Abeona Therapeutics Inc (ABEO) 2026 Q1 法說會逐字稿

Good day, ladies and gentlemen, and welcome to the Abeona Therapeutics first quarter 2026 earnings conference call. (Operator Instructions). And please note, this conference is being recorded.

各位女士、先生，大家好，歡迎參加 Abeona Therapeutics 2026 年第一季財報電話會議。(接線員指示)。並請注意，本次會議將被錄音。

I will now turn the conference over to your host, Mr. Joe Vazzano, Chief Financial Officer of Abeona Therapeutics. Sir, the floor is yours.

現在我將把會議交給本次會議主持人——Abeona Therapeutics 財務長 Joe Vazzano 先生。先生，請開始。

Thank you, operator. Good morning, and thank you for joining us on our first quarter 2026 results and business update conference call. During this call, we will refer to the press release issued this morning announcing the financial results, which is available on our corporate website at www.abeonatherapeutics.com.

謝謝，接線員。各位早安，感謝各位參加我們 2026 年第一季業績與業務更新電話會議。在本次電話會議中，我們將引用今天早上發布、公布財務結果的新聞稿；該新聞稿可於我們公司網站 www.abeonatherapeutics.com 查閱。

We anticipate making projections and forward-looking statements during today's call, 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 due to various factors, including, but not limited to, those outlined in our Form 10-K and periodic reports filed with the Securities and Exchange Commission. These documents are available on our website at www.avonathapeutics.com.

我們預期在今天的電話會議中將提出預測與前瞻性陳述，該等陳述係依據聯邦證券法的「安全港」條款作出。這些前瞻性陳述係基於目前的預期，且可能發生變動。由於各種因素，實際結果可能與前瞻性陳述中明示或暗示的內容存在重大差異，包括但不限於我們在 Form 10-K 及向美國證券交易委員會提交的定期報告中所列示的因素。這些文件可於我們網站 www.avonathapeutics.com 查閱。

Joining me on today's call with prepared remarks are Dr. Vishwas Seshadri, Chief Executive Officer; and Dr. Madhav Vasanthavada, Chief Commercial Officer. With that, I will now turn the call over to Dr. Seshadri to kick us off. Vish?

今天與我一同出席並準備發言的有：執行長 Vishwas Seshadri 醫師，以及商務長 Madhav Vasanthavada 醫師。接下來，我將把電話交給 Seshadri 醫師為我們開場。Vish？

Thank you, Joe, and good morning, everyone. First, we are excited to share updates on leading indicators of ZEVASKYN adoption that signals strong momentum since treating our first commercial patients in December. We have now activated six qualified treatment centers or QTCs, treated our fifth commercial patient with manufacturing underway for the sixth and scheduled additional patients throughout the current quarter. The recent acceleration of onboarding efforts of QTCs further underscores their conviction about the role that ZEVASKYN will play in addressing the unmet needs of patients suffering from recessive dystrophic epidmolysis bullosa or RDEB. Three of the ZEVASKYN treatments reported to date took place in Q1 2026 and translated into net revenue of $8.7 million for that quarter.

謝謝你，Joe，各位早安。首先，我們很高興分享 ZEVASKYN 採用的領先指標更新；自 12 月治療首批商業患者以來，這些指標顯示出強勁動能。我們目前已啟用六家合格治療中心（QTC），已治療第五位商業患者，第六位患者的製造作業正在進行中，並已在本季安排更多患者。近期 QTC 導入（onboarding）工作的加速，進一步凸顯他們對 ZEVASKYN 在滿足隱性營養不良性表皮分解性水皰症（recessive dystrophic epidermolysis bullosa，RDEB）患者未被滿足需求方面所扮演角色的信心。截至目前回報的 ZEVASKYN 治療中，有三例發生在 2026 年第一季，並為該季帶來 870 萬美元的淨營收。

Second, we're sharing meaningful updates to our R&D pipeline, featuring a potentially game-changing radically novel engineered T cell technology for advanced prostate cancer. By leveraging our proven expertise in advancing complex cell and gene therapies from academia through commercialization, we are well positioned to advance this exciting technology.

第二，我們將分享研發產品線的重要更新，內容包括一項可能改變遊戲規則、極具突破性的全新工程化 T 細胞技術，用於晚期前列腺癌。藉由運用我們在推進複雜細胞與基因療法從學術端走向商業化方面已被驗證的專業能力，我們具備良好條件來推進這項令人振奮的技術。

But before going there, I'll first turn the call over to Dr. Madhav Vasanthavada to elaborate on the ZEVASKYN launch, which is our foundational and primary focus for Abeona. Madhav?

不過在談到那部分之前，我先把電話交給 Madhav Vasanthavada 醫師，請他進一步說明 ZEVASKYN 的上市推進情況；這是 Abeona 的基石，也是我們的首要重點。Madhav？

Thank you, Vish, and good morning, everyone. Launch momentum for ZEVASKYN and our commercial story continues to build, and we are beginning to see results on multiple fronts.

謝謝你，Vish，各位早安。ZEVASKYN 的上市動能與我們的商業故事持續累積，我們開始在多個面向看到成果。

I'd like to start off by providing you with visibility not only to patients treated so far, but also biopsies expected this quarter. As previously shared, one patient, our very first commercial patient was treated in the fourth quarter of 2025 and three patients were treated in the first quarter of this year.

我想先讓各位更清楚了解：截至目前已治療的患者情況，以及本季預期進行的活檢。如先前分享，我們的第一位商業患者於 2025 年第四季接受治療，而今年第一季共有三位患者接受治療。

Additionally, one patient has been treated so far this quarter for a total of five patients treated to date with ZEVASKYN since launch. The forward-looking momentum of patients in queue is also picking up with one patient biopsy and manufacturing for that patient currently underway and six additional patients expected to be biopsied this quarter, three of whom actually just as of this morning, four of whom have scheduled biopsies.

此外，本季迄今已有一位患者接受治療，使得自上市以來使用 ZEVASKYN 治療的患者總數達到五位。前瞻性的排隊患者動能也在加快：目前已有一位患者完成活檢且該患者的製造作業正在進行中，並預期本季另有六位患者將接受活檢，其中三位（截至今天早上）已完成活檢，另有四位已排定活檢時間。

I'd like to add that all patients treated to date and those scheduled for biopsies are from our first two activated QTCs. The other QTCs have identified patients and are not far behind in scheduling for biopsy, which will further add to ZEVASKYN treatments in the coming quarters. While we are pleased to see patients beginning to clear the upstream procurement process and receiving ZEVASKYN treatment, we are equally encouraged by the strong demand reflected in the near-term identified pool of more than 100 patients across our QTCs and the community-based physicians.

我也想補充說明：截至目前所有已治療患者以及已排定活檢的患者，皆來自我們最先啟用的兩家 QTC。其他 QTC 也已辨識出患者，距離安排活檢不遠，這將在未來幾季進一步增加 ZEVASKYN 的治療量。我們很高興看到患者開始通過上游的取得（procurement）流程並接受 ZEVASKYN 治療；同時，我們也同樣受到強勁需求的鼓舞——在我們的 QTC 與社區型醫師網絡中，短期內已辨識出的患者池超過 100 位。

Our field teams are executing well, building deep relationships, expanding awareness and driving broad reach across dermatology, pediatric dermatology and subspecialties involved in the care of EB patients. We continue to engage with referral physician community and have active conversations ongoing with 45 physicians. These are not just one-off touch points, but action-oriented back-and-forth interactions, which shows real clinical interest and their intent to refer patients for ZEVASKYN.

我們的外勤團隊執行表現良好，建立深厚關係、擴大認知度，並在皮膚科、小兒皮膚科以及參與 EB 患者照護的次專科領域中推動廣泛觸及。我們持續與轉診醫師社群互動，目前正與 45 位醫師進行積極對話。這些互動並非一次性的接觸點，而是以行動為導向的雙向往來，顯示出真實的臨床興趣，以及他們轉介患者使用 ZEVASKYN 的意圖。

Beyond the numbers, early qualitative launch insights are also encouraging and reinforce our conviction. Importantly, we are hearing positive feedback from QTCs that have treated patients and their experience with the end-to-end process is getting better with every patient treated. To elaborate further on the types of initial patients that have been treated and those in the queue, we are happy to note that the initial uptake of ZEVASKYN is not confined to a narrowly defined patient or payer type, but has spanned across both adults and children with one patient as young as five years of age.

除了數字之外，早期的質性上市洞察也令人鼓舞，並強化了我們的信心。重要的是，我們從已治療患者的 QTC 收到正面回饋，他們表示端到端流程的體驗隨著每位患者的治療而持續改善。進一步說明已治療的初期患者類型以及排隊中的患者，我們很高興指出：ZEVASKYN 的初期採用並未侷限於狹義界定的患者或支付方類型，而是涵蓋成人與兒童，其中一位患者年僅 5 歲。

Our payer mix consists of both commercial and Medicaid insurers in getting the breadth of ZEVASKYN coverage. We are seeing that geographic proximity to QTC has not been a barrier because patients have traveled significant distances, including across state lines to receive treatment and our Abeona Assist patient and caregiver support programs have received positive feedback.

我們的支付方組合同時包含商業保險與 Medicaid 保險，顯示 ZEVASKYN 覆蓋面的廣度。我們也看到，與 QTC 的地理距離並未構成障礙，因為患者為了接受治療已長途跋涉，甚至跨州就醫；同時，我們的 Abeona Assist 患者與照護者支持計畫也獲得正面回饋。

Among the patients treated is our very first patient in the commercial setting who was biopsied in August of 2025, but as you may recall, could not receive ZEVASKYN due to a false positive result from a stability assay. This patient came back to be re-biopsied early this year, and we are pleased to tell you that this patient was treated successfully. Such determination of patients, families and physicians to pursue ZEVASKYN speaks volumes about what this therapy means to them.

在已治療的患者中，包括我們在商業環境下的第一位患者；該患者於 2025 年 8 月完成活檢，但如各位所記得，因穩定性檢測（stability assay）出現偽陽性結果而無法接受 ZEVASKYN。該患者於今年年初回來重新活檢，我們很高興告訴各位，該患者已成功接受治療。患者、家屬與醫師為了使用 ZEVASKYN 所展現的決心，充分說明了這項療法對他們的意義。

On the market access front, payer coverage continues to strengthen with the percentage of commercially covered lives with published ZEVASKYN policies now reaching 95%. This is a significant accomplishment in the first year post ZEVASKYN approval. That said, we are navigating a lengthy insurance approval process, which is typical of any high-cost gene therapy at launch, particularly for out-of-state Medicaid patients. Even so, we have seen no patient attrition and no final payer denials to date, further underscoring the strength of ZEVASKYN's value proposition to RDEB patients and their families.

在市場准入方面，支付方覆蓋持續強化；目前已發布 ZEVASKYN 給付政策的商業保險覆蓋人數占比已達 95%。這是在 ZEVASKYN 獲批後第一年內的一項重大成就。儘管如此，我們仍在應對冗長的保險核准流程；這是任何高成本基因療法在上市初期的典型情況，尤其是對於外州的 Medicaid 患者。即便如此，截至目前我們未見患者流失，也未出現任何最終的支付方拒賠，進一步凸顯 ZEVASKYN 對 RDEB 患者及其家庭的價值主張之強勁。

As we continue to follow patients from our Phase 1/2a and Phase 3 trials, we are excited to share that new data will be presented later this week at the Society for Investigative Dermatology, SID, featuring five-year follow-up of our VIITAL Phase 3 trial as well as a single patient 12 years of follow-up from Phase 1/2a study, all of which reinforce durable wound healing and favorable safety profile after a onetime product application. On the patient side, our Strong Together Network continues to be a powerful voice with patients and caregivers sharing their experiences from clinical trials and helping to generate patient self-referrals. As our initial ZEVASKYN commercial patients share their experiences over time, we expect these stories to become one of the most powerful demand drivers available to us in this rare disease setting.

隨著我們持續追蹤第 1/2a 期與第 3 期試驗中的患者，我們很高興分享，本週稍晚將於皮膚科研究學會（Society for Investigative Dermatology，SID）發表新數據，內容包括我們 VIITAL 第 3 期試驗的五年追蹤結果，以及第 1/2a 期研究中單一患者長達 12 年的追蹤結果；這些結果皆再次證實，在一次性產品施用後，傷口癒合具持久性且安全性概況良好。在患者端，我們的 Strong Together Network 持續成為強而有力的發聲平台，患者與照護者分享其臨床試驗經驗，並協助促成患者自我轉介。隨著我們初期的 ZEVASKYN 商業化患者隨時間分享其經驗，我們預期這些故事將成為在此罕見疾病情境中我們可運用的最強需求驅動因素之一。

Lastly, we continue to onboard more ZEVASKYN treatment centers. As announced, we activated NewYork-Presbyterian/Columbia University last month. And Monday of this week, we announced the activation of Children's Hospital of Philadelphia, CHOP, as our sixth QTC. I want to sincerely thank all my team members involved in the onboarding of these centers and to recognize our QTC physician champions and their team's conviction in ZEVASKYN as they successfully navigated a several month long onboarding process.

最後，我們持續導入更多 ZEVASKYN 治療中心。如先前宣布，我們於上月啟用紐約長老會醫院／哥倫比亞大學（NewYork-Presbyterian/Columbia University）。而在本週一，我們宣布啟用費城兒童醫院（Children's Hospital of Philadelphia，CHOP）作為我們第六家 QTC。我想由衷感謝所有參與這些中心導入工作的團隊成員，並肯定我們的 QTC 醫師領袖及其團隊對 ZEVASKYN 的信念，他們成功完成了長達數月的導入流程。

As you can gather from the map, we importantly have QTCs spanning the nation across geographically distinct regions: California, Colorado, Texas and the Gulf Coast, Chicago and now the East Coast. We continue to have active discussions with additional centers and remain well on track to achieving our goal of having a total of seven QTCs onboarded this year and ensuring even greater access for patients and families across the country.

從地圖上各位可以看出，我們的重要 QTC 佈局已橫跨全美、涵蓋地理上不同的區域：加州、科羅拉多州、德州與墨西哥灣沿岸、芝加哥，以及現在的東岸。我們仍與其他中心進行積極討論，並持續穩步朝今年導入合計七家 QTC 的目標前進，以確保全國各地的患者與家庭能獲得更大的可近性。

To close, we are progressing through the launch, accruing positive early feedback from treating physicians, a growing referral base, expanding QTC network and achieving broad payer acceptance. Every successful biopsy, every treatment and every positive patient story is reinforcing our conviction in ZEVASKYN.

總結而言，我們正推進上市進程，並持續累積治療醫師的正面早期回饋、擴大的轉介基礎、擴張中的 QTC 網絡，以及廣泛的支付方接受度。每一次成功的活檢、每一次治療，以及每一則正面的患者故事，都在強化我們對 ZEVASKYN 的信心。

With that, I'll turn the call back to Dr. Seshadri for an update on our R&D pipeline. Vish?

接下來，我把電話交回給 Seshadri 醫師，請他更新我們的研發產品線。Vish？

Thank you, Madhav. Now, I'll share some important pipeline updates that highlight our focus on assets that align with our core competencies and what we believe would deliver the greatest long-term value. As part of this focused effort, we have deprioritized our in-house ophthalmology preclinical programs. Abeona has demonstrated capabilities with ZEVASKYN over the past years in end-to-end development and commercialization of personalized high-value cell therapies with durable clinical benefits for patients with debilitating diseases.

謝謝你，Madhav。接下來我將分享一些重要的產品線更新，凸顯我們聚焦於與核心能力一致、且我們相信能帶來最大長期價值的資產。作為此一聚焦努力的一部分，我們已降低對內部眼科臨床前計畫的優先順序。過去數年，Abeona 透過 ZEVASKYN 已展現端到端開發與商業化個人化高價值細胞療法的能力，並為罹患致殘性疾病的患者帶來持久的臨床效益。

Today, we announced the in-licensing of a radically novel cell therapy asset that targets PSMA or prostate-specific membrane antigen, a validated target for the treatment of advanced prostate cancer, a leading cause of cancer mortality with more than 30,000 deaths annually in the US. The CAR T-technology was pioneered by Dr. Preet Chaudhary, Founder of Angelist Therapeutics and Professor of Medicine at the University of Southern California. He has more than 200 granted or pending patents worldwide in the field of cell therapy. We have included a link to a recent talk by Dr.

今天，我們宣布引進授權一項極具突破性的全新細胞療法資產，該資產鎖定 PSMA（前列腺特異性膜抗原），這是治療晚期前列腺癌的已驗證標的；前列腺癌是癌症死亡的主要原因之一，在美國每年造成超過 30,000 人死亡。此 CAR T 技術由 Angelist Therapeutics 創辦人、南加州大學醫學教授 Preet Chaudhary 醫師所開創。他在細胞療法領域於全球擁有超過 200 件已核准或申請中的專利。我們在今日的簡報中附上 Chaudhary 醫師近期演講的連結。

Chaudhary in today's slides, elaborating on the uniqueness and promise of this technology in oncology. PSMA-SIR-T or ABO-701 is an autologous engineered T-cell therapy that carries a PSMA-directed synthetic immune receptor purposefully structured to overcome the limitations of CARs or chimeric antigen receptors and TCRs, which is T-cell receptors. The SIR-T technology is unique in that it can directly recognize and bind a target membrane antigen like a CAR does without the need for antigen presentation. However, it retains the physiologic signaling and regulatory features of a native T-cell receptor, which enables more controlled, durable immune-mediated cell death.

該演講進一步闡述此技術在腫瘤學上的獨特性與潛力。PSMA-SIR-T（或 ABO-701）是一種自體工程化 T 細胞療法，攜帶一個以 PSMA 為導向的合成免疫受體，其設計目的在於克服 CAR（嵌合抗原受體）與 TCR（T 細胞受體）的限制。SIR-T 技術的獨特之處在於，它可如同 CAR 一樣直接辨識並結合目標膜抗原，而無需抗原呈現。然而，它仍保留原生 T 細胞受體的生理性訊號傳導與調控特徵，使免疫介導的細胞死亡更可控且更持久。

In preclinical studies, PSMA-SIR-T demonstrated the ability to achieve deep and durable PSMA-specific antitumor responses in mouse models and display exceptionally modest levels of cytokine release in vitro, a profile that has been elusive for other engineered cell therapies in solid tumors. The elimination of tumors in most mice treated with PSMA-SIR-T and its superior performance versus corresponding PSMA CAR-T comparator controls suggests a more controlled and durable immune activation in treated mice. We believe these data support a compelling hypothesis that SIR-T technology may overcome key limitations that have historically constrained engineered T-cell therapies in solid tumors. We anticipate IND filing and first-in-human studies to commence in the second half of 2027. In the near-term, we will gain regulatory alignment beginning with a pre-IND meeting with the FDA on June 3, 2026, and engage a CDMO for supply readiness, while our internal teams maintain operational focus on ZEVASKYN commercialization.

在臨床前研究中，PSMA-SIR-T 在小鼠模型中展現達成深度且持久的 PSMA 特異性抗腫瘤反應的能力，並在體外呈現極低的細胞激素釋放水準；此一特徵在其他針對實體腫瘤的工程化細胞療法中一直難以達成。PSMA-SIR-T 在多數受治療小鼠中可消除腫瘤，且相較於對應的 PSMA CAR-T 對照組表現更佳，顯示在受治療小鼠中可引發更可控且更持久的免疫活化。我們相信這些數據支持一個具吸引力的假說：SIR-T 技術可能克服歷來限制工程化 T 細胞療法於實體腫瘤應用的關鍵瓶頸。我們預期將於 2027 年下半年提交 IND，並啟動首次人體試驗。短期內，我們將自 2026 年 6 月 3 日與 FDA 的 pre-IND 會議開始取得法規一致性，並與 CDMO 合作以確保供應就緒；同時，我們內部團隊將維持對 ZEVASKYN 商業化的營運聚焦。

With that, I'll now pass the call to our Chief Financial Officer, Joe Vazzano, to discuss our first quarter financial results. Joe?

接下來，我將把電話交給我們的財務長 Joe Vazzano，請他說明第一季財務結果。Joe？

Thank you, Vish. I would like to remind everyone that you could find additional details on our financial results for the first quarter ending March 31, 2026, in our most recent 10-Q. We reported total net product revenue of $8.7 million for the first quarter of 2026. All three patients treated in the quarter were commercially insured patients. This reflects a strong quarter-over-quarter increase of $6.3 million compared to $2.4 million in the fourth quarter of 2025.

謝謝你，Vish。我想提醒各位，關於截至 2026 年 3 月 31 日第一季的財務結果，更多細節可在我們最新的 10-Q 中查閱。我們在 2026 年第一季的產品淨營收總額為 870 萬美元。本季接受治療的三位患者皆為商業保險患者。相較於 2025 年第四季的 240 萬美元，這代表季對季大幅增加 630 萬美元。

The growth was driven by early commercial traction following the launch of ZEVASKYN. Cost of sales for the quarter was $2.7 million compared to $1 million in the prior quarter. The increase was primarily driven by the scaling of commercial ZEVASKYN with three patient treatments in Q1 versus one treatment in Q4.

此成長主要由 ZEVASKYN 上市後的早期商業動能所帶動。本季銷貨成本為 270 萬美元，較前一季的 100 萬美元增加。增加的主因是商業化 ZEVASKYN 的規模擴張：第一季完成三位患者治療，而第四季僅一位。

Turning to operating expenses. R&D expenses were $9.6 million compared to $9.9 million in the first quarter of 2025. Notably, Q1 2026 includes a $7 million upfront payment related to the in-licensing of our PSMA-SIR-T asset. Excluding this transaction, R&D expenses declined meaningfully, reflecting the transition of certain manufacturing costs capitalized to inventory and engineering runs that are no longer considered R&D following the FDA approval of ZEVASKYN.

接著談營運費用。研發費用為 960 萬美元，較 2025 年第一季的 990 萬美元略降。值得注意的是，2026 年第一季包含與引進授權 PSMA-SIR-T 資產相關的 700 萬美元一次性預付款。若排除此交易，研發費用顯著下降，反映部分製造成本已轉為資本化並計入存貨，以及在 FDA 核准 ZEVASKYN 後，某些工程批次（engineering runs）不再被視為研發費用。

Selling, general and administrative expenses were $19.5 million, representing an increase of $9.7 million year-over-year first quarter. This increase was expected and reflects our continued investment in commercial infrastructure post approval. Key drivers include $5.4 million in personnel and stock-based compensation, $1.9 million of costs related to engineering runs with the remainder due to other commercialization costs. Net loss for the quarter was $17.1 million or $0.30 per basic and diluted common share compared to a net loss of $12 million or $0.24 per basic and diluted common share in the first quarter of 2025. The year-over-year change primarily reflects increased commercial investment and the PSMA-SIR-T licensing transaction.

銷售、一般及行政費用為 1,950 萬美元，較去年同期第一季增加 970 萬美元。此增幅符合預期，反映我們在核准後持續投資商業化基礎建設。主要驅動因素包括 540 萬美元的人員與以股份為基礎之酬勞、190 萬美元與工程批次相關的成本，其餘則來自其他商業化成本。本季淨損為 1,710 萬美元，或每股基本及稀釋普通股淨損 0.30 美元；相較之下，2025 年第一季淨損為 1,200 萬美元，或每股基本及稀釋普通股淨損 0.24 美元。年對年變動主要反映商業化投資增加以及 PSMA-SIR-T 授權交易的影響。

We ended the quarter with $168.3 million in cash, cash equivalents and short-term investments compared to $191.4 million at the end of 2025. Our balance sheet remains strong and positions us well to support continued commercial execution and pipeline advancement. We anticipate minimal R&D expenditures for the PSMA program, limited to low single-digit million dollars for the remainder of this year. Overall, we are encouraged by the early commercial progress of ZEVASKYN and remain disciplined in our capital allocation as we scale the business.

我們在本季末持有現金、約當現金及短期投資合計1.683億美元，相較於2025年底的1.914億美元。我們的資產負債表仍然穩健，使我們能夠有利地支持持續的商業執行與產品線（pipeline）推進。我們預期PSMA專案的研發支出將維持在最低水準，今年剩餘期間僅限於低個位數百萬美元。整體而言，我們對ZEVASKYN早期的商業進展感到鼓舞，並在擴大業務規模的同時，持續嚴守資本配置紀律。

With that, I'll pass the call back to Vish for closing remarks before opening the call for Q&A. Vish?

接下來，我會把電話交回給Vish，請他在開放問答前做結語。Vish？

To summarize, we are encouraged by favorable trends in leading indicators of ZEVASKYN launch performance. That is the foundation on which we have taken a bold step in advancing PSMA SIR-T development. Every milestone we discussed ultimately connects back to patients with serious diseases who are waiting for better, more innovative medicines. Our mission is not just a statement, but a commitment that guides how we allocate capital, how we prioritize our pipeline and how we measure success.

總結來說，我們對ZEVASKYN上市表現之領先指標所呈現的正向趨勢感到鼓舞。這正是我們大膽推進PSMA SIR-T開發的基礎。我們討論的每一個里程碑，最終都與正在等待更好、更具創新藥物的重症患者息息相關。我們的使命不僅是一句口號，而是一項承諾，指引我們如何配置資本、如何排定產品線優先順序，以及如何衡量成功。

With that, I request the operator to open the floor for questions.

接下來，我請接線員開放提問。

(Operator Instructions) Kristen Kluska, Cantor Fitzgerald.

（接線員指示）Kristen Kluska，Cantor Fitzgerald。

Congrats on all the progress here around ZEVASKYN. So now that you have five patients treated and quite a few in the biopsy pipeline, can you give us a sense of what the typical patient profile has looked like across treatment? Are these more severe patients? Are any of that who have come back from the clinical trial to get another cycle? And for those that have undergone the procedure already, have they commented on whether they would be interested in potentially coming back in the future for another cycle?

恭喜你們在ZEVASKYN方面取得的所有進展。既然目前已治療五位患者，且在活檢流程中還有不少患者，能否請你們說明一下，整體治療下來典型的患者輪廓大概是什麼？這些患者是否屬於較嚴重的個案？是否有任何先前參與臨床試驗的患者回來接受第二個療程？另外，對於已完成手術程序的患者，他們是否有提到未來可能願意再回來接受另一個療程？

Hi, Kristen, thanks for that question. Yes. So with regard to your first question about the patient profile, what we hear from physicians are these are severe patients as we had expected. And many of those patients treated would require more than 12 sheets of ZEVASKYN.

嗨，Kristen，謝謝你的問題。是的。關於你第一個問題、也就是患者輪廓，我們從醫師那裡聽到的是，這些確實是如我們預期的重症患者。而且許多已治療的患者需要超過12片的ZEVASKYN。

So there is still an unmet need even in these treated patients. Of course, it's early to say how many of these patients will come back and at what point in time will they come back for a second treatment, but there is definitely a clinical need from that standpoint.

因此，即使在這些已治療的患者身上，仍存在未被滿足的需求。當然，現在還太早，無法判斷有多少患者會回來、以及會在什麼時間點回來接受第二次治療，但從這個角度來看，臨床上的需求確實存在。

The second part, clinical trial patients, they are interested, and we know that patient consults are happening. It's just a matter of for those patients, when would be the right time for them to come in for a retreatment with ZEVASKYN. And so, we'll keep you updated if we have that kind of information. From the patients who have received ZEVASKYN already, yes, I think I already addressed that part, which is we don't know exactly when they'll come in, but there is certainly a need for.

第二部分，關於臨床試驗患者，他們是有興趣的，我們也知道患者諮詢正在進行。只是對那些患者而言，何時才是回來以ZEVASKYN進行再次治療的適當時機。因此，如果我們有這類資訊，會持續向各位更新。至於已經接受過ZEVASKYN的患者，是的，我想我剛才已經回覆到這一點，也就是我們不確定他們會在何時回來，但確實存在這方面的需求。

And then, just as we think about how to model this out for 2Q, we know one patient has officially been treated. And all this color is really helpful around the biopsy schedule. But just what can you tell us about your sense of how many patients you ultimately believe will have the procedure, meaning you get paid for it in 2Q versus how we should be thinking about maybe some of these trickling into next quarter?

另外，當我們思考如何為第二季建模時，我們知道已有一位患者正式完成治療。你們提供的關於活檢排程的補充說明非常有幫助。但你們能否談談，你們認為第二季最終會有多少患者完成該程序（也就是你們能因此取得款項）？以及我們應如何看待其中一些可能延後到下一季才陸續發生？

Thanks for that question, Kristen. It's hard to precisely place how many of the -- I think we gave visibility to at least eight patients today in the call, one treated, one in manufacturing process and six that are in the biopsy scheduling process. So we could anticipate that maybe one or two of those patients who may receive who may be biopsied in June, anything after the first week of June would fall into July treatment. Is it one? Is it two? Is it three? It's very hard to predict because some may be in the border line and the manufacturing turnaround time is not a very precise number, even though we have it approximately 23 or 24 days. That is something that we have to see. But a good chunk of the patients that we have described today should fall under quarter 2 treatment.

謝謝你的問題，Kristen。要精準判斷有多少位——我想我們今天在電話會議中至少讓大家看見了八位患者的能見度：一位已治療、一位在製造流程中，以及六位正在安排活檢排程。因此我們可以預期，可能有一到兩位若在六月接受活檢，且是在六月第一週之後的，治療可能會落在七月。是一位嗎？是兩位嗎？是三位嗎？這很難預測，因為有些可能處於臨界點，而製造周轉時間也不是非常精準的數字，儘管我們大約是23或24天。這需要再觀察。但我們今天描述的患者中，有相當一部分應該會落在第二季的治療。

Maury Raycroft, Jefferies.

Maury Raycroft，Jefferies。

Congrats on the progress. Maybe as a follow-up to Kristen's last question. For the patients treated so far, from my understanding, they've been treated at Laury and Stanford. Can you clarify what other QTCs are fully activated? And it may be too early for this, but can you provide some bookending for what patient volume could look like per QTC or across the QTCs for 2026 and maybe what steady state could look like eventually as well?

恭喜進展。或許接續Kristen上一個問題。就我理解，目前已治療的患者是在Lurie和Stanford接受治療。你能否釐清還有哪些QTC已完全啟動？另外，可能現在還太早，但你能否提供一些上下界，說明2026年每個QTC或整體QTC的患者量可能會是什麼樣子，以及最終達到穩態時可能會呈現什麼規模？

Yes, Maurice. So in addition to Lurie Children's and Stanford Children's, we have Colorado Children's Hospital that's active and UTMB University of Texas and Galveston, which is also active. And of course, the most recent ones were Columbia and CHOP. We do know that Colorado and UTMB have patients actively identified and they are working through the administrative process to put them on, and we expect that we should receive biopsy schedule requests for their patients imminently.

好的，Maurice。除了Lurie Children's和Stanford Children's之外，我們還有Colorado Children's Hospital已啟動，以及德州大學醫學分部（UTMB）加爾維斯頓也已啟動。當然，最近啟動的還有Columbia和CHOP。我們確實知道Colorado與UTMB已主動辨識出患者，並正在完成行政流程以將他們納入，我們預期很快就會收到他們患者的活檢排程申請。

And in terms of the volume, these are centers. Colorado is actually a very well-known institution for EB care. And in terms of the cadence, what we have been hearing from QTC is treating one patient a month at a steady state is quite doable. So it's just a matter of getting these patients initiated with biopsy and the treatment cadence.

至於量能方面，這些都是中心。Colorado其實是EB照護領域非常知名的機構。就治療節奏而言，我們從QTC聽到的是，在穩態下每月治療一位患者是相當可行的。因此，關鍵只是讓這些患者開始進入活檢與治療節奏。

Based on the comments in the prepared remarks around the length of the insurance approval process, it seems like ultimately, this is not limiting usage, but is this something that you have line of sight on that you can improve? And how can improving this factor into your ability to fine-tune projections? And then do you anticipate there could be greater pushback or friction when it comes to retreating patients?

根據你們在事先準備的講稿中對保險核准流程時間長度的評論，看起來最終這並不會限制使用量，但你們是否已掌握可改善的方向？而改善這點將如何影響你們微調預測的能力？另外，你們是否預期在患者再次治療時，可能會遇到更大的反對或摩擦？

Yes. Definitely, the process will improve. Oftentimes, with gene therapy, especially high-cost gene therapy, the initial process of payer clearance, especially if a patient is traveling from out of state, there is additional layers of paperwork that need to be secured, starting with physicians also need to be enrolled. It's a onetime enrollment.

是的。這個流程一定會改善。很多時候，在基因治療，尤其是高成本基因治療上，最初的付款方核准流程——特別是當患者需要跨州就醫時——會有額外的文件層級需要完成，其中也包括醫師需要先完成登錄。這是一次性的登錄。

For example, if a patient is coming from traveling from a different state to receive treatment in one of these QTC states, then the physician from the qualified center, whether it's a surgeon, anesthesiologist or the EB physician need to be enrolled in sort of the out-of-state patient state. So that is a one-time process as well as just providing a fee schedule.

例如，如果一位病患從其他州旅行前來，欲在這些 QTC 州之一接受治療，那麼合格中心的醫師——不論是外科醫師、麻醉科醫師或 EB 醫師——都需要在該外州病患所屬州完成某種形式的加入/登錄程序。因此，這是一個一次性的流程，同時也只需要提供一份費率表。

Sometimes when you have an established product, there is a fee schedule that's already in place. So you don't need additional letters of agreement or a single case agreement for those patients. So because we are navigating these initial payer processes, it takes a little additional time. But once that is secured, then it gets better over time. So that's been our experience, and that's how it's panning out.

有時候當你有一個已建立的產品時，既有的費率表已經到位。因此，對於那些病患，你不需要額外的協議函（letters of agreement）或單一個案協議（single case agreement）。所以因為我們正在處理這些初始的付款方流程，會需要多一點時間。但一旦這些都確立之後，之後就會隨時間推進而改善。這就是我們的經驗，也是目前的進展情況。

Stephen Willey, Stifel.

Stephen Willey，Stifel。

Maybe just a little bit of a follow-up. What is the average scheduling lead time for biopsies right now? Just curious how far out these procedures are being scheduled.

也許再追問一下。目前活檢的平均排程提前期（scheduling lead time）是多少？只是好奇這些手術大概會排到多遠之後。

Yes. Thank you, Steve. If you look at the time that a patient is identified as a ZEVASKYN patient and then the time that it takes for them to actually get biopsied, that is the timeline that you're talking about.

是的。謝謝你，Steve。如果你看的是病患被辨識為 ZEVASKYN 病患的時間點，到他們實際完成活檢所需的時間，這就是你所說的那段時間軸。

It's very variable. I think the factors that determine that are the type of payer, how recent the QTC is to the process. For example, now Lurie's, as you all know, has treated some patients and maybe they've gotten into rhythm and there's a lot of precedent that's been set, whereas the other sites that are just about starting, this is the first time. So it's very hard to generalize an average time because we have examples of patients where, when we activated Lurie's, I think the first patient was biopsied in August. This is pretty early.

差異非常大。我認為決定因素包括付款方類型，以及該 QTC 對於流程的熟悉程度有多新。例如，現在 Lurie’s，如各位所知，已經治療過一些病患，可能已經進入節奏並建立了很多先例；而其他剛要開始的據點，這是第一次。所以很難概括一個平均時間，因為我們有一些病患的例子：當我們啟用 Lurie’s 時，我想第一位病患是在 8 月做了活檢。這算是相當早。

It was two months or something since activation, whereas we have seen certain sites that have been active for six or seven months and they're just coming up for their first patient biopsy, preparing for that. So it's a very variable thing. And with only five to six sites, it's very hard to say this is a trend. But I think a good estimate is four to five months is what it's taking for any site that gets active to get their first patient on a biopsy schedule there. I hope that answered your question.

從啟用到活檢大概兩個月左右；但我們也看到某些據點已經啟用六、七個月，才正要迎來第一位病患的活檢、正在為此做準備。所以這是一個變異很大的事情。而且只有五到六個據點時，很難說這是一個趨勢。但我認為一個不錯的估計是：任何一個據點啟用後，要讓第一位病患排上活檢時程，大約需要四到五個月。希望有回答到你的問題。

No, it did. And then I guess the PSMA SIR-T looks conceptually pretty interesting. I think you spoke to the $7 million licensing fee. Can you speak to any additional economics that might be owed on the progression of that product?

有，有回答到。然後我想 PSMA SIR-T 在概念上看起來相當有趣。我記得你提到 700 萬美元的授權費。你能談談在該產品推進過程中，可能還需要支付的其他經濟條款嗎？

Then I know you're in the process of tech transfer now, but what are the implications for manufacturing in terms of the need to build out additional suites to potentially accommodate the clinical development of this product?

另外我知道你們現在正在進行技術移轉，但就製造端而言，為了可能支援這個產品的臨床開發，是否需要擴建額外的製造套間（suites）？其影響是什麼？

Sure. In terms of deal economics, it's the upfront payment that we shared of $7 million. And Abeona is going to develop this asset until end of Phase 1. So there's going to be dose escalation and dose expansion. And those first-in-human studies do not start until second half of 2027, as I mentioned.

當然。就交易經濟條款而言，就是我們分享過的 700 萬美元預付款。而 Abeona 將把這項資產開發到第一期結束。因此會有劑量遞增與劑量擴展。而我如先前提到，這些首次人體（first-in-human）研究要到 2027 年下半年才會開始。

There is just about $1 million of milestone payments up to that time point through the end of Phase 1. That happens with the first patient dosed and the last patient maybe. So if you look at that, the upfront payment is really the main substantial payment that's done right now.

在那個時間點之前、直到第一期結束，里程碑付款總額大約是 100 萬美元。這些會在第一位病患給藥以及最後一位病患（可能）等節點發生。所以如果你這樣看，預付款其實是目前已支付的主要、也是最實質的一筆款項。

In terms of deal structure, at the end of Phase 1 data, we have two potential paths. And one could be a 50/50 development with Angelas, so we share the cost and we share the proceeds later, or it could be an outright licensing deal where we'll have some buyouts and royalties and Abeona will fully own the program but provide royalties to Angelas. So which of these paths is going to actually prevail, it's going to be a long journey to even discovering that because the data will determine those. So it's early to comment on that.

就交易架構而言，在第一期數據出來之後，我們有兩條可能路徑。其中一條可能是與 Angelas 以 50/50 共同開發，也就是我們分攤成本、之後也分攤收益；另一條可能是直接授權交易，屆時會有一些買斷金（buyouts）與權利金（royalties），Abeona 將完全擁有該計畫，但會向 Angelas 支付權利金。至於最終會走哪一條路，還需要很長一段時間才能知道，因為數據將決定這些。所以現在評論還太早。

But in terms of cost implications, I wanted to make sure this is very well understood. So until first-in-human studies begin, there's not a big cost load on Abeona because once the upfront payment has been done, it's low single-digit millions of a CDMO developing the process.

但就成本影響而言，我想確保大家非常清楚。因此，在首次人體研究開始之前，Abeona 不會承擔很大的成本負擔，因為一旦預付款支付完成，後續主要是由 CDMO 進行製程開發，費用大約是低個位數百萬美元。

As you know, engineered T-cells, it's not as complex as ZEVASKYN, fortunately, but it's going to be mostly a cut-and-paste kind of process. We already have GMP-grade vector that has been produced. And it's a matter of locking down process. And these processes are fairly standard.

如你所知，工程化 T 細胞的複雜度不像 ZEVASKYN 那麼高，這點很幸運，但大多會是偏「剪貼」式（cut-and-paste）的流程。我們已經生產出 GMP 等級的載體（vector）。接下來就是把製程定案（locking down process）。而這些製程相當標準化。

So it's going to be done by an external CDMO, and we're not going to disturb our internal teams in Cleveland. We're laser-focused on the ZEVASKYN commercialization. So there's a very small team that's just going to drive the project out of a CDMO.

所以會由外部 CDMO 來完成，我們不會打擾克里夫蘭的內部團隊。我們會高度聚焦在 ZEVASKYN 的商業化。因此只會有一個非常小的團隊，負責在 CDMO 端推動這個專案。

And when the time comes and the regulatory team is also involved in getting clarity and alignment with the regulatory agencies on what our trial design looks like and how we go about that. So other than that, from a personnel standpoint, Abeona is laser-focused on ZEVASKYN commercialization and any significant costs will not hit us until we get into human clinical studies, which happens in the second half of 2027. I hope that gives a little bit of some color on what we are undertaking for the near term with PSMA SIR-T.

等到時機成熟，法規團隊也會參與，與監管機構釐清並對齊我們的試驗設計長什麼樣，以及我們要如何推進。除此之外，就人力角度而言，Abeona 仍會高度聚焦在 ZEVASKYN 的商業化，而任何顯著成本都要等到我們進入人體臨床研究時才會反映在我們身上，也就是 2027 年下半年。希望這能讓大家對我們近期在 PSMA SIR-T 上要做的事情有更多脈絡。

Yes. No, that's helpful. The external CDMO kind of addresses the question on the manufacturing front. Can you just say whether or not the 50-50 co-promote, I'm presuming that decision is made by Angelas based upon a review of Phase 1 data?

是的。這很有幫助。外部 CDMO 也算是回答了製造端的問題。你能否說明一下，50-50 的共同推廣（co-promote），我猜這個決定是由 Angelas 在審視第一期數據後做出，對嗎？

Correct. Yes. Actually, the option for us to pursue the program is after the Phase 1. Angelas has the option to either do the 50-50 co-development or a license agreement with what Vish had mentioned with predefined financial terms for an agreement that will be agreed upon later.

正確。是的。其實，我們在第一期之後才有選擇權來推進這個計畫。Angelas 有選擇權：要嘛做 50-50 的共同開發，要嘛簽訂授權協議，並採用 Vish 提到的、已預先定義的財務條款，至於具體協議會在之後再行敲定。

Raghuram Selvaraju, H.C. Wainwright.

Raghuram Selvaraju，H.C. Wainwright。

Congrats on the quarter and on activating the new QTCs. This is Ahmed on for Ram. I just had a few questions. One was what have been the key challenges associated with setting up additional qualified treatment centers? And how do you think those will play out in the future?

恭喜本季表現，也恭喜啟用新的 QTC。我是代替 Ram 出席的 Ahmed。我有幾個問題。第一個是，設立更多合格治療中心（QTC）所面臨的主要挑戰是什麼？你們認為未來這些挑戰會如何發展？

My second question was on the patients receiving ZEVASKYN. How often does cell harvesting from RDEB patients fail due to insufficiency? Thank you.

第二個問題是關於接受 ZEVASKYN 的病患。RDEB 病患在細胞採集時，因樣本不足而失敗的情況有多常見？謝謝。

So the first question you asked was the key challenges with activating QTC centers. I think more than challenges, I'll just say, what are all the various milestones in the journey that have to check a box.

你第一個問題是關於啟用 QTC 中心的關鍵挑戰。我覺得與其說是挑戰，不如說是在這段旅程中需要逐一達成、打勾確認的各項里程碑。

I mean this is a huge undertaking by a QTC. An EB physician has to gather a multidisciplinary team first, and they need to have anesthesiologists and plastic surgeons who are familiar with the RDEB patients and what types of care they need.

我的意思是，對 QTC 來說這是一項非常龐大的工程。EB 醫師首先必須組建一個多學科團隊，並且需要熟悉 RDEB 病患及其照護需求的麻醉科醫師與整形外科醫師。

Once such a team forms and they feel that feasibility from a center's perspective and the ability to deliver this exists, they have to make a business case for their management. That itself is a few months' journey because every buy-and-build that they have to put some financial risk on their P&L is going to be scrutinized carefully.

一旦這樣的團隊成形，且從中心角度認為具備可行性並有能力提供此項治療，他們就必須向管理層提出商業論證。這本身就需要幾個月，因為任何「買入並建置」且會讓其損益表（P&L）承擔一定財務風險的投入，都會被非常仔細地審視。

So all that is in itself a months-long process, and then we have the onboarding once that has been checked off and everybody in that QTC has agreed that they're going to go with this journey. Then you're going to have onboarding, medical onboarding as well as clinical training and quality training and all those types of events.

因此，這整套流程本身就可能長達數月；接著在上述事項都完成、且該 QTC 的所有人都同意要走這段旅程之後，我們才會開始導入（onboarding）。然後會有導入流程，包括醫療端導入，以及臨床訓練、品質訓練等各類活動。

Then there's numerous legal policies, trade policies, the master service agreements. Those are all, again, legal steps that take several months. So that's the reason why the journey of actually the first handshake with the QTC to when they're ready to treat a patient has been several months, sometimes even more than a year long. And we started that process with our first set of QTCs very early.

接著還有許多法律政策、貿易政策，以及主服務協議（master service agreements）。這些同樣都是法律程序，往往需要數個月。因此，從我們與 QTC 第一次握手到他們準備好治療病患，通常需要好幾個月，有時甚至超過一年。而我們很早就開始與第一批 QTC 啟動這個流程。

And it's what you alluded to, is there an unlimited number of QTCs that we can activate? And the answer is no because the multidisciplinary team is the key for which QTCs can actually activate, and that's something that we always carefully weigh in because that's important from a patient experience and patient care and outcome perspective.

你也提到，我們是否能啟用無限多的 QTC？答案是否定的，因為多學科團隊是 QTC 能否真正啟用的關鍵；我們一直都會非常審慎地評估這點，因為這對病患體驗、病患照護與治療結果都很重要。

And of the 23 centers where there are EB patients cared for today, a good 5 to 10 centers already have these multidisciplinary teams in place, and those are our focus areas. And as we had stated earlier, our goal was to have about seven centers active because when seven centers are active and produce at least one biopsy a month. So we want to ramp up as we ramp up our capacity as well. So those are all factors that speak to the overall QTC numbers.

在目前照護 EB 病患的 23 家中心中，大約有 5 到 10 家中心已經具備這些多學科團隊，而這些就是我們的重點。如同我們先前所說，我們的目標是啟用約七個中心，因為當七個中心啟用並且每月至少產出一次活檢。因此，我們希望在提升產能的同時也同步拉升（ramp up）。以上這些因素都會影響整體 QTC 的數量。

Anything else, Madhav?

還有其他要補充的嗎，Madhav？

Yes. I'll just add that you summed it up well, Vish. I mean just in terms of challenges, every QTC has a different risk tolerance. We observed that some institutions started right after ZEVASKYN approval. There were other institutions that wanted to wait for the actual FDA approval to happen last year before they began to invest their time and energy. Yet there were some other institutions that wanted to see reimbursement pathways established.

有。我補充一下，你總結得很好，Vish。就挑戰而言，每一家 QTC 的風險承受度都不同。我們觀察到有些機構在 ZEVASKYN 獲准後就立刻開始。也有其他機構希望等到去年 FDA 正式核准後，才開始投入時間與精力。另外還有一些機構想先看到報銷（reimbursement）途徑建立起來。

So now we are beginning to see greater engagement with the tail of these other centers, EB centers, and the traction is picking up. I mean, with the recent announcement and additional centers, as we said, we are well on track. We believe we'll be able to get another QTC also activated.

因此，我們現在開始看到這些其他中心、EB 中心的後段（tail）有更高的參與度，推進力道也在加快。我的意思是，隨著近期的公告與新增中心，如我們所說，我們進度非常符合預期。我們相信也能再啟用另一家 QTC。

The second question that you asked about patients getting the harvest. Can you please elaborate on your question? Is this the biopsy-to-delivering-the-sheet manufacturing success rate? Or was it something else that you were referring to here?

你第二個問題是關於病患進行採集（harvest）。你能否再說明一下你的問題？你指的是從活檢到製造並交付貼片（sheet）的製造成功率嗎？還是你指的是其他事項？

Yes, exactly. Basically after the biopsy, is there kind of a failure rate between the biopsy and the patient receiving the treatment?

是的，沒錯。基本上就是活檢之後，在活檢到病患實際接受治療之間，是否存在某種失敗率？

Yes. Our experience so far in the commercial setting is that every time that we have received a valid biopsy, we have been able to produce sheets, the numbers could be variable, but in majority of cases, we are actually producing a double-digit number of sheets. So we're happy with what we're seeing in terms of success rate.

是。以我們目前在商業化環境下的經驗來看，只要我們收到有效的活檢樣本，我們每次都能製作出貼片；數量可能會有差異，但在多數情況下，我們實際上能製作出兩位數數量的貼片。因此，就成功率而言，我們對目前看到的結果感到滿意。

But beyond that, I think the timing of how long it takes from skin to skin, as you know, is a variable time. It can be anywhere as early as 23 days in some cases, and it can be as lengthy as 26 days. So I think that's still a very tight window, but that's kind of our range of turnaround time we've seen so far.

除此之外，我想你也知道，從皮膚到皮膚（skin to skin）所需時間的長短仍會有所變動。有些案例最早可在 23 天完成，也可能長至 26 天。我認為這仍是一個非常緊湊的時間窗，但這就是我們目前看到的周轉時間範圍。

If I may just have one quick follow-up is, I guess, what is Abeona's plan to optimize ZEVASKYN value outside of the US?

如果可以的話，我再追問一個很快的問題：Abeona 計畫如何在美國以外的市場優化 ZEVASKYN 的價值？

Yes, that's something that's been on top of our mind. We're already looking at what are the markets that we can first supply from our Cleveland site because that is the lowest-hanging fruit in terms of timing. If you're looking at markets like Europe and Japan.

是的，這一直是我們最關注的議題之一。我們已經在評估哪些市場可以先由我們克里夫蘭（Cleveland）廠供應，因為就時程而言那是最容易先達成的。例如歐洲與日本等市場。

The logistical challenges in delivering from Cleveland, more than product delivery, could be related to bringing the biopsies of the patients and cold chain and things like that. And that's something that we're working out, but we should have such updates in the following quarterly calls.

從克里夫蘭出貨的物流挑戰，可能不僅是產品配送本身，更可能與病患活檢樣本的運送、冷鏈等相關。這些我們正在梳理中，並且我們應該會在接下來的季度電話會議中提供更新。

Right now, our teams are already spread thin in making sure that every aspect of the US launch is maximized. There's a sub-team that is looking at these external opportunities. So hopefully, in later quarterly calls, we'll give some better color to what that path looks like.

目前，我們的團隊已經相當吃緊，正全力確保美國上市的每個環節都能達到最大化。我們有一個子團隊在評估這些海外機會。因此希望在之後的季度電話會議中，我們能更清楚地說明這條路徑會是什麼樣子。

Jeff Jones, Oppenheimer.

Jeff Jones，Oppenheimer。

Congrats on a great quarter. Maybe following up on QTC activation, with six onboard and a target of seven by end of year, it seems a pretty low bar for you to get one more in by year-end. Just how are you thinking about building out additional QTCs as we look ahead into additional quarters and into next year? As you mentioned, how that aligns with capacity?

恭喜本季表現非常出色。我想延續 QTC 啟用的話題，目前已有六家完成導入，且目標是在年底前達到七家；看起來你們只要在年底前再增加一家，門檻並不高。展望未來幾季以及明年，你們如何思考擴建更多 QTC？以及如你所提到的，這如何與產能相匹配？

Then maybe on pipeline, you've deprioritized the ophthalmology programs and you brought onboard an oncology program. How are you thinking about pipeline moving forward? Are you thinking about oncology specifically? Or maybe outline for us, how you're thinking about that strategically?

那麼也許在研發管線方面，你們已降低眼科專案的優先順序，並納入了一個腫瘤學專案。你們如何看待未來的管線規劃？你們是在特別考慮腫瘤學嗎？或者能否為我們概述一下，你們在策略上是如何思考的？

Great. So first, I'll ask Madhav to respond to the QTC question.

很好。首先，我會請 Madhav 回答關於 QTC 的問題。

So Jeff, yes, I mean, we continue to work with a few more centers. Based on the knowledge we have, a total of 10 EB centers have this infrastructure that Vish alluded to earlier, cross-functional discipline of multidisciplinary teams as well as EB patients that frequent those centers.

Jeff，是的，我們確實仍在與另外幾個中心合作。根據我們掌握的資訊，總共有 10 個 EB 中心具備 Vish 先前提到的那套基礎設施，也就是由多學科團隊組成的跨職能體系，並且有經常前往這些中心就診的 EB 病患。

So we are working with these institutions and are at various stages of onboarding. I think if we get to that kind of a number, 9 or 10 centers, we are in pretty good shape because we continue to hear from centers about one patient a month being a good cadence that we can expect for these centers to treat. And if we maintain that, that would be really our steady state. So let's see, this year, next year, we should be able to get all these other centers also active.

因此我們正與這些機構合作，並處於不同階段的導入流程。我認為如果能達到那樣的數量，也就是 9 或 10 個中心，我們的狀況就相當不錯，因為我們持續從中心端聽到的回饋是：每月一位病患是這些中心可預期的良好治療節奏。如果我們能維持這樣的節奏，那就會是我們真正的穩態。所以看看今年、明年，我們應該能讓其他這些中心也都啟動運作。

Yes. And also, you asked this question, how are we building our internal capacity, we’re very diligent in building up, and we had announced six at launch and six this year, and we're already in ramp-up mode to bring it up to 10 by end of the year. So the numbers that Madhav shared in terms of QTC numbers goes hand-in-hand with how we are building our internal capacity. So we'll be able to match the demand.

是的。另外，你也問到我們如何建立內部產能；我們在擴建方面非常謹慎且積極。我們在上市時宣布了 6 個（產能/站點），今年再增加 6 個，而且我們已經在加速爬坡，目標是在年底前提升到 10 個。因此 Madhav 分享的 QTC 數量，與我們建立內部產能的方式是相互配合的。所以我們將能夠匹配需求。

And from a longer-term perspective, definitely, we have work that has progressed on getting additional suites designed and we haven't started construction yet, but a lot of the design work has already happened, and we're ready to go. So it's the right trigger, and that's not very far away. We can again speak about that in the upcoming quarterly updates.

從更長期的角度來看，我們在設計額外的作業套間（suites）方面也已取得進展；雖然尚未開始施工，但大量設計工作已經完成，我們已準備就緒。因此只要觸發條件到位，就能啟動，而且那個時間點不會太遠。我們也可以在接下來的季度更新中再談這件事。

But rest assured, we are not going to artificially restrict ourselves to seven sites. As Madhav mentioned, if there are more sites that show that multidisciplinary teams are pulled together and they have EB experience, that's an added advantage as well. So we are well on our way to get a healthy number of QTCs activated even just in 2026.

但請放心，我們不會人為地把自己限制在 7 個站點。如 Madhav 所提到的，如果有更多站點能證明其多學科團隊已到位，且具備 EB 經驗，那也會是額外的優勢。因此，即使只看 2026 年，我們也正穩步推進，將啟動相當健康數量的 QTC。

And your second question was about our move from ophthalmology to oncology. I just wanted to reiterate one thing. I think where our strengths are and where we have done well learning from the ZEVASKYN experience is really how do we develop complex biologics that have the types of profiles of long-term durable clinically meaningful benefit for patients with serious diseases.

你的第二個問題是關於我們從眼科轉向腫瘤學。我想重申一點。我認為我們的強項，以及我們從 ZEVASKYN 經驗中學得並做得很好的地方，主要在於：我們如何開發複雜的生物製劑，使其具備長期、持久且在臨床上對嚴重疾病患者具有實質意義的療效特徵。

We're not defining ourselves as a rare disease or an ophthalmology or an oncology company, but where our strength can actually, if you look at the CMC aspect of it, you will see a perfect fit. I mean, in fact, some of these engineered T-cell therapies are a little bit even more advanced and defined than the types of autologous cells we are working with. And it feels a little easier, even a little bit of a breath of air in that sense.

我們並不把自己定義為罕見疾病公司、眼科公司或腫瘤公司；但若看我們的強項，尤其從 CMC（化學、製造與管制）的角度，你會看到非常契合。事實上，其中一些工程化 T 細胞療法甚至比我們正在處理的自體細胞類型更為先進且更為明確。從這個意義上說，感覺反而更容易一些，甚至有種鬆一口氣的感覺。

But if you look at our commercial teams, we're all from the CAR-T world. We've done launches of Breyanzi and Abecma. And in fact, Dr. Preet Choudhary, with whom we have done this deal, was one of our customers when we were in the hematology CAR-T launching expedition at that time.

但如果你看我們的商業團隊，我們都來自 CAR-T 領域。我們曾推動 Breyanzi 和 Abecma 的上市。而且，與我們完成這筆交易的 Preet Choudhary 醫師，當年在我們於血液腫瘤 CAR-T 上市推進時期，曾是我們的客戶之一。

And we've continued to discuss what are the unmet needs and how do we really get breakthroughs there. And as an innovator, we've held that dialogue from those days. So you see that the strength in the oncology field really is not something that we have to start from ground zero here. And so every little angle that you're looking from, we have that.

我們也持續討論有哪些未被滿足的需求，以及我們如何真正實現突破。作為創新者，我們從那時起就一直保持這樣的對話。因此你可以看到，我們在腫瘤領域的實力並不是需要從零開始。所以從你觀察的每一個角度來看，我們都具備相應的基礎。

Of course, clinical development, we will build it over the clinical trial experience. But the move from ophthalmology to oncology was really, I would call it semi-opportunistic, but with a lot of synergies with the CMC path that we've learned and how to work with the FDA and what they expect in this kind of technology, and also knowing what the unmet needs are in the solid tumor space generally.

當然，在臨床開發方面，我們會透過臨床試驗經驗逐步建立。但從眼科轉向腫瘤學，我會稱之為半機會驅動（semi-opportunistic），同時也與我們在 CMC 路徑上所學到的經驗高度協同，包括如何與 FDA 合作、他們對這類技術的期待，以及我們也了解整體實體腫瘤領域的未滿足需求。

And prostate specifically, of course, some of us have launched products in this prostate space in our past lives. So that's also bringing us the relationship. Also the KOLs that have interacted with in ad boards even before we licensed this asset have taken a look at a lot of the data, and these are the top international six or eight KOLs who are opine and they're very eager and interested in participating in these trials, even putting their patients on this type of technology.

而在前列腺癌方面，當然，我們其中一些人在過去的職涯中曾在這個前列腺領域推動產品上市。因此這也為我們帶來了相關的人脈關係。此外，在我們授權引進這項資產之前，就已在諮詢委員會（ad boards）中互動過的 KOL 也已檢視了大量數據；這些是國際頂尖的 6 到 8 位 KOL，他們提出意見，並且非常渴望且有興趣參與這些試驗，甚至願意讓他們的病患使用這類技術。

When you have everything from a capability standpoint lining up to take us to a disease where, of course, the market potential is a log order bigger from where we are in the rare disease space, why not? And we were waiting for the right moment, which was ZAVASKYN is in a good place with its launch.

當你在能力面上各項條件都到位，能把我們帶往一個疾病領域，而其市場潛力相較於我們目前的罕見疾病領域大上好幾個數量級，那為什麼不做呢？而我們也在等待合適的時機，也就是 ZAVASKYN 的上市進展已處於良好狀態。

We're already seeing early indicators that this is taking off. And that's what we had kept this. I mean, this has been a diligence that we've been doing for quite a while. And so this was the right time. So that's really where we've shifted. This doesn't mean to say we're not putting a stake in the ground, saying we're going to be an oncology company. If our technologies, for example, CD19 as a CAR-T field, found great application beyond hematology, where we started. And now everybody who has a CD19 asset is in the autoimmune space. That is, I mean, still leveraging their strength in a completely different disease area.

我們已經看到一些早期指標顯示它正在起飛。這也是我們一直保留這項計畫的原因。我的意思是，這是一項我們已經做了相當一段時間的盡職調查。因此現在是正確的時點。這就是我們轉向的原因。但這並不表示我們要在地上插旗，宣稱我們將成為一家腫瘤公司。如果以我們的技術為例，例如 CD19 在 CAR-T 領域，最初在血液腫瘤起步，後來在血液腫瘤之外也找到了很好的應用。而現在，幾乎所有擁有 CD19 資產的人都在自體免疫領域布局。也就是說，他們仍是在完全不同的疾病領域中延伸運用其強項。

We're going to follow such a path where we have good science that takes us to solving big problems, and there is huge long-term value in that. So that's how this asset really checked all the boxes that we're describing here.

我們也會走類似的路徑：以良好的科學為基礎，去解決重大問題，而這其中蘊含巨大的長期價值。因此，這項資產確實符合我們在此所描述的所有條件。

Jim Molloy, Alliance Global Partners.

Jim Molloy，Alliance Global Partners。

Just a couple of quick questions on pricing and so gross to net. Mechanistically, looking at the revenue number you guys printed in the quarter at $3.1 million per, it looks like a much more favorable gross to net discount for you guys on the quarter. Can you talk a little bit about how you're seeing the payer mix come through on that? And is the pricing holding there?

我有幾個關於定價以及毛額到淨額（gross-to-net）的快速問題。從機制上看，你們本季公布的營收數字為 310 萬美元，這看起來你們本季的毛額到淨額折扣更為有利。你們能否談談你們觀察到的付款方組合（payer mix）在其中的表現？以及定價是否仍能維持？

And then I guess a follow-up would be on the OpEx, ex the $7 million one-timer, these are the R&D and G&A numbers we should expect, sort of going forward through the rest of '26?

然後我想追問一下關於營業費用（OpEx），扣除那筆 700 萬美元的一次性項目後，這些研發（R&D）與一般及行政（G&A）數字，是否就是我們在 2026 年剩餘期間大致可以預期的水準？

Thanks, Jim. Yes. So regarding the gross to net for Q1, all three patients treated in the quarter were commercial compared to Q4, where it was a Medicaid patient. So on the commercial patients, there are far less rebates and discounts than the government 23.1% rebate that was for the Medicaid patient. So going forward, again, when things normalize with more patients, we think the gross to net will be in the mid- to upper teens when we have more patients treated.

謝謝你，Jim。是的。關於第一季的毛額到淨額（gross to net），本季治療的三位病患全部都是商業保險，相較於第四季是 Medicaid 病患。因此在商業保險病患上，回扣與折扣遠少於針對 Medicaid 病患的政府 23.1% 回扣。所以往後看，當病患數增加、情況回歸正常時，我們認為毛額到淨額會落在十幾個百分點的中段到高段（mid- to upper teens），也就是在治療更多病患時。

And then for your second question, yes, so if you exclude the $7 million upfront payment for R&D and SG&A, the total spend will be pretty much the same for the rest of the year. Again, as we treat more patients and get more volume in there, some of the costs will come out of SG&A, the engineering runs, and they will go to cost of goods sold. But the overall run rate, again, throwing out the $7 million expense, is reflective of the rest of the year.

至於你的第二個問題，是的，如果把研發與銷售、一般及行政（SG&A）的 700 萬美元預付款排除在外，今年剩餘期間的總支出將大致維持相同。同樣地，隨著我們治療更多病患、量能增加，部分成本會從 SG&A（工程批次/工程運行）轉出，並歸入銷貨成本（cost of goods sold）。但整體的支出運行率（run rate），同樣地，扣掉那 700 萬美元費用後，就能反映今年剩餘期間的水準。

A quick follow-up, if I could, please. Any guidance on the six to seven people potentially to shoot for the second quarter, what that mix looks like on commercial versus Medicare, Medicaid?

如果可以的話，我想快速追問一下。針對第二季可能目標的 6 到 7 位病患，有沒有任何指引？在商業保險與 Medicare、Medicaid 之間的組合大概會是什麼樣子？

A similar kind of mix that we have. And overall, we can expect, based on our claims data and what we've understood of the market, about 60% commercial, about 30% to 33%, or something like that, is Medicaid. So that is the split we're looking at.

會是與我們目前相近的組合。整體而言，根據我們的理賠數據以及對市場的理解，大約 60% 是商業保險，大約 30% 到 33% 左右是 Medicaid。所以這就是我們在看的分布。

And have you guys put any guidance on when you anticipate being profitable? I know last year, you put some guidance. Obviously, things have changed since then.

另外，你們是否提供過任何關於何時預期能獲利的指引？我知道去年你們曾給過一些指引。顯然自那之後情況已經有所改變。

We maintain the assumption that we had in the last call, that we believe, depending on how these biopsies come out, that Vish and Madhav had spoken about earlier, we believe we can achieve monthly profitability starting potentially in June, so next month.

我們維持上次電話會議時的假設：我們相信，取決於 Vish 和 Madhav 先前提到的那些活檢結果如何，我們可能自 6 月起、也就是下個月開始，達成按月獲利（monthly profitability）。

David Bautz, Zacks Small-Cap Research.

David Bautz，Zacks Small-Cap Research。

Given the fact that most solid tumor CAR-T programs have struggled in the past, I'm just curious what was it specifically about 701 that gives you confidence that it could be successful?

鑑於過去多數實體腫瘤 CAR-T 計畫都曾遭遇困難，我想請教，究竟是 701 的哪些特點讓你們有信心它可能會成功？

Thanks, David, for that question. First of all, we have to underscore that CRS is not a CAR-T. The synthetic immune receptors are fundamentally differently structured.

謝謝你，David，提出這個問題。首先，我們必須強調，CRS 並不是 CAR-T。合成免疫受體（synthetic immune receptors）的結構在根本上就不同。

So a lot of the innovation in the CAR-T field has been about better signaling domains or the domains, and they're built on an existing CAR structure. I mean, it's physiologically very different from the natural TCRs that you have. And then, of course, the TCR technologies themselves have failed due to other reasons, which are to do with MSC restriction and various population-based constraints.

因此，CAR-T 領域的許多創新，都是圍繞更好的訊號傳導結構域（signaling domains）或相關結構域，並且是在既有的 CAR 架構上建構。我的意思是，它在生理上與你所擁有的天然 TCR 有很大差異。當然，TCR 技術本身也因其他原因而失敗，這些原因與 MHC 限制以及各種族群基礎的限制有關。

What the SIR-T technology does is actually take the best of both worlds. It will probably take me two days to describe all the components of the technology that make us believe that it's different. But if you look at the money slide, the preclinical data that we shared, we've used a CAR control with the same kind of binding domain, which is the receptor, which recognizes and binds to PSMA, but the rest of the structure is all like a CAR versus the SIR. And you can see that in a preclinical model in mice, you already see that difference.

SIR-T 技術實際上是取兩者之長。要我把所有讓我們相信它與眾不同的技術組成講完，可能得花我兩天時間。但如果你看我們分享的那張重點投影片（money slide）上的臨床前數據，我們使用了一個 CAR 對照組，具有相同類型的結合結構域，也就是能辨識並結合 PSMA 的受體，但其餘結構完全是 CAR 的樣子，而不是 SIR。你可以看到，在小鼠的臨床前模型中，你已經能看到那個差異。

How can you generate persistent serial killer T cells that go after a tumor-specific membrane antigen? That's what we are encouraged with. And these experiments have been repeated many, many times with variations in the manufacturing process and everything. So we're excited. Our KOL community is excited that this is a new hope. So we're not doing exactly the same thing that has been done in the past. There is true novelty structurally as well as functionally in this approach.

你如何產生具持久性、可連續殺傷（persistent serial killer）的 T 細胞，去攻擊腫瘤特異性的膜抗原？這正是讓我們受到鼓舞之處。而且這些實驗已經在製造流程等各方面做了許多、許多次的重複與變化。所以我們很興奮。我們的關鍵意見領袖（KOL）社群也很興奮，認為這是一個新的希望。因此我們並不是在做過去已經做過的完全相同的事情。這個方法在結構上與功能上都具有真正的新穎性。

So that's what really gives us -- and we have included a link that takes you to a talk by the inventor himself, and that has a lot of technical details. If you're interested, I would encourage anyone to go and listen to that. So I hope I answered your question.

所以這就是我們真正的信心來源——而且我們也附上了一個連結，會帶你去聽由發明者本人所做的一場演講，裡面有很多技術細節。如果你有興趣，我會鼓勵任何人去聽聽看。希望我有回答到你的問題。

Thank you. Ladies and gentlemen, this does conclude today's Q&A session and also today's call. You may disconnect your lines at this time, and we thank you for your participation.

謝謝。各位女士、先生，今天的問答環節以及今天的電話會議到此結束。您現在可以掛線，我們感謝各位的參與。