to balance out where we focus our efforts leading up to the launch, right?

我們已經在進行一些預先批准，以培訓更多的製造人員。然而，我認為批准後也會繼續增加，只是為了平衡我們在發布前的重點工作，對嗎？

So the timing by which we will be operating at that full 10-a-month capacity is a little bit in flux, but we think that by the end of 2025, we should be achieving at least close to that capacity. And the additional space that we talked about, it's a bit early. We have leased the space.

因此，我們以每月 10 個月的滿載運行的時間有點不穩定，但我們認為到 2025 年底，我們應該至少能接近這個產能。我們談到的額外空間還為時過早。我們已租用該空間。

We are engaging with design engineers who have experience in designing for such types of manufacturing GMP spaces for these types of autologous processing suites. And we're trying to see how best to optimize that. I think there are multiple scenarios we are considering right now.

我們正在聘請具有為此類自體加工套件設計此類製造 GMP 空間的經驗的設計工程師。我們正在嘗試了解如何最好地優化它。我認為我們現在正在考慮多種場景。

So as we get closer and closer to the approval time, we'll give you more color as to how that ramp-up will look like, how quickly we can do because there are multiple factors we are optimizing for. One is the speed at which we can add more additional suites.

因此，隨著我們越來越接近批准時間，我們將向您提供更多關於加速的外觀以及我們可以多快完成的信息，因為我們正在優化多個因素。一是我們增加更多套件的速度。

And the other is how can we do that without disturbing existing manufacturing space because you can't compromise on that. I think balancing these two as well as how much space do we really need to bring into additional GMP manufacturing, I think these are all parts of the equation that will get clearer and clearer as we do our homework a little bit.

另一個問題是我們如何在不干擾現有製造空間的情況下做到這一點，因為你不能在這一點上妥協。我認為平衡這兩者以及我們真正需要為額外的 GMP 生產帶來多少空間，我認為這些都是等式的一部分，當我們做一點功課時，它們會變得越來越清晰。

So hopefully, by the next call, Maury, we should have a little bit more clarity on that. I hope at least conceptually, it gives you an idea of how we're looking at capacity because as Madhav mentioned, clearly, the demand is going to be a lot more than our supply at the time of approval.

因此，莫里，希望在下次電話會議時我們能對此有更清晰的了解。我希望至少從概念上講，它能讓您了解我們如何看待產能，因為正如馬達夫所提到的那樣，顯然，在批准時需求將遠遠超過我們的供應。

James Molloy, Alliance Global Partners.

詹姆斯·莫洛伊，聯盟全球合作夥伴。

And actually a little more on the question from before or the answer from before. When you say the 10 patient runs per month, what does that translate into the per year of that 225 patients in the target that you walked through the 30% of the 750 that are eligible should you get approval?

實際上，對先前的問題或之前的答案有更多的了解。當您說每月運行 10 名患者時，如果您獲得批准，您完成的 750 名合格患者中的 30% 的目標中的 225 名患者每年會運行多少？

How should we, on the outside looking and be thinking about when you guys could be able to potentially supply that 225 or potentially even expand that going forward?

從外部來看，我們該如何看待和思考何時能夠提供 225 台，甚至未來可能擴大這一規模？

Yes. Thanks, Jim. So yes, the 10 patients a month, which translates to about 120 a year, our goal is to go beyond that, right? So that's why we are looking at additional space and lease agreements and things like that. How quickly will that come to effect?

是的。謝謝，吉姆。所以，是的，每月 10 名患者，相當於每年約 120 名患者，我們的目標是超越這個數字，對嗎？這就是為什麼我們正在考慮額外的空間和租賃協議以及類似的事情。這要多快才能生效？

I think it's a little premature to speak about it because there are multiple components in that, and there is also regulatory review involved because once the additional space is created and we start to do engineering runs, we have to submit those designs, and there's going to be a need for regulatory review as well.

我認為現在談論它還為時過早，因為其中涉及多個組件，並且還涉及監管審查，因為一旦創建了額外的空間並且我們開始進行工程運行，我們就必須提交這些設計，並且也需要進行監管審查。

I think approximately, the lead time is going to be anywhere between 18 to 24 months. That is just based on analogs we've seen in the past from these types of therapies. So can it be sooner or even later?

我認為，交貨時間大約是 18 至 24 個月。這只是基於我們過去從這些類型的療法中看到的類似情況。那麼可以早一些還是晚一點呢？

I think that is something that we will have more clarity as these designs for new building space get crystallized a little bit and our dialogue with the FDA will ensue that step. So we'll get more clarity. But I think a good ballpark is 1.5 years to 2 years after we've made a clear design print on the paper. So that's a very high-level estimation.

我認為，隨著這些新建築空間的設計變得更加具體化，我們將更加清晰地了解這一點，我們與 FDA 的對話將隨之而來。這樣我們就會更清楚。但我認為一個好的大概是我們在紙上列印出清晰的設計後 1.5 年到 2 年。所以這是一個非常高水準的估計。

Probably the best you can do at this point, of course. Then given the demand versus supply, you guys will be facing and you guys are very good at giving ideas on pricing, you obviously see Vyjuvek's pricing. What do you guys think on pricing power to raise pricing going forward as you're expanding capacity?

當然，這可能是您此時能做的最好的事情。然後考慮到需求與供應，你們將面臨，你們非常擅長給出定價想法，你們顯然看到了 Vyjuvek 的定價。你們對在擴大產能時提高定價的定價能力有何看法？

Do you mean pricing for pz-cel treatment, Jim?

吉姆，你是指 pz-cel 治療的定價嗎？

Yes. Yes, of course.

是的。是的當然。

Yes. I mean I think price for pz-cel treatment, yes, definitely, there is value here that we are bringing to the patients and the community, and we will want to capture that value through pricing. Given the durability, et cetera, we have talked about it as comparable to one-time gene therapy price that pz-cel can secure. And that is really what our going-in assumption is.

是的。我的意思是，我認為 pz-cel 治療的價格，是的，毫無疑問，我們為患者和社區帶來了價值，我們希望透過定價來獲取該價值。考慮到耐久性等因素，我們認為它與 pz-cel 可以確保的一次性基因療法價格相當。這確實是我們的假設。

We haven't guided in terms of the numbers. I think in our last call, we said a floor of $1.5 million per PG treatment. We are continuing to explore the upper bounds of this because our main goal is not to limit patient access, right?

我們沒有以數字為指導。我想在我們上次的電話會議中，我們說過每次 PG 療程的底價為 150 萬美元。我們正在繼續探索其上限，因為我們的主要目標不是限制患者的訪問，對吧？

We want to make sure payers are comfortable, and we are exploring our options in terms of -- especially when you have a product like pz-cel with its efficacy and the outcomes that we have, what options are there on the table.

我們希望確保付款人感到舒適，並且我們正在探索我們的選擇，尤其是當您擁有像 pz-cel 這樣的產品及其功效和我們所擁有的結果時，我們有哪些選擇。

So we are being creative and want to, of course, make sure that we recognize the value that we are bringing and are able to capture it through pricing is certainly one lever. As I mentioned, patients, we do expect them to come back for a repeat treatment for the previously untreated areas, which will be its own billing cycle and its own price there, right?

因此，我們正在發揮創造力，當然希望確保我們認識到我們所帶來的價值，並能夠透過定價來獲取它，這無疑是一個槓桿。正如我所提到的，患者，我們確實希望他們回來對以前未治療的區域進行重複治療，這將是其自己的計費週期和價格，對吧？

So that's how we are thinking about it. And the fact that we are in-house, we are not relying on a third party to produce our vector. It's all done by Abeona. That in and of itself gives us a lot of control autonomy as well as a profitable model to be able to exercise that, right?

這就是我們的想法。事實上，我們是內部的，我們不依賴第三方來生產我們的載體。這一切都是阿貝奧納完成的。這本身給了我們很大的控制自主權以及能夠行使這種自主權的獲利模式，對吧？

So it is helping us from both sides that we have the ability to control the speed and also manage the cost while we think about the manufacturing scale.

因此，在考慮製造規模的同時，我們有能力控制速度並管理成本，這對我們雙方都有幫助。

It certainly seems a pretty straightforward calculation to show the value of the treatment you're helping these patients avoid. Did I hear correctly, $1.5 million a year annual cost of therapy roughly?

這看起來確實是一個非常簡單的計算，可以顯示您幫助這些患者避免的治療的價值。我沒聽錯吧，每年的治療費用大約是 150 萬美元？

It's not a year. It's per treatment, which is a flash price.

這不是一年。這是按每次治療計算的，這是一個閃現價。

(Operator Instructions) Kristen Kluska, Cantor Fitzgerald.

（操作員說明）Kristen Kluska、Cantor Fitzgerald。

Congrats on the resubmission and I appreciate all the transparency you've shared with us over the last few months. It's been very helpful. So as you think about potential conversations with surgeons and doctors if you are approved, I mean, I think the data and the durability really speak for themselves.

恭喜您重新提交，我感謝您在過去幾個月中與我們分享的所有透明度。這非常有幫助。因此，當您考慮如果獲得批准後與外科醫生和醫生進行潛在的對話時，我的意思是，我認為數據和耐用性確實說明了一切。

But can you share with us how you're going to talk to them about the actual surgical procedure, including how the patients in the trial experienced in the past have responded from it? Do you see this as a potential barrier?

但您能否與我們分享您將如何與他們談論實際的手術過程，包括過去參與試驗的患者對此有何反應？您認為這是一個潛在的障礙嗎？

Yes. Thanks for that question, Kristen. We don't see this as a barrier, but I can give you a high-level answer. So the surgeons don't consider this as anything especially new beyond what plastic surgery procedure would entail. We don't see the surgery aspects of applying pz-cel itself as a barrier.

是的。謝謝你提出這個問題，克里斯汀。我們不認為這是一個障礙，但我可以給你一個高層次的答案。因此，外科醫生並不認為這是什麼特別新的事情，超出了整形手術所需的範圍。我們不認為應用 pz-cel 本身的手術方面是一個障礙。

As you know, two of the five centers potentially could be our clinical sites and our clinical sites have had a lot of that experience through our trials. And we've seen through the experience of onboarding the UMass site that was onboarded around the later half of our VITAL study that the surgical aspects were actually pretty smooth in the communications between the doctors that were at Stanford and at UMass.

如您所知，五個中心中的兩個可能是我們的臨床中心，我們的臨床中心透過我們的試驗累積了許多經驗。透過在 VITAL 研究的後半部分登入麻省大學網站的經驗，我們發現史丹佛大學和麻省大學的醫生之間的溝通實際上在手術方面非常順利。

And we see the site onboarding path to be seamless that way because we've been through that an experience once. It's just replicating best practices that we need to achieve. I think from a patient perspective, having these additional sites should only be more helpful because our goal is to minimize their travel time and some of the logistic burden that comes with that.

我們認為網站的登入路徑是無縫的，因為我們已經經歷過一次這樣的經歷。它只是複製我們需要實現的最佳實踐。我認為從患者的角度來看，擁有這些額外的站點只會更有幫助，因為我們的目標是盡量減少他們的旅行時間以及隨之而來的一些後勤負擔。

Madhav, let me ask you to add more color based on our recent interactions with potential sites that we're already working with in terms of the multidisciplinary aspects of how pz-cel application can be cross-pollinated for best practices across these centers.

Madhav，讓我請您根據我們最近與潛在網站的互動添加更多色彩，我們已經就 pz-cel 應用程式如何在這些中心之間交叉授粉以實現最佳實踐的多學科方面進行了合作。

Right. Yes. No, absolutely. Everything that Vish mentioned, that's really the sentiment we are seeing from the other sites also, Kristen, that we are interacting with. Surgically, this is not -- they are so comfortable because this is technologically difficult for them from the actual procedure standpoint.

正確的。是的。不，絕對是。維什提到的一切，這確實是我們從其他網站上看到的情緒，克里斯汀，我們正在與之互動。從手術角度來說，這並不是——他們之所以感到如此舒適，是因為從實際手術的角度來看，這對他們來說在技術上是困難的。

In fact, even dermatological surgeons have applied pz-cel. It's just that we say surgeons or plastic surgeons because they tend to have privileges to the OR. But these surgeons are part of the conversations we are having and they are seeing the technology. And for them, this is not at all a difficult thing.

事實上，甚至皮膚外科醫生也已經應用了pz-cel。只是我們說外科醫生或整形外科醫生是因為他們往往擁有手術室的特權。但這些外科醫生是我們正在進行的對話的一部分，他們正在看到這項技術。而對他們來說，這根本就不是什麼困難的事。

We are also working with the cell therapy coordinators at these centers. I think that's the beauty because these centers have onboarded multiple cell therapies in their institutions. And from that standpoint, there is an infrastructure, there is a process flow.

我們也與這些中心的細胞治療協調員合作。我認為這就是美妙之處，因為這些中心在其機構中採用了多種細胞療法。從這個角度來看，有一個基礎設施，有一個流程。

Yes, of course, there are nuances for pz-cel because pz-cel when approved, will be the first ex vivo autologous nonsystemic therapy, right, PD. I mean, across all of the technologies out there. So because of that, there are certain nuances of, okay, areas to be treated and mapping that out with the patient.

是的，當然，pz-cel 存在細微差別，因為 pz-cel 一旦獲得批准，將成為第一個離體自體非全身療法，對吧，PD。我的意思是，涵蓋所有技術。因此，需要治療的領域存在某些細微差別，並與患者一起制定。

The fact that the clinical trial experience we have gained now with our Phase 3b study is also just helping build that experience curve for us. So no real issues that we are seeing from a procedural standpoint. And as Vish mentioned, yes, the patients, they are coming back for untreated areas that in and of itself is a pretty positive thing that they see the benefit.

事實上，我們現在透過 3b 期研究獲得的臨床試驗經驗也有助於我們建立經驗曲線。因此，從程序的角度來看，我們沒有看到真正的問題。正如維什所提到的，是的，患者們回到未經治療的地區，這本身就是一件非常積極的事情，他們看到了好處。

And then when you resubmitted the BLA and the resubmission was accepted for review, did you get any sense from the agency whether the focus during the time from now to the PDUFA is solely going to be on those red line items that were new essentially from the first time? Or are they going to be looking at everything holistically?

然後，當您重新提交 BLA 並且重新提交的內容被接受審查時，您是否從該機構得到任何信息，即從現在到 PDUFA 期間的重點是否僅集中在那些本質上是新的紅線項目上？ 第一次？或者他們會從整體上看待一切？

Thank you, Kristen. The short answer is, no, we don't have a sense yet. We just had the two-week period is they're going to just look at all the components of data that have been requested and is our application complete in many ways, right? So we do not have any sense to whether everything that has been previously reviewed is going to be opened up again versus focus on the delta, right?

謝謝你，克里斯汀。簡短的回答是，不，我們還沒有任何感覺。我們剛剛有兩週的時間，他們將只查看所要求的數據的所有組成部分，以及我們的申請在很多方面是否完整，對吧？因此，我們不知道之前審查過的所有內容是否會再次開放，而不是專注於三角洲，對嗎？

But our assumption is that a majority of the review will focus on those additional items just based on our conversations leading up to the resubmission and also the nature of what a CRL really is. By process, a CRL is supposed to be a comprehensive list of things that having reviewed the entirety of the package, these are the things that you need to deliver for an approval.

但我們的假設是，大部分審核將集中在這些附加項目上，這些項目僅基於我們重新提交先前的對話以及 CRL 的真正性質。按照流程，CRL 應該是已審核整個套件的內容的綜合列表，這些內容是您需要交付以獲得批准的內容。

So in that spirit, our assumption is that, yes, the focus is going to be on those additional items. But very soon, we're going to have that dialogue pick up because it's just been about that two-week period since we made the resubmission. So we'll definitely, as we've been transparent all these months, we will continue to do that, Kristen.

因此，本著這種精神，我們的假設是，是的，重點將放在這些附加項目上。但很快，我們就會繼續進行對話，因為自從我們重新提交以來剛剛過去了大約兩週的時間。所以我們肯定會繼續這樣做，因為這幾個月我們一直保持透明，克里斯汀。

Well, we appear to have reached the end of our question-and-answer session. So I will now hand back over to Vish for his closing remarks.

好吧，我們的問答環節似乎已經結束了。現在我將請 Vish 發表閉幕詞。

Thank you so much. And before I give you the closing remarks, I just wanted to clarify one of the Q&A questions was about a pricing floor. We have not really disclosed any or even finalized pricing for pz-cel at this point in time. Jim, the question that you had asked was about the $1.5 million was just the floor that we had communicated.

太感謝了。在我做結束語之前，我只想澄清其中一個問答問題是關於定價下限的。目前我們還沒有真正披露 pz-cel 的任何信息，甚至還沒有最終確定定價。吉姆，你問的問題是關於 150 萬美元，這只是我們溝通過的下限。

We do see from a lot of our dialogue so far that there could be an upside. So we're still investigating that. So just wanted to clarify that, so people don't think that we have priced pz-cel at any given price point yet. So with that, I think in closing, we're one step closer to a potential approval for pz-cel, and we believe that we're well positioned for the significant opportunity ahead at Abeona, and we look forward to finishing this year strong and to a potentially transformative 2025 for the company.

從迄今為止的大量對話中，我們確實看到了可能的好處。所以我們仍在調查此事。所以只是想澄清這一點，所以人們不認為我們已經在任何給定的價格點對 pz-cel 進行定價。因此，我認為最後，我們距離 pz-cel 的潛在批准又近了一步，我們相信我們已經為 Abeona 未來的重大機會做好了準備，我們期待今年的強勁表現以及公司潛在變革的 2025 年。

And I know we did not talk much about our pipeline, which we will do in our future calls. We just wanted to focus today's time on the upcoming preparations for pz-cel. So thank you, everyone, for joining us today for the business update, and we'll talk to you again soon.

我知道我們並沒有過多談論我們的管道，我們將在未來的電話會議中進行討論。我們只是想把今天的時間集中在即將到來的 pz-cel 準備工作上。謝謝大家今天加入我們的業務更新，我們很快就會再次與您交談。

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

非常感謝。今天的會議到此結束。此時您可以斷開電話線，度過美好的一天。感謝您的參與。