Abeona Therapeutics Inc (ABEO) 2024 Q4 法說會逐字稿

Greetings. Welcome to Abeona Therapeutics full year 2024 results and business update conference call. (Operator Instructions) Please note this conference is being recorded. I will now turn the conference over to your host, Greg Gin, VP of Investor Relations, and Communications at Abeona. Greg, you may begin.

問候。歡迎參加 Abeona Therapeutics 2024 年全年業績和業務更新電話會議。（操作員指示）請注意，本次會議正在錄音。現在，我將會議交給主持人、Abeona 投資者關係和通訊副總裁 Greg Gin。格雷格，你可以開始了。

Thank you, Paul. Good morning, and thank you for joining us on our full year 2024 results and business update conference call. During this call, we will refer to the press release issued this morning announcing the financial results. It's available on our corporate website at www.abeonatherapeutics.com.

謝謝你，保羅。早安，感謝您參加我們的 2024 年全年業績和業務更新電話會議。在本次電話會議中，我們將參考今天上午發布的宣布財務結果的新聞稿。您可以在我們公司網站 www.abeonatherapeutics.com 上找到它。

Remarks made during today's call may contain projections and forward-looking statements, which 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. Actual results may differ materially from those expressed or implied in the forward-looking statements.

今天電話會議上的言論可能包含根據聯邦證券法的安全港條款所做的預測和前瞻性陳述。這些前瞻性陳述是基於當前預期，可能會發生變化。實際結果可能與前瞻性陳述中表達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 Securities and Exchange Commission. These documents are available on our website at www.abeonatherapeutics.com.

可能導致實際結果不同的因素包括但不限於我們向美國證券交易委員會提交的 10-K 表格和定期報告中「風險因素」部分所列的因素。這些文件可在我們的網站 www.abeonatherapeutics.com 上找到。

On the call today with prepared remarks are Dr. Vish Seshadri, Chief Executive Officer; Dr. Madhav Vasanthavada, Chief Commercial Officer, and Head of Business Development; Dr. Brian Kevany, Chief Technical Officer; and Joe Vazzano, Chief Financial Officer.

今天參加電話會議並發表演講的有首席執行官 Vish Seshadri 博士、首席商務官兼業務發展主管 Madhav Vasanthavada 博士、首席技術官 Brian Kevany 博士和首席財務官 Joe Vazzano。

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

現在，我將把電話交給 Vish Seshadri，讓他帶領我們開始。維什？

Thank you, Greg. We appreciate everybody joining the call this morning. We're less than six weeks away from our PDUFA date of April 29 for prademagene zamikeracel, or pz-cel for receptive dystrophic epidermolysis bullosa or RDEB. Since resubmitting our pz-cel biologics license application in late October and receiving FDA acceptance in early November, we have been working with the FDA on the review process.

謝謝你，格雷格。我們感謝今天早上參加電話會議的各位。距離 4 月 29 日 prademagene zamikeracel 或用於治療接受性營養不良型大皰性表皮鬆解症（RDEB）的 pz-cel 的 PDUFA 日期還有不到六週的時間。自 10 月下旬重新提交 pz-cel 生物製劑許可申請並於 11 月初獲得 FDA 批准以來，我們一直在與 FDA 合作進行審查流程。

As a reminder, the FDA has not indicated the need for additional site inspections or an outcome as part of this review. On March 14, 2025, we received from the FDA a marked-up version of the draft USPI to initiate label discussions. We have also received some post-marketing commitments and requirements from the FDA.

提醒一下，FDA 尚未表示需要進行額外的現場檢查或作為本次審查的一部分得出結果。2025 年 3 月 14 日，我們收到了 FDA 發出的 USPI 草案的標記版本，以啟動標籤討論。我們也收到了FDA的一些上市後承諾和要求。

The potential approval of pz-cel would be a significant moment for the RDEB community a major milestone for our company and would be the main driver of growth and profitability for Abeona for years to come. In anticipation of the PDUFA date, our launch preparations for pz-cel for RDEB are well underway. While five renowned EB treatment centers in the US are undergoing onboarding and activation activities to become pz-cel qualified treatment centers or QTCs.

pz-cel 的潛在批准對於 RDEB 社區來說是一個重要時刻，對於我們公司來說是一個重要的里程碑，並將成為未來幾年 Abeona 成長和盈利的主要驅動力。為了迎接 PDUFA 日期，我們針對 RDEB 的 pz-cel 的發布準備工作正在順利進行中。而美國五家知名的 EB 治療中心正在進行入職和活化活動，以成為 pz-cel 合格治療中心或 QTC。

We anticipate that sites will be activated and ready to biopsy patients starting in the third quarter of this year, approximately three months after the PDUFA date. Similar to past autologous cell therapy launches. In other words, we anticipate launching pz-cel in the third quarter of this year, pending FDA approval.

我們預計，這些站點將從今年第三季（PDUFA 日期後約三個月）開始啟動並準備對患者進行活檢。與過去推出的自體細胞療法類似。換句話說，我們預計今年第三季推出 pz-cel，等待 FDA 批准。

The RDEB community continues to highlight the unmet medical need for new therapies. These patients continue to seek reliable and durable treatment option for their wounds, the strength of our clinical data, manufacturing capabilities, commercial readiness efforts and significant cell and gene therapy launch experience of our commercial team, Abeona is poised to serve the US RDEB community following an approval.

RDEB 社區繼續強調對新療法尚未滿足的醫療需求。這些患者繼續尋求可靠且持久的傷口治療方案，憑藉我們強大的臨床數據、製造能力、商業準備工作以及我們商業團隊豐富的細胞和基因治療推出經驗，Abeona 已準備好在獲得批准後為美國 RDEB 社區提供服務。

We are confident that if approved, pz-cel and its differentiating clinical profile will transform the treatment paradigm for RDEB patients. I'll end my remarks with a brief mention of our partnered program with [Ultragenyx] for Sanfilippo syndrome Type A or MPS IIIA.

我們相信，如果獲得批准，pz-cel 及其差異化臨床特徵將改變 RDEB 患者的治療模式。最後，我想簡單介紹一下我們與 [Ultragenyx] 合作的針對 Sanfilippo 症候群 A 型或 MPS IIIA 的計畫。

In December, Ultragenyx submitted a BLA to the FDA seeking accelerated approval for UX 111 for patients with MPS IIIA. Last month, Ultragenyx announced that the FDA granted the BLA a priority review with a PDUFA action date of August 18, 2025. So 2025 is shaping up to be a very exciting and momentous year in our company's history with the potential for two FDA approvals of our internal and partnered products.

12 月，Ultragenyx 向 FDA 提交了 BLA，尋求加速批准 UX 111 用於治療 MPS IIIA 患者。上個月，Ultragenyx 宣布 FDA 授予 BLA 優先審查，PDUFA 行動日期為 2025 年 8 月 18 日。因此，2025 年將成為我們公司歷史上非常令人興奮且意義重大的一年，我們的內部和合作產品有可能獲得 FDA 的兩項批准。

I'll now hand the call to our Chief Commercial Officer, Madhav Vasanthavada to highlight our commercial launch preparations for pz-cel. Madhav?

現在我將把電話交給我們的商務長 Madhav Vasanthavada，以介紹我們為 pz-cel 進行商業發布的準備工作。馬達夫？

Thanks, Vish, and hello, everyone. With less than six weeks until our PDUFA date and label discussions started with the FDA, we are looking ahead to potential pz-cel approval and the prospect of positively impacting the lives of people living with recessive dystrophic EB.

謝謝，Vish，大家好。距離我們與 FDA 開始 PDUFA 日期和標籤討論還有不到六週的時間，我們期待 pz-cel 獲得潛在批准，並期待著對患有隱性營養不良性 EB 患者的生活產生積極影響。

Before I share the status of our launch readiness, let me briefly recap how we see the commercial opportunity with pz-cel. In the US, based on medical claims data, we estimate there are approximately 1,300 dystrophic EB patients.

在我分享我們的發布準備狀態之前，讓我先簡單回顧一下我們如何看待 pz-cel 的商業機會。在美國，根據醫療索賠數據，我們估計約有 1,300 名營養不良型 EB 患者。

Our research indicates that about 750 of those EB patients are RDEB patients with moderate to severe wounds who are potential candidates for pz-cel. An average RDEB patient has greater than 30% of their body wounded, and we estimate two treatment cycles of pz-cel would be needed to cover the vast majority of their existing wounds.

我們的研究表明，其中約 750 名 EB 患者是患有中度至重度傷口的 RDEB 患者，是 pz-cel 的潛在候選人。一名 RDEB 患者平均全身有超過 30% 的面積受損，我們估計需要兩個週期的 pz-cel 治療才能覆蓋其現有的絕大部分傷口。

In other words, with an average of two treatments per patient and about 750 patients, we foresee about 1,500 treatment opportunities for pz-cel in the US. At this time, we are not accounting for potential future RDEB wounds that patients may develop and other factors which could require additional rounds of pz-cel treatment for patients.

換句話說，平均每位患者接受兩次治療，約有 750 名患者，我們預計美國將有約 1,500 次 pz-cel 治療機會。目前，我們還沒有考慮到患者未來可能出現的 RDEB 傷口以及可能需要患者接受額外幾輪 pz-cel 治療的其他因素。

Even with the conservative floor of $1.5 million per treatment, we believe pz-cel can have a cumulative revenue potential of more than $2 billion in the US alone. Our confidence in realizing this opportunity stems from the continued enthusiasm we hear from patients, leading EB physicians and payers.

即使以每次治療 150 萬美元的保守底價計算，我們相信 pz-cel 僅在美國就能累積創造超過 20 億美元的收入潛力。我們對實現這一機會的信心源於我們從患者、領先的 EB 醫生和付款人那裡聽到的持續熱情。

People with RDEB are desperate for therapeutic options that can reliably close multiple stubborn wounds in one treatment application and provide relief from the perpetual burden of chronic wound care. pz-cel has the potential to be a groundbreaking therapy designed to deliver years of wound healing and pain reduction after a single treatment application even in the tough large chronic rebounds.

患有 RDEB 的人迫切需要一種治療方法，可以在一次治療中可靠地閉合多個頑固傷口，並減輕慢性傷口護理的永久負擔。 pz-cel 有可能成為一種突破性的治療方法，旨在即使在艱難的大型慢性反彈中，一次治療後也能實現多年的傷口癒合和疼痛減輕。

Many of the EB physicians we spoke with at the recent American Association of Dermatologists Annual Conference indicated that they are looking forward to the approval of pz-cell. That said, pz-cel treatment, like other autologous cell and gene therapies is an involved process that requires extensive treatment planning, travel, and care coordination.

在最近的美國皮膚病學會年會上，我們採訪的許多 EB 醫生都表示，他們期待 pz-cell 的批准。也就是說，pz-cel 治療與其他自體細胞和基因療法一樣，是一個複雜的過程，需要大量的治療計劃、旅行和護理協調。

But despite this, patients are excited about the potential approval of pz-cel because of the robust clinical efficacy and durability seen in clinical trials. In a self-reported patient survey conducted two years after pz-cel treatment in a Phase I/IIa study, all seven participants reported that they would undergo pz-cel treatment again and would recommend the procedure to other patients with RDEB.

但儘管如此，患者對於 pz-cel 的潛在獲批仍感到興奮，因為臨床試驗中看到了其強大的臨床療效和持久性。在 I/IIa 期研究中，在接受 pz-cel 治療兩年後進行的一項自我報告患者調查中，所有七名參與者都表示他們將再次接受 pz-cel 治療，並會向其他 RDEB 患者推薦該治療。

In fact, in our ongoing Phase IIIb trial, the majority of our patients are repeat patients, who have elected again to receive pz-cel for their previously nontreated wound areas. One patient even came back for a third round. We want to make pz-cel available and accessible to this patient community as soon as possible following approval.

事實上，在我們正在進行的 IIIb 期試驗中，大多數患者都是複診患者，他們再次選擇接受 pz-cel 治療他們之前未接受過治療的傷口區域。一名患者甚至回來進行第三輪治療。我們希望在獲得批准後儘快讓 pz-cel 可供該患者群體使用。

As such, to talk about our launch readiness and expectations, we are in dialogue with five well-recognized epidermolysis bullosa EB centers to onboard them for pz-cel treatment. These centers are geographically dispersed each with the large catchment areas of patients and collectively can treat a sizable proportion of our estimated 750 pz-cel eligible patients. We expect the process of site activation to vary from site to site but based on our experience with the cell and gene therapy launches, site activation could take approximately three months post FDA approval.

因此，為了討論我們的發布準備和期望，我們正在與五家知名的大皰性表皮鬆解症 EB 中心進行對話，以讓他們接受 pz-cel 治療。這些中心在地理位置上分散，每個中心都擁有大片的患者聚集區，共同可以治療我們估計的 750 名 pz-cel 合格患者中的相當一部分。我們預計站點激活的過程會因站點而異，但根據我們在細胞和基因治療啟動方面的經驗，站點激活可能需要在 FDA 批准後大約三個月的時間。

During these initial months post approval, our teams will work with the centers to discuss eligible patients and secure the necessary payer authorizations. Our goal is to activate our target centers during this launch year that is 2025, so that centers can begin treating patients with pz-cel in third quarter of this year as Vish indicated.

在批准後的最初幾個月內，我們的團隊將與中心合作討論符合條件的患者並獲得必要的付款人授權。我們的目標是在 2025 年這個啟動年啟動我們的目標中心，以便中心可以在今年第三季開始使用 pz-cel 治療患者，正如 Vish 所指出的那樣。

We will announce the names of the pz-cel qualified treatment centers as they become activated. Once a site is activated, they can begin to schedule patients for biopsy based on available manufacturing slots and treat patients approximately 25 days from the time of biopsy collection. Revenue recognition will occur after the patient is treated with the product.

我們將在 pz-cel 合格治療中心啟動後公佈其名稱。一旦站點啟動，他們就可以根據可用的製造時段開始安排患者進行活檢，並在活檢採集後約 25 天內對患者進行治療。患者接受該產品治療後即確認收入。

During the early launch phase, we expect to see a gradual ramp-up at the QTCs with centers treating one patient to two patients first as they get accustomed to the pz-cel treatment process, patient journey, and the reimbursement flow. Then as the sites become more experienced with pz-cel, we expect greater uptake of the product. This gradual ramp-up at the sites this year will be happening in parallel with Abeona ramping up our manufacturing capacity to increase launch supply as we have always indicated we would, a topic that Brian will cover later on this call.

在早期啟動階段，我們預計 QTC 的治療規模將逐步擴大，中心將首先治療一名到兩名患者，以便他們熟悉 pz-cel 治療流程、患者旅程和報銷流程。隨著網站對 pz-cel 的使用經驗越來越豐富，我們預期該產品的採用率會更高。今年，這些工廠的產能逐步提升將與 Abeona 提高製造能力以增加發射供應量同時進行，正如我們一直表示的那樣，Brian 將在本次電話會議的稍後部分討論這個主題。

So from an overall demand standpoint, we are confident of pz-cel's growth. And over time, with capacity ramped up in 2026, we intend to qualify additional treatment centers while also driving patient referrals into the QTCs from the community practices.

因此從整體需求的角度來看，我們對 pz-cel 的成長充滿信心。隨著時間的推移，隨著 2026 年治療能力的提升，我們打算建立更多的治療中心，同時將患者從社區診所轉診到 QTC。

Let me now turn to market access where our two main goals are to ensure favorable access policies for the patients and reimbursement for the treatment centers. We remain confident in meeting both these objectives based on our payer interactions so far.

現在讓我來談談市場准入問題，我們的兩個主要目標是確保患者享有優惠的准入政策和治療中心的報銷。根據迄今為止與付款人的互動，我們仍然有信心實現這兩個目標。

Abeona will give QTCs the flexibility to procure pz-cel either as a buy and bill product, all through a specialty pharmacy and sites like this flexibility to be able to choose. Because access and reimbursement are critical success factors for any high-cost gene therapy, we have been laser-focused on educating the payer community and have had over 40 pre-approval information exchanges communicating our clinical trial results.

Abeona 將為 QTC 提供靈活性，使其能夠以購買和計費產品的方式採購 pz-cel，也可以透過專業藥房和類似網站靈活地進行選擇。由於取得和報銷是任何高成本基因療法成功的關鍵因素，我們一直專注於教育付款人社區，並進行了超過 40 次批准前資訊交流，傳達我們的臨床試驗結果。

To remind you, in terms of the payer mix, our data shows us that 60% to 65% of RDEB lives are covered by commercial plans about 30% to 35% by Medicaid and the remaining less than 10% by Medicare. Payers recognize the persistent unmet need in the RDEB treatment landscape and the tremendous clinical value of pz-cel as they review pz-cel's ability to cover large areas of the body and clinical data showing multiple years of wound healing and pain reduction after a single treatment application.

提醒您，就付款人組合而言，我們的數據顯示，60% 至 65% 的 RDEB 生活由商業計劃覆蓋，約 30% 至 35% 由醫療補助 (Medicaid) 覆蓋，其餘不到 10% 由醫療保險 (Medicare) 覆蓋。付款人認識到 RDEB 治療領域持續存在的未滿足需求以及 pz-cel 的巨大臨床價值，他們審查了 pz-cel 覆蓋大面積身體的能力，以及臨床數據顯示單次治療後多年的傷口癒合和疼痛減輕。

Just last week, we were an emerging biotech sponsor at the PCMA Annual Meeting, one of the industry's most important payer conferences, and we were pleased with the positive reactions. We have received on the clinical and economic value of pz-cel during multiple private in-person engagements with the largest national payers in the country covering the majority of RDEB commercial lives.

就在上週，我們成為 PCMA 年會（該行業最重要的付款人會議之一）的新興生物技術贊助商，我們對積極的反應感到高興。我們在與國內最大的國家付款人進行多次私人面對面交流期間，了解了 pz-cel 的臨床和經濟價值，這些交流涵蓋了大部分 RDEB 商業活動。

At approval, we plan to file for the Medicaid Drug Rebate application or MDRA and apply for a pz-cel-specific J code, so that state Medicaid programs will have an opportunity to carve out pz-cel from the bundled payment methodology with the goal of speeding up access and reimbursement. Finally, I want to mention Abeona assist our integrated support program designed to help eligible patients navigate their entire pz-cel treatment journey, including travel and logistics.

獲得批准後，我們計劃提交醫療補助藥品回扣申請或 MDRA，併申請 pz-cel 特定的 J 代碼，以便州醫療補助計劃有機會從捆綁支付方法中分離出 pz-cel，以加快獲取和報銷速度。最後，我想提一下 Abeona 協助我們的綜合支持計劃，該計劃旨在幫助符合條件的患者完成整個 pz-cel 治療過程，包括旅行和物流。

With that, I would like to hand the call over to Dr. Brian Kevany, our Chief Technical Officer, to discuss commercial manufacturing for pz-cel. Brian?

接下來，我想將電話交給我們的技術長 Brian Kevany 博士，討論 pz-cel 的商業化生產。布賴恩？

Thanks, Madhav. We continue to prepare our curtain good manufacturing practices facility in Cleveland, Ohio for manufacturing commercial-grade pz-cel drug products. As a reminder, our Cleveland site is a completely non-CDMO dependent facility for the production of pz-cel as well as our retroviral vector.

謝謝，馬達夫。我們將繼續在俄亥俄州克利夫蘭市籌備我們的良好生產規範工廠，以生產商業級 pz-cel 藥品。提醒一下，我們的克利夫蘭工廠是一個完全不依賴 CDMO 的設施，用於生產 pz-cel 以及我們的逆轉錄病毒載體。

As Vish mentioned, we anticipate launching pz-cel in the third quarter of this year, pending FDA approval and we are on track from a manufacturing perspective to be prepared to begin receiving biopsy samples from qualified treatment centers and initiating the manufacturing process to support the planned launch.

正如 Vish 所提到的，我們預計將於今年第三季推出 pz-cel，等待 FDA 批准，從製造角度來看，我們正準備開始從合格的治療中心接收活檢樣本，並啟動製造流程以支持計劃中的發布。

Our facility is led by a team of highly skilled production, process and [assay] development and quality control scientists with expertise in cell and gene therapy, particularly in cell culture, analytical testing and wet lab techniques. The maximum capacity at our CGMP facility supports pz-cel manufacturing for up to 10 patients or manufacturing runs per month.

我們的設施由一支高技能的生產、製程和[分析]開發和品質控制科學家團隊領導，他們在細胞和基因治療方面擁有專業知識，特別是在細胞培養、分析測試和濕實驗室技術方面。我們的 CGMP 設施的最大容量支援每月為最多 10 名患者或生產批次進行 pz-cel 製造。

We have previously communicated that pz-cel will be a supply measured launch and there will be a ramp-up period to get to maximum capacity. We currently estimate our initial manufacturing capacity during the early launch phase to be about four treatments per month. And we plan to gradually ramp that monthly capacity to six treatments starting roughly at the end of 2025 to early 2026 towards achieving a maximum capacity of 10 monthly treatments in the first half of 2026. We believe the planned manufacturing capacity ramp-up will sync up well with the ramp for QTCs treating our first patients during the early launch phase this year.

我們之前曾表示，pz-cel 將以供應量投放，並且會有一個上升期以達到最大產能。我們目前估計，早期發布階段的初始生產能力約為每月四次治療。我們計劃從 2025 年底到 2026 年初逐步將每月治療能力提高到 6 次，並在 2026 年上半年實現每月 10 次治療的最大治療能力。我們相信，計畫中的生產能力提升將與今年早期啟動階段治療首批患者的 QTC 的提升同步。

It is important to note that we have a planned annual manufacturing shutdown for maintenance on equipment in the facility, which is standard for cell therapy facilities. This planned shutdown is crucial to maintain operational integrity, regulatory compliance, and product safety, and we schedule it during the December-January holiday time frame to minimize any temporary impact on our production capacity.

值得注意的是，我們計劃每年停產一次，以維護工廠的設備，這是細胞治療設施的標準。此計畫停工對於維護營運完整性、法規遵循和產品安全至關重要，我們將其安排在 12 月至 1 月的假期期間，以盡量減少對我們生產能力的暫時影響。

We have already taken steps to enable capacity expansion beyond our current manufacturing footprint. During the fourth quarter of 2024, we leased additional building space in Cleveland adjacent to our existing facility which will allow us to create additional manufacturing space to increase production capacity for pz-cel. In the coming months, we will be assessing design plans, and we'll share more on our increased manufacturing capacity on a future call.

我們已經採取措施，擴大產能，超越目前的製造規模。2024 年第四季度，我們在克利夫蘭現有工廠附近租賃了額外的建築空間，這將使我們能夠創造額外的製造空間，以增加 pz-cel 的生產能力。在接下來的幾個月裡，我們將評估設計計劃，並將在未來的電話會議上分享更多有關我們提高的製造能力的資訊。

Our long-term plan is to build manufacturing capacity to support 200-plus annual pz-cel treatment by the second half of 2027. I'd like to hand the call over to our Chief Financial Officer, Joe Vazzano, to discuss our financial results.

我們的長期計劃是到 2027 年下半年建立生產能力，以支持每年 200 多次 pz-cel 治療。我想將電話交給我們的財務長喬·瓦扎諾 (Joe Vazzano)，討論我們的財務表現。

Thank you, Brian. I would like to remind everyone that you can find additional details on our financial results for the full year ended December 31, 2024, in our most recent Form 10-K, 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 $98.1 million as of December 31, 2024. This compares to $52.6 million as of December 31, 2023.

謝謝你，布萊恩。我想提醒大家，您可以在我們網站上最新的 10-K 表格中找到有關截至 2024 年 12 月 31 日的全年財務業績的更多詳細資訊。從我們資產負債表上的財務資源開始，截至 2024 年 12 月 31 日，我們擁有現金、現金等價物、短期投資和受限現金 9,810 萬美元。相比之下，截至 2023 年 12 月 31 日，這一數字為 5,260 萬美元。

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. Our cash runway assumptions 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. So upon its potential approval, we believe that we are eligible to receive a PRV.

根據我們目前的營運計劃和現有現金資源的假設，我們估計我們有足夠的財務資源來資助 2026 年的營運。我們的現金流量假設並未考慮 pz-cel 商業銷售的任何潛在收入或 FDA 授予的優先審查券 (PRV) 的銷售收益。我要提醒你，pz-cel 已被 FDA 授予罕見兒科疾病治療藥物稱號。因此，一旦獲得批准，我們相信我們有資格獲得 PRV。

Research and development expenses were $34.4 million for the full year ended December 31, 2024, and compared to $31.1 million for the full year ended December 31, 2023. Our spend on general and administrative activities was $29.9 million for the full year ended December 31, 2024, compared to $19 million for the full year ended December 31, 2023.

截至 2024 年 12 月 31 日全年的研發費用為 3,440 萬美元，而截至 2023 年 12 月 31 日全年的研發費用為 3,110 萬美元。截至 2024 年 12 月 31 日的全年，我們在一般及行政活動上的支出為 2,990 萬美元，而截至 2023 年 12 月 31 日的全年支出為 1,900 萬美元。

The increase in general and administrative expenses is primarily due to commercial launch preparation costs, which included the hiring of a well-experienced commercial team to support the potential launch. Net loss was $63.7 million for the full year ended December 31, 2024, or $1.55 loss per common share as compared to $54.2 million or $2.53 loss per common share for the full year of 2023.

一般及行政開支的增加主要是由於商業發射準備成本，其中包括聘請經驗豐富的商業團隊來支援潛在的發射。截至 2024 年 12 月 31 日的全年淨虧損為 6,370 萬美元，即每股普通股虧損 1.55 美元，而 2023 年全年淨虧損為 5,420 萬美元，即每股普通股虧損 2.53 美元。

Lastly, we have received a question from investors about when we will start recognizing revenue after pz-cel's commercially launched. As we stated earlier on this call, we are planning for a commercial launch in the third quarter of this year if pz-cel is approved. Revenue recognition occurs upon pz-cel administration. That is the surgical procedure. So that would give you a sense of the time it would take from a patient being biopsied through the 25-day pz-cel manufacturing process to when we recognize revenues.

最後，我們收到了投資者的詢問，關於 pz-cel 商業化推出後我們何時開始確認收入。正如我們之前在電話會議上所說，如果 pz-cel 獲得批准，我們計劃在今年第三季進行商業發布。收入確認發生在 pz-cel 管理時。這就是手術過程。這樣您就可以了解從對患者進行活檢到 25 天的 pz-cel 製造過程再到我們確認收入所需的時間。

And with that, I will open the call for Q&A. Operator, can you please open the Q&A session of this call.

現在，我將開始問答環節。接線生，您能否開啟本次通話的問答環節。

(Operator Instructions)

（操作員指示）

Kristen Kluska, Cantor.

克里斯汀·克魯斯卡（Kristen Kluska），領唱。

Hi, good morning, everyone. Thanks for taking my question and just want to say, very much appreciate how transparent you've been with us during this review process, including the last year. So considering where are in discussions right now with the agency, do you feel that the FDA is satisfied with the work you've done on the CMC side that resulted in the CRL last year. Have you fielded more questions specific on that? Do you have a sense of any work that you'll need to do on that side?

大家好，早安。感謝您回答我的問題，我只想說，非常感謝您在這次審查過程中，包括去年，對我們的坦誠相待。那麼，考慮到目前與該機構的討論情況，您是否認為 FDA 對您在 CMC 方面所做的工作以及去年獲得的 CRL 感到滿意。您是否回答過更多有關該問題的具體問題？您是否知道在那方面需要做哪些工作？

Good morning, Kristen. Thank you for the question. I would say this, we have addressed all the asks of the FDA from our side that came out from the CRL. We did not negotiate any of those points. I mean those 12 items that we had articulated last year and so we took the approach of comprehensively addressing each and every one of those, and we believe we've done that.

早安，克里斯汀。謝謝你的提問。我想說的是，我們已經解決了 FDA 在 CRL 中提出的所有要求。我們沒有就上述任何一點進行談判。我指的是我們去年闡明的 12 個項目，因此我們採取了全面解決其中的每一個項目的方法，我們相信我們已經做到了這一點。

And the reason we believe we've done that is because how many touch points we've had like five or six informal meetings with them in the time between the CRL and the resubmission and also a formal Type A meeting where we got a lot of our questions answered. So we feel like we have addressed all of those.

我們認為我們之所以這樣做，是因為在 CRL 和重新提交之間的這段時間裡，我們與他們進行了五六次非正式會議，還進行了一次正式的 A 類會議，在會上我們得到了很多問題的答案。因此我們覺得我們已經解決了所有這些問題。

And then, of course, through the current review process after the resubmission, the types of questions we -- IR that we have received and how we addressed them even the amount of time that has gone past our resubmission, we just put two and two together. And if you look at the holistic picture, we feel like we're in a good place, of course. Technically, we cannot say the FDA said you've satisfied all these items because the review is still ongoing. But from our end, we have really checked those boxes. I hope that addresses your question.

然後，當然，透過重新提交後的當前審查流程，我們收到的問題類型 - IR以及我們如何解決這些問題，甚至重新提交之後所花的時間，我們都將兩者結合起來。如果你從整體上看，我們當然會覺得我們處於一個很好的位置。從技術上講，我們不能說 FDA 說你已經滿足了所有這些項目，因為審查仍在進行中。但從我們的角度來看，我們確實檢查了這些箱子。我希望這能解答你的問題。

Yes, it does. Thank you. And then of the five centers that you referenced, have you had conversations with them specifically just to get an early sense of maybe the percent of their patients that may be eligible for this therapy over time.

是的。謝謝。然後，在您提到的五個中心中，您是否與他們進行過專門的交談，只是為了儘早了解他們的患者中可能有多少比例的人可能隨著時間的推移有資格接受這種治療。

Great. And for that question, I'll actually turn over to Madhav to address it as the person that is directly in dialogue here.

偉大的。對於這個問題，我實際上將交給馬達夫來回答，因為他是這裡直接對話的人。

Yes, Kristen, thanks for that question. These centers that we are talking to, they are large, as we have discussed in the past, large economic institutions with patients. We will get more closer soon after approval in terms of how many actual number of patients, but we continue to hear from them that there are patients who they see pz-cel as a really good option for the moderate severe RDEB patients primarily complementary to the existing treatment options. So from that standpoint, they have patients that they are thinking about. But as soon as we have approval, we'll begin to start clearing all of the access related topics that we discussed.

是的，克里斯汀，謝謝你的提問。我們正在討論的這些中心規模很大，正如我們過去討論過的，它們都是擁有患者的大型經濟機構。在獲得批准後，我們很快就會得知實際患者數量，但我們不斷聽到他們說，有些患者認為 pz-cel 是中度重度 RDEB 患者的一個非常好的選擇，主要可以補充現有的治療方案。因此從這個角度來看，他們正在考慮病人。但一旦我們獲得批准，我們將開始清理我們討論過的所有與訪問相關的主題。

In terms of the numbers of patients, about 30% of the 750 patients are primarily based on the claims analysis are in seven centers of excellence, five of these centers, we are already in discussions with. So we are looking at least a triple-digit number of patients that are currently housed within these institutions. And a pretty sizable number of those triple-digit number of patients would be at least at the early phase of launch candidates and then they will come on the product over time.

就患者數量而言，根據索賠分析，750 名患者中約有 30% 來自七個卓越中心，我們已經與其中五個中心進行了討論。因此，我們正在尋找目前居住在這些機構中的至少三位數的患者。在這些三位數的患者中，有相當一部分至少處於候選藥物的早期階段，然後他們會隨著時間的推移開始使用該產品。

Okay. Thanks. And last question for me. I've seen a lot of new investor faces on the story over the last few weeks and months in particular. So can you just remind us what's really going to be the top reason for patients wanting to seek this therapy?

好的。謝謝。這是我的最後一個問題。特別是過去幾週和幾個月以來，我在這個故事中看到了許多新投資者的面孔。那麼，您能否提醒我們患者想要尋求這種療法的首要原因是什麼？

Is it just the wound profile itself and having to deal with the advantages is it in particular because these wounds make patients more susceptible to things like squamous cell carcinoma and infections, which are leading causes of mortality here? Just what is like really drilling down the key reason? Thank you so much, again.

是否只是傷口輪廓本身以及必須處理的優勢，特別是因為這些傷口使患者更容易受到鱗狀細胞癌和感染等疾病的影響，而這些疾病是導致死亡的主要原因？真正深入探究關鍵原因是什麼呢？再次感謝您。

I think the key reason from what we are hearing is the need to have a durable or a longer-term closure of the wounds. Really, these patients have multiple wounds on their bodies and if there is a treatment option that can minimize chances of infections, minimize reduction in the bandage and the wound dressing changes.

我認為，我們所聽到的主要原因是需要持久或長期地治癒傷口。事實上，這些患者身上有多處傷口，如果有一種治療方案可以最大限度地減少感染的機會，最大限度地減少繃帶和傷口敷料的更換。

These are really the quality of life impact that they would have squamous cell carcinoma is certainly a significant factor that is riding not just for patients and caregivers, also from physicians' standpoint. So -- and we know that there is a high relation between squamous cell carcinoma [crane] as the wounds get more and more chronic. So they're always watching out for that.

這些確實會對生活品質產生影響，鱗狀細胞癌無疑是一個重要因素，不僅對病人和照護者如此，對醫生而言也是如此。所以—我們知道，隨著傷口變得越來越慢性，鱗狀細胞癌與 [crane] 之間存在著密切的關係。所以他們總是在關注這一點。

And therefore, especially for chronic wounds, if you're able to treat and heal these wounds then that's a significant impact for them. So that's really the primary driver. And over time, I think that we will learn many more quality-of-life impact as patients get treated.

因此，特別是對於慢性傷口，如果你能夠治療和治癒這些傷口，那麼這對它們來說將是一個重大的影響。所以這確實是主要驅動因素。隨著時間的推移，我認為我們將了解到患者接受治療對生活品質的更多影響。

Thanks again, and wishing you all the best in the next six weeks.

再次感謝，並祝福您在接下來的六週內一切順利。

Thank you, Krist.

謝謝你，克里斯特。

Maury Raycroft, Jefferies.

莫里‧雷克羅夫特（Maury Raycroft），傑富瑞集團（Jefferies）。

Hi. This is Ami on for Maury. Two from us. First, on the label draft that you mentioned, is the draft that you received from the FDA in line with your vision for pz-cel? Or do you anticipate further modifications and discussions with the FDA?

你好。這是 Ami 為 Maury 表演的。我們有兩個。首先，關於您提到的標籤草案，您從 FDA 收到的草案是否符合您對 pz-cel 的願景？或者您預計會與 FDA 進行進一步的修改和討論？

And the second question is probably for Madhav, on upon the approval, do you expect a patient backlog until you reach maximum manufacturing capacity or do you think patient interest and manufacturing capacity will increase gradually without creating that backlog.

第二個問題可能是針對 Madhav 的，在獲得批准後，您是否預計在達到最大生產能力之前會出現患者積壓，或者您是否認為患者興趣和生產能力會逐漸增加，而不會產生積壓。

Thank you for the question. I'll take the first one and then pass it on to Madhav. So your question was whether the drag label is aligned or consistent with our expectations. The short answer is yes. As you can imagine, there are a lot of different details where it will get refined over back and forth between the FDA and the sponsor. So that's very routine process. But in terms of the big ticket items of how we look at the label, there are no big surprises here. Madhav?

謝謝你的提問。我會拿第一個，然後把它傳給 Madhav。所以你的問題是阻力標籤是否與我們的預期一致。簡短的回答是肯定的。正如您所想像的，FDA 和申辦者之間需要反覆討論和完善許多不同的細節。這是一個非常常規的過程。但就我們如何看待標籤上的大件商品而言，這裡沒有什麼大的意外。馬達夫？

Yes. And I would add with regards to the patient backlog, we do anticipate that happening and I know we had mentioned that early on, and we continue to feel that this particular launch early on, for sure, is supply gated for the several months coming up at the very least as we are ramping up to 10 patients a month. To Kristen's earlier question as well, we have received interest, right, from these physicians who are who are identifying patients that we will work with them to clear the -- to begin to have the patients get on manufacturing slots.

是的。我想補充一點，關於病人積壓的問題，我們確實預料到了這種情況會發生，我知道我們早就提到過這一點，我們仍然認為，早期的這次特別推出肯定會在接下來的幾個月裡受到供應限制，因為我們每月要接待 10 名病人。對於克里斯汀先前提出的問題，我們也收到了這些醫生的關注，他們正在確定病人，我們將與他們合作，清理病人，開始讓病人進入製造時段。

So early on at the time of launch for the several months as we release the manufacturing slots, we want to make sure that these slots are occupied for the foreseeable months. And we will continue to keep you updated in terms of how that is shaping up. But yes, we are very much expecting there will be a queue of patients.

因此，在發布後的幾個月內，當我們發布製造時段時，我們希望確保這些時段在可預見的幾個月內被佔用。我們將繼續向大家通報事情的進展。但是的，我們非常期待會有一大群病人排隊。

Thank you very much.

非常感謝。

Francois Brisebois, Oppenheimer.

弗朗索瓦·布里斯博瓦，奧本海默。

Hi. Thanks for taking the question. And thanks for the updates on the developments here. I was just wondering, you mentioned your conferences recently, can you help us understand maybe the physician education that's necessary?

你好。感謝您回答這個問題。感謝您通報這裡的進度。我只是想知道，您最近提到了您的會議，您能否幫助我們了解必要的醫生教育？

The trials have been going on for a while. So I'm just wondering if this is a situation where the doctors are just waiting for it? Or is there still based on the fact that it's not approved yet, is there still a lot of physician education and training that's necessary at launch? Then I'll have a follow-up. Thank you.

審判已經進行了一段時間。所以我只是想知道這是否是醫生在等待的情況？或者基於尚未獲得批准的事實，在推出時是否仍需要大量的醫生教育和培訓？然後我會跟進。謝謝。

So physician is -- yes, physician education is certainly needed. I mean in terms of the focus areas for us, we are really discussing in greater detail with the qualified or the centers that we are looking to onboard and qualify. But for the rest of the physicians out in the community, as we understand, there are about 23 centers of excellence that have a coordinated practice for EB clinics. And we are really targeting at the time of launch, a subset of those.

所以醫生是——是的，醫生教育肯定是需要的。我的意思是，就我們的重點領域而言，我們正在與合格的機構或我們希望加入和獲得資格的中心進行更詳細的討論。但對於社區中其他的醫生來說，據我們了解，大約有 23 個卓越中心為 EB 診所提供協調實踐。我們在發佈時真正瞄準的是其中的一部分。

So for the rest of the centers as well as for the community positions, we never -- we have not gone out and discussed really pz-cel with them because we are still waiting for the approval to happen. So as we begin to raise our promotional measures and efforts as well as engage with other patients who have gone through clinical trials in the past and share their experiences that is definitely going to get more of the word and attention out there. So did that answer your question, Frank or in terms of the training aspect and awareness?

因此，對於其餘中心以及社區職位，我們從未 - 我們沒有出去與他們討論真正的 pz-cel，因為我們仍在等待批准。因此，當我們開始加強我們的宣傳措施和力度，並與過去參加過臨床試驗的其他患者交流並分享他們的經驗時，這肯定會得到更多的宣傳和關注。那麼，弗蘭克，這回答了你的問題嗎，或者就培訓方面和意識而言？

Yes. No, no, absolutely. And I think the competitor having a product out there is probably helped as well for awareness. But I guess so on the physician side and then on the patient angle, is this an RDEB community, this is so severe that I'm just wondering how tightly knit -- is the patient community? And I'm not talking a full-blown DTC, but is there an angle on the digital side? Is there an online community that makes these patients very aware quickly of the opportunity? Or how do you market to that? Thank you.

是的。不，不，絕對不是。我認為競爭對手推出的產品也可能有助於提高知名度。但我想從醫生的角度和病人的角度來說，這是一個 RDEB 社區嗎？情況非常嚴重，我只是想知道患者社群的關聯有多緊密？我並不是說全面的 DTC，而是在數位方面是否存在一個角度？是否有一個線上社群可以讓這些患者快速意識到這個機會？或者您如何進行行銷？謝謝。

Absolutely. We do have -- it's a very tight as you said, very tightened patient community. We have advocacy groups that have been great partners with us even through the approval process. So we will have a digital presence. We are in discussions with these patients who will be able to share sort of their stories as well as -- of course, our website presence and social media, we will be there to raise this awareness.

絕對地。我們確實有——正如您所說，這是一個非常緊密的患者社區。我們有一些倡導團體，他們甚至在整個審批過程中都是我們很好的合作夥伴。因此我們將擁有數位化存在。我們正在與這些患者進行討論，他們將能夠分享他們的故事——當然，我們的網站和社交媒體，我們將在那裡提高這種意識。

And as you mentioned, there is a lot of coming in second to market in this particular space as a gene therapy has its own advantages, not only in terms of some of the early patients and patient identification, but also from other aspects of payers and access clearances.

正如您所提到的，在這個特定領域，有很多公司進入第二市場，因為基因療法有其自身的優勢，不僅在一些早期患者和患者識別方面，而且在付款人和准入許可等其他方面。

So in terms of our digital presence, we will be there to do all of the right things there, but also in terms of physical presence and field personnel we'll have members of our team who will be out in the community to raise their awareness and begin to start to help with the referral of these patients into the qualified treatment centers.

因此，就我們的數位存在而言，我們將在那裡做所有正確的事情，但就實際存在和現場人員而言，我們也會派團隊成員到社區去提高他們的認識，並開始幫助將這些患者轉診到合格的治療中心。

So that's really going to be our two-pronged approach. But given our supply capacity, we are also trying to titrate as to how much of rigor we want to put on these different measures because we do know that the unmet need exists. And from what we are hearing from physicians, it's really a complementary aspect to existing treatment, so it's really going to be over a period of time, how we get our marketing resources.

所以這實際上將是我們的雙管齊下的方法。但考慮到我們的供應能力，我們也在嘗試確定對這些不同措施要採取多大的嚴格措施，因為我們確實知道存在未滿足的需求。從我們從醫生那裡聽到的情況來看，這實際上是對現有治療的一個補充，因此，在一段時間內，我們將真正了解如何獲得行銷資源。

Thank you.

謝謝。

Ram Selvaraju, H.C. Wainwright.

拉姆‧塞爾瓦拉朱 (Ram Selvaraju)，H.C.溫賴特。

Hi. Thanks for taking my question. Firstly, on the PRV, I was just wondering if you could enumerate any potential factors that might preclude the PRV from being granted or if at this juncture, you fully expect it to be awarded? And also if you could comment on the recent environment for PRV resales.

你好。感謝您回答我的問題。首先，關於 PRV，我只是想知道您是否可以列出任何可能阻止授予 PRV 的潛在因素，或者在這個時刻，您是否完全預期它會被授予？另外，您是否可以評論一下 PRV 轉售的近期環境？

Good morning, Ram. Thank you for the question. We do not anticipate any potential factors that may preclude a PRV for us. In general, the big requirement items for PRV eligibility or you should have a rare pediatric designation, which we do.

早安，拉姆。謝謝你的提問。我們預計不會有任何潛在因素阻礙我們獲得 PRV。一般來說，PRV 資格的主要要求項目是您應該具有罕見的兒科資格，我們就是這樣的。

The second is we should have a priority review in the current review process, which we do. And finally, this has to be a new drug. It's prademagene zamikeracel, the first-time launch, right? So if you look at past examples, it's the same drug is rebranded, but it's the same vector or whatever process you had, then there was an example where PRV was not granted. We don't believe that applies to us because it's a first time launch for us.

第二，我們應該在目前的審查過程中優先審查，而我們確實這樣做了。最後，這必須是一種新藥。這是 prademagene zamikeracel，首次推出，對嗎？因此，如果你看看過去的例子，你會發現雖然是同一種藥物被重新命名，但載體或任何過程都是相同的，那麼就有 PRV 未被授予的例子。我們認為這並不適用於我們，因為這是我們第一次推出。

So those were the three big topics on checking off. Are we really gearing up for PRV here. But to flip back that question, if there are anything else that we have not discussed as eligibility factors that you've heard, we'd definitely like to hear, but we have not -- we don't have any such things right now.

這就是需要核對的三個重要主題。我們真的準備好迎接 PRV 了嗎？但回到這個問題，如果還有其他我們尚未討論的資格因素，而您又聽說過，我們當然希望聽到，但我們還沒有——我們現在沒有任何這樣的事情。

And your second part of the question was the recent trend in PRV sale, right? We have seen that after the program is -- has been put on hold or we don't know the future of the PRV program where it's headed, and that uncertainty may have created a different pricing trend for the last four PRVs, I think, which are sold for north of $150 million.

您問題的第二部分是 PRV 銷售的近期趨勢，對嗎？我們已經看到，在該計劃被擱置之後，或者我們不知道 PRV 計劃的未來走向，這種不確定性可能導致最後四艘 PRV 的定價趨勢不同，我認為，這四艘 PRV 的售價超過 1.5 億美元。

Our goal is to optimize the pricing. We're not really in a rush that the PRV has to sell. The day we get granted if we get granted, we have the time now since we have our cash runway to optimize for pricing over speed. So that's where I would leave it. But if you have any follow-up questions on this topic, happy to address it.

我們的目標是優化定價。我們其實並不急於出售 PRV。如果我們獲得批准，那麼當我們獲得批准的那一天，我們現在就有時間，因為我們有現金跑道來優化定價而不是速度。所以我就把它留在那裡了。但如果您對此主題有任何後續問題，我們很樂意解答。

And also just to add into that, one of those four PRVs was sold within a week of actually getting approval at the full $150 million price tag.

另外，補充一點，這四艘 PRV 中的一艘在獲得批准後一周內就以 1.5 億美元的價格售出。

Okay. Very helpful. And then the only other follow-up was with respect to potential positioning and deployment of pz-cel in markets outside of the US. In particular, I wanted to see if you had any other comments regarding logistical potential deployment of this for treatment of European RDEB patients and what other high-priority markets you potentially see as being viable for pz-cel ex US. Thanks.

好的。非常有幫助。然後唯一的後續行動是關於 pz-cel 在美國以外市場的潛在定位和部署。特別是，我想看看您是否對將此技術應用於治療歐洲 RDEB 患者的後勤潛在部署還有其他評論，以及您認為除美國以外還有哪些其他高優先級市場可能適合 pz-cel。謝謝。

Sure. I will start with Madhav. Feel free to add your thoughts. We do have a lot of interest from Europe and also some Asian markets for pz-cel, just from a bandwidth perspective, we haven't been able to entertain those discussions in much depth. We have those inquiries in a warm relationship right now because we wanted to get through the pz-cel approval in the US first, and then we will be able to have more meaningful dialogues.

當然。我先從 Madhav 開始。請隨意添加您的想法。歐洲和一些亞洲市場確實對 pz-cel 很感興趣，但僅從頻寬角度來看，我們還無法深入討論這些主題。我們目前正與這些諮詢保持著密切的關係，因為我們希望首先獲得美國的 pz-cel 批准，然後我們才能進行更有意義的對話。

But the interest, the markets, they exist as we understand, we need to do a little bit more homework on the timing of when we will be able to supply there. And also whether there are creative -- I mean, our default assumption is that we will need to set up manufacturing shop in a European location, but we're also now more recently hearing there are ways depending on the shelf life of a product, even for a transplant like product that has very limited shelf life. There are ways in which you could supply from a US location to European countries, but we will explore that optionality after launch.

但正如我們所知，利益和市場是存在的，我們需要做更多的功課來確定何時能夠在那裡供應。還有是否有創意——我的意思是，我們的預設假設是我們需要在歐洲某個地方設立製造工廠，但我們最近也聽說有辦法取決於產品的保質期，即使對於保質期非常有限的移植產品也是如此。您可以透過多種方式從美國某個地方向歐洲國家供貨，但我們將在推出後探索這種可選性。

Madhav, sorry, please do add any other --

Madhav，抱歉，請添加任何其他--

Yes, you've addressed most. And I would just say, even within Europe, right, Ram, we'll look to see what's really the market where the reimbursement landscape also in the market access looks positive. We know that, for instance, Austria and Germany, maybe are a couple of markets where there may be a greater appetite and we understand that, especially for these kinds of surgeries Austria is a big center where patients already travel to the EB center.

是的，您已經解決了大部分問題。我想說的是，即使在歐洲，對吧，拉姆，我們也會關注真正的市場，看看報銷前景以及市場准入是否看起來是積極的。我們知道，例如，奧地利和德國可能是兩個需求更大的市場，而且我們知道，特別是對於這類手術，奧地利是一個很大的中心，患者已經前往 EB 中心。

So from a logistics standpoint, it's not like we have to go into multiple markets, but rather already take advantage of the infrastructure and the patient pathway that exists in these kinds of markets. And if we are able to just deploy in one or maybe two centers, over time, that can really be a huge benefit from a cost and speed to market kind of standpoint.

因此，從物流的角度來看，我們並不需要進入多個市場，而是可以利用這些市場中現有的基礎設施和病患通道。如果我們能夠在一個或兩個中心進行部署，那麼隨著時間的推移，從成本和上市速度的角度來看，這確實可以帶來巨大的好處。

And then other markets, of course, we will continue -- we need to do more of the homework on this, as we said, but we also understand Japan could be a potential or Asia Pacific, but these are more over time as we have the US FDA approval once we have it, then that's our next chapter we want to get there.

然後當然，我們會繼續開拓其他市場——正如我們所說的，我們需要在這方面做更多的功課，但我們也知道日本可能是一個潛在的市場，或者亞太地區，但隨著時間的推移，隨著我們獲得美國 FDA 的批准，這些都是我們想要實現的下一個篇章。

Thank you.

謝謝。

(Operator Instructions)

（操作員指示）

David Bautz, Zacks Small Cap Research.

David Bautz，Zacks Small Cap Research。

Hey, good morning, everyone. Thanks for the overview this morning. You had mentioned that you want to get five treatment centers online with launch when you launch but what is the ultimate goal for the total number of treatment centers you'd like to see one day? Is it to get it up to 10, 15? What do you see in terms of that?

嘿，大家早安。感謝您今天上午的概述。您曾提到，您希望在啟動時就讓五個治療中心上線，但您希望有一天看到的治療中心總數的最終目標是什麼？是不是要達到 10、15 呢？您對此有何看法？

I think total number, not -- at this point in time, we don't anticipate going more than 10. And Yes. So 5 this year and building it up slowly. Part of the reason is because we see that patients already in the status quo are used to traveling to centers that have specialty to be able to do multiple procedures, right, either for infections or surgical procedures, syndactaly and for various reasons, they are comfortable traveling not for pediotic application, but for these kinds of onetime important procedures.

我認為總數，不是——目前，我們預計不會超過 10。是的。所以今年有 5 個，並且慢慢增加。部分原因是，我們看到現況患者已經習慣前往具有專科治療中心進行多項手術，無論是感染還是外科手術、並指症，出於各種原因，他們願意前往就診，不是為了兒科治療，而是為了這些一次性的重要手術。

Now which is a therapeutic options, we see and we have heard from patients that there is a very high willingness to travel to these centers as far as 200, 250 miles radius. So we want to keep the number of centers tight and build the experience curve for these centers. So that as they do more patients and more applications, they just get better and better at it. So that's really our goal.

現在，這是一種治療選擇，我們看到並且聽到患者表示，他們非常願意前往半徑 200 至 250 英里範圍內的中心接受治療。因此，我們希望控制中心數量，並為這些中心建立經驗曲線。因此，隨著他們治療的患者越來越多、應用越來越多，他們的技術就會越來越好。這確實是我們的目標。

And that also matches up with our supply, right? Because one of the things having launched CAR T cell therapies in our past is we got ran into issues with supply and keeping up the expectations. So we are -- we're careful in terms of keeping that experience going given where we are with regards to our supply situation as well. And as we ramp our supply, then yes, certainly, we will explore ways to have more centers onboarded.

這也與我們的供應相符，對嗎？因為我們過去推出 CAR T 細胞療法時遇到的問題之一就是供應和保持預期方面的問題。因此，考慮到我們的供應狀況，我們會謹慎地保持這種經驗。隨著我們增加供應量，我們當然會探索讓更多中心加入的方法。

Okay. Great. Thanks for that. And in terms of treatment, you mentioned that most patients are probably going to require two treatments. And I'm just curious, what is the maximum service area that a patient can be treated with at one time? And then what kind of dictates that number?

好的。偉大的。謝謝。在治療方面，您提到大多數患者可能需要兩種治療。我只是好奇，病人一次可以接受治療的最大服務區域是多少？那是什麼決定了這個數字呢？

So the maximum surface area that can be treated is we can supply up to 12 sheets in collagen producing [genocide] sheets and 40-centimeter square is the size for each of the sheet. So 12 times 40 up to 480-centimeter square.

因此，我們可以提供最多 12 張可產生膠原蛋白的 [種族滅絕] 薄片，每張薄片的尺寸為 40 平方公分。因此 12 乘以 40 等於 480 平方公分。

From the natural history studies, we've seen that patients have, on average, 30%, actually some patients have even up to 80% of their bodies wounded for recessive DEB. So if we go with 30% and we come back of the envelope body surface area analysis, we estimate around 1,000, 1,000 centimeter square of body is covered in large chronic wounds and non-flyxian parts of the body.

從自然史研究中，我們發現患者平均有 30% 的身體因隱性 DEB 而受損，實際上有些患者甚至高達 80% 的身體因隱性 DEB 而受損。因此，如果我們採用 30% 的比例，然後重新進行體表面積分析，我們估計大約有 1,000 平方厘米的身體面積被大面積慢性傷口和非飛行部位所覆蓋。

So that -- with that calculation, we are estimating around two rounds because 2 times, 480 but then there is -- we cannot guarantee that there's going to be 12 sheets for each and every patient, right, because it's a cellular engineered cell therapy and so for that reason, there is a chance that we may not be probably it's 8 or 10.

因此 — — 透過這個計算，我們估計大約需要兩輪，因為 2 乘以 480，但是 — — 我們不能保證每個病人都有 12 張紙，對吧，因為這是一種細胞工程細胞療法，因此，我們有可能不是 8 張或 10 張。

So that just -- those are the factors that we'll have to see over time how many treatment cycles will be required and also patients coming back to get their additional parts of their body treated. So that's sort of the math behind how we estimate about two treatments per patient.

所以這只是——這些因素，我們必須隨著時間的推移觀察需要多少個治療週期，以及患者是否需要回來接受身體其他部位的治療。這就是我們估算每位患者需要接受兩次治療的數學原理。

Okay. Great. I appreciate the information there. Thanks for taking my question.

好的。偉大的。我很感謝那裡的資訊。感謝您回答我的問題。

Thank you. There were no other questions in queue at this time. I would now like to hand the call back to Vish Seshadri for closing remarks.

謝謝。此時隊列中沒有其他問題。現在我想把發言權交還給 Vish Seshadri，請他做最後發言。

Thank you, Paul. Our top priorities continue to be to secure FDA approval for and launching pz-cel in the US as a new treatment for RDEB patients. We believe we are well positioned for the significant opportunity ahead of Abeona, and we look forward to a potentially transformative 2025 for the company. Thank you, everyone, for joining us for today's business update. We'll talk to you again soon.

謝謝你，保羅。我們的首要任務仍然是獲得 FDA 批准並在美國推出 pz-cel 作為 RDEB 患者的新治療方法。我們相信，我們已準備好迎接 Abeona 面臨的重大機遇，並期待公司在 2025 年實現變革。感謝大家收看今天的業務更新。我們很快會再和你聯絡。

Thank you. This does conclude today's conference. You may disconnect your lines at this time. Thank you for your participation.

謝謝。今天的會議到此結束。現在您可以斷開線路。感謝您的參與。