Capricor Therapeutics, Inc. (CAPR) 2026 Q1 法說會逐字稿

Good afternoon, ladies and gentlemen, and welcome to the Capricor Therapeutics first quarter 2026 conference call. (Operator Instructions) This call is being recorded on Tuesday, May 12, 2026.

各位女士、先生，下午好，歡迎參加 Capricor Therapeutics 2026 年第一季電話會議。(接線員指示) 本通話將於 2026 年 5 月 12 日（星期二）錄音。

I would now like to turn the conference over to our CFO, AJ Bergmann, for the forward-looking statements.Please go ahead.

現在我想把會議交給我們的財務長 AJ Bergmann 進行前瞻性陳述說明。請開始。

Thank you, and good afternoon, everyone. Before we start, I would like to state that we will be making certain forward-looking statements during today's presentation. These statements may include statements regarding, among other things, the efficacy, safety and intended utilization of our product candidates, potential regulatory developments involving our product candidates, our future R&D plans, our anticipated conduct and timing of preclinical and clinical studies, enrollment of patients in our clinical studies.

謝謝，各位下午好。在開始之前，我想先聲明，我們在今天的簡報中將會作出若干前瞻性陳述。這些陳述可能包括但不限於：我們產品候選藥物的療效、安全性與預期用途；與我們產品候選藥物相關的潛在監管發展；我們未來的研發計畫；我們預期進行的臨床前與臨床研究之執行方式與時程；以及臨床研究的病患招募情況。

Our plans to present or report additional data, our plans regarding regulatory filings, potential regulatory inspections, revenue and reimbursement estimates, projected terms of definitive agreements, manufacturing capabilities, our financial position, our possible uses of existing cash and investment resources and statements regarding our litigation with Nippon Shinyaku and NS Pharma Inc., including the nature of the dispute, our expectations regarding any legal proceedings and our ability to commercialize deramiocel independent of our existing distribution agreement.

我們提出或公布更多數據的計畫、我們關於監管申報的計畫、潛在的監管稽查、營收與給付（報銷）估計、最終協議的預期條款、製造能力、我們的財務狀況、我們現有現金與投資資源的可能用途，以及關於我們與 Nippon Shinyaku 與 NS Pharma Inc. 之訴訟的陳述，包括爭議性質、我們對任何法律程序的預期，以及我們在不依賴現有經銷協議的情況下商業化 deramiocel 的能力。

These forward-looking statements are based on our current information, assumptions, and expectations that are subject to change and involve a number of risks and uncertainties that may cause actual results to differ materially from those contained in the forward-looking statements.

這些前瞻性陳述係基於我們目前掌握的資訊、假設與預期，可能會有所變動，並涉及多項風險與不確定性，可能導致實際結果與前瞻性陳述所載內容出現重大差異。

These and other risks are described in our periodic filings with the SEC, including our quarterly and annual reports. You are cautioned not to place undue reliance on these forward-looking statements, and we disclaim any obligation to update such statements. With that, I'll turn the call over to Linda Marbán, CEO.

這些及其他風險已在我們向美國證券交易委員會（SEC）提交的定期申報文件中說明，包括我們的季度與年度報告。敬請注意勿過度依賴這些前瞻性陳述，我們亦不承擔更新此等陳述的任何義務。接下來，我把電話交給執行長 Linda Marbán。

Good afternoon, everyone, and thank you for joining Capricor's first quarter 2026 earnings call. To our investors, collaborators, the Capricor team, and especially the Duchenne patient community, thank you for your continued support and belief in our mission. We are at a truly defining moment in this company's history, and I want to take a few minutes to give you a clear picture of where we stand across our three most important priorities, our regulatory path to approval, our commercial readiness, and our legal action against NS Pharma and Nippon Shinyaku.

各位下午好，感謝各位參加 Capricor 2026 年第一季財報電話會議。致我們的投資人、合作夥伴、Capricor 團隊，尤其是杜興氏肌肉萎縮症（Duchenne）病友社群，感謝各位持續的支持與對我們使命的信念。我們正處於公司歷史上一個真正關鍵的時刻，我想花幾分鐘，清楚說明我們在三項最重要優先事項上的現況：我們通往核准的監管路徑、商業化準備度，以及我們對 NS Pharma 與 Nippon Shinyaku 採取的法律行動。

I'll start with the most significant development. Our biologics license application, or BLA, for deramiocel is currently under active review by the FDA with a PDUFA target action date of August 22, 2026.

我先從最重要的進展談起。我們針對 deramiocel 的生物製劑許可申請（BLA）目前正由 FDA 積極審查中，PDUFA 目標行動日期為 2026 年 8 月 22 日。

As a reminder, following the receipt of the complete response letter in July of 2025, we moved quickly and decisively. We submitted our response based on the HOPE-3 Phase III trial results, which we believe represent one of the most compelling data sets generated for DMD to date. The FDA accepted our response as complete and classified the resubmission as Class II, resuming its full review of the BLA.

提醒各位，在 2025 年 7 月收到完整回覆函（CRL）後，我們迅速且果斷地採取行動。我們根據 HOPE-3 第三期試驗結果提交回覆；我們相信這是迄今為止針對 DMD 所產生最具說服力的數據集之一。FDA 已接受我們的回覆為完整，並將此次重新提交分類為 Class II，恢復對 BLA 的完整審查。

There has been a significant number of information requests from the FDA to Capricor, all of which we have been able to provide answers to. We look forward to continuing our active dialogue with the FDA and are looking forward to labeling discussions in the very near future. As this is a BLA resubmission review, there will not be a mid-cycle review meeting.

FDA 向 Capricor 提出了相當多的資訊要求，我們皆已能提供回覆。我們期待持續與 FDA 進行積極對話，並預期在不久的將來展開標示（labeling）討論。由於這是 BLA 重新提交的審查，因此不會有期中審查（mid-cycle review）會議。

Now, the data submitted as a response to the CRL, the HOPE-3 data, demonstrated statistical as well as clinical significance across a variety of endpoints. To remind you, HOPE-3 was a pivotal Phase III multicenter, randomized, double-blind, placebo-controlled study that enrolled 106 patients in a 1:1 randomization scheme.

接著，作為對 CRL 回覆所提交的數據——HOPE-3 數據——在多項終點上展現統計顯著性以及臨床顯著性。提醒各位，HOPE-3 是一項關鍵性的第三期多中心、隨機、雙盲、安慰劑對照研究，共納入 106 名病患，採 1:1 隨機分派。

The trial met its primary efficacy endpoint, which was the performance of the upper limb, otherwise known as the PUL version 2.0, with an approximate 54% reduction in disease severity at 12 months and a p-value of 0.029, as well as all type 1 error-controlled secondary endpoints also hitting significance.

該試驗達成其主要療效終點，即上肢功能表現（Performance of the Upper Limb，PUL）2.0 版，在 12 個月時疾病嚴重度約降低 54%，p 值為 0.029；此外，所有經第一類錯誤控制（type 1 error-controlled）的次要終點亦皆達到顯著性。

As the current BLA was for the indication of cardiomyopathy, it was very important that the key secondary endpoint of left ventricular ejection fraction demonstrated an approximate 91% slowing of disease progression in all evaluable patients. Further, in a subset of patients with a confirmed diagnosis of cardiomyopathy, the LVEF effect showed an approximate 120% slowing of disease and achieved a p-value of 0.01, further supporting deramiocel's potential to address the DMD-associated cardiomyopathy.

由於目前的 BLA 申請適應症為心肌病變，因此關鍵次要終點——左心室射出分率（LVEF）——在所有可評估病患中顯示疾病進展約減緩 91%，這點非常重要。此外，在一個已確認診斷為心肌病變的病患子群中，LVEF 的效果顯示疾病減緩約 120%，且 p 值達 0.01，進一步支持 deramiocel 具備處理 DMD 相關心肌病變的潛力。

We are also pleased that we presented additional HOPE-3 data at the 2026 MDA, Muscular Dystrophy Association Clinical and Scientific Conference, also with the American Academy of Neurology and the American Society of Cell and Gene Therapy, being held this week.

我們也很高興在 2026 年 MDA（肌肉萎縮症協會）臨床與科學會議上發表更多 HOPE-3 數據；此外，本週也在美國神經學學會（American Academy of Neurology）以及美國細胞與基因治療學會（American Society of Cell and Gene Therapy）會議上進行發表。

Two highlights are worth emphasizing. The Duchenne Video Assessment of eat 10 bites measures a home-based caregiver's captured video assessment of upper limb function. These showed statistically significant improvement in treated patients versus placebo. This is a real-world home-based outcome measure that directly reflects independence and quality of life.

其中有兩項重點值得強調。Duchenne 影片評估（Duchenne Video Assessment）的「吃 10 口」（eat 10 bites）量表，衡量的是居家照護者所拍攝影片中對上肢功能的評估。結果顯示，治療組相較於安慰劑組有統計上顯著的改善。這是一項真實世界的居家結局指標，能直接反映獨立性與生活品質。

In addition, the cardiac MRI data demonstrated a significant stabilization in the progression of cardiac fibrosis in treated patients versus placebo. Fibrosis is cumulative and irreversible. Its attenuation before functional symptoms appear is one of the most clinically meaningful things we can demonstrate. These data will be central to labeling discussions and support the case for early treatment initiation.

此外，心臟 MRI 數據顯示，治療組相較於安慰劑組在心肌纖維化進展方面有顯著的穩定效果。纖維化是累積性且不可逆的。在功能性症狀出現之前就能減緩其惡化，是我們能展示的最具臨床意義的成果之一。這些數據將成為標示討論的核心，並支持及早啟動治療的論點。

The full HOPE-3 data set has been submitted for publication in a major peer-reviewed journal, and we look forward to that publication following acceptance in due course.

完整的 HOPE-3 數據集已提交至主要同儕審查期刊投稿，我們期待在適當時點於獲接受後發表。

On safety, we have now completed more than 800 intravenous infusions across multiple clinical studies. Deramiocel continues to demonstrate a consistent and well-tolerated safety profile. Several of our young men in the HOPE-2 open-label extension have been receiving continuous infusions for up to five years, with approximately 90 patients in our collective OLE studies. Importantly, the long-term safety profile of deramiocel is supported by these five years of open-label extension data. This is reassuring for the DMD community as well as supportive of regulatory approval.

在安全性方面，我們目前已在多項臨床研究中完成超過 800 次靜脈輸注。Deramiocel 持續展現一致且耐受性良好的安全性概況。在 HOPE-2 開放標籤延伸（OLE）研究中，我們有數位年輕男性受試者已持續接受輸注長達五年；我們整體 OLE 研究約有 90 名病患。重要的是，deramiocel 的長期安全性概況獲得這五年的開放標籤延伸數據支持。這對 DMD 社群而言令人安心，也有助於監管核准。

Lastly, we also expect to be eligible to receive a priority review voucher upon potential approval. PRVs are transferable and can be monetized through sale, representing a meaningful potential source of non-dilutive capital. We will continue to provide updates to the market on the progress toward the path to approval for deramiocel to treat DMD.

最後，我們也預期在潛在獲核准後，將符合取得優先審查憑證（PRV）的資格。PRV 可轉讓，並可透過出售變現，代表一項具意義的非稀釋性資金來源。我們將持續向市場更新 deramiocel 用於治療 DMD 的核准進程。

Now I'd like to turn your attention to directly address our legal action against Nippon Shinyaku and its US subsidiary, NS Pharma, which we announced last week on May 7, 2026. We filed suit seeking rescission of our US distribution agreement through an expedited process, requesting a preliminary injunction that would allow Capricor to distribute deramiocel to patients either on our own or through other distributors pending FDA approval.

現在我想請各位注意，我將直接說明我們對 Nippon Shinyaku 及其美國子公司 NS Pharma 所採取的法律行動；我們已於上週 2026 年 5 月 7 日對外宣布。我們已提起訴訟，尋求透過加速程序撤銷我們的美國經銷協議，並申請初步禁制令，使 Capricor 在 FDA 核准前得以自行或透過其他經銷商向病患供應 deramiocel。

Now let me be clear about what drove this decision and why we believe it is both necessary and correct. The core problem is related to pricing. The pricing structure embedded in our commercialization and distribution agreement with NS Pharma contains a fundamental flaw that if left unaddressed, would make it economically impossible to deliver deramiocel to patients covered by Medicare, Medicaid or private insurance.

現在讓我清楚說明是什麼驅動了這項決定，以及為什麼我們相信這既是必要的也是正確的。核心問題與定價有關。我們與 NS Pharma 的商業化與經銷協議中所內嵌的定價架構存在一項根本性缺陷；若不加以處理，將使得把 deramiocel 提供給由 Medicare、Medicaid 或私人保險涵蓋的病患在經濟上變得不可能。

Providers would face potentially hundreds of thousands of dollars in reimbursement shortfalls per dose. This is not a commercial preference. It is a structural barrier to patient access. We tried to fix the pricing structure, but NS Pharma refused to address the flaw on acceptable terms.

醫療服務提供者每一劑可能面臨高達數十萬美元的給付差額。這不是商業偏好問題。這是一個阻礙病患取得治療的結構性障礙。我們嘗試修正定價架構，但 NS Pharma 拒絕在可接受的條件下處理該缺陷。

After we identified this issue, we engaged in good faith negotiations with senior leaders at NS Pharma to find a resolution acceptable to both parties that NS Pharma refused to compromise and demanded that Capricor agreed to a structure that would result in Capricor seeding all control of the products we developed, our regulatory relationships and our commercial and brand identity as a condition for resolution. That arrangement was just not acceptable to us.

在我們識別出此問題後，我們本著誠意與 NS Pharma 的高階主管進行協商，試圖找到雙方都能接受的解決方案；但 NS Pharma 拒絕妥協，並要求 Capricor 同意一種架構，作為解決條件，將導致 Capricor 交出我們所開發產品的所有控制權、我們的法規監管關係，以及我們的商業與品牌識別。那樣的安排對我們而言完全無法接受。

At the point of the CRL in July of 2025, it was clear that NS Pharma failed to continue the commercial investments needed to ensure a successful launch of deramiocel upon regulatory approval. NS Pharma did not provide status updates to inform Capricor on their launch planning efforts specific to the commercial interest of deramiocel, patients, caregivers, HCPs and payers. These were core requirements of our partnership that they did not uphold.

在 2025 年 7 月收到 CRL 時，很明顯 NS Pharma 未能持續投入確保 deramiocel 在取得法規核准後能成功上市所需的商業投資。NS Pharma 未提供狀態更新，以讓 Capricor 了解其針對 deramiocel 的上市規劃進度，尤其是與 deramiocel 的商業利益、病患、照護者、醫療專業人員（HCP）及付款方相關的部分。這些都是我們合作關係的核心要求，而他們並未履行。

With the PDUFA date of August 22, 2026, approaching, we could not allow a flawed contractor and a distributor who is unwilling to fix it stand between DMD patients and deramiocel. Every month of delay for boys and young men with DMD means irreversible loss, muscle destroyed, cardiac function permanently diminished or independence that may never be returned.

隨著 2026 年 8 月 22 日的 PDUFA 日期逼近，我們不能讓一份有缺陷的合約與一個不願修正缺陷的經銷商，阻擋 DMD 病患取得 deramiocel。對於 DMD 的男孩與年輕男性而言，每延遲一個月都意味著不可逆的損失：肌肉被破壞、心臟功能永久下降，或是可能再也無法恢復的獨立生活能力。

We did not rush this decision. After exhausting every alternative to address NS Pharma is in action, we concluded that litigation was our only acceptable option. The FDA review process and the PDUFA date are unaffected by this lawsuit. And as I mentioned earlier, our BLA remains under active review.

我們並未倉促做出這項決定。在用盡所有替代方案以處理 NS Pharma 的不作為之後，我們得出結論：訴訟是我們唯一可接受的選項。FDA 的審查流程與 PDUFA 日期不受本次訴訟影響。而且如我先前提到的，我們的 BLA 仍在積極審查中。

Capricor has hired and continues to hire key individuals to support the successful commercialization of deramiocel. We have been working to ensure that our commercial readiness and a product launch plan that considers patients, health care providers and payers with a focus on seamless patient access. We are thoughtfully scaling our manufacturing capacity to meet the needs of those patients, exiting our clinical trials and in preparation for commercialization.

Capricor 已聘用並持續聘用關鍵人才，以支持 deramiocel 的成功商業化。我們一直在努力確保商業就緒度，並制定一個兼顧病患、醫療服務提供者與付款方的產品上市計畫，重點在於無縫的病患可近性。我們正審慎擴充製造產能，以滿足這些病患的需求，並在結束臨床試驗、為商業化做準備之際同步推進。

Now our regulatory pathway is only meaningful when we deliver the therapy directly to patients, and that is exactly what we are building towards with the same discipline and urgency we have applied to the science over the past two decades. Now let me walk you through some of the respect of -- specifics with respect to manufacturing.

現在，我們的法規路徑只有在我們能將療法直接交付給病患時才有意義，而這正是我們正在打造的目標；我們以過去二十年投入科學研究同樣的紀律與緊迫感來推進。接下來我將帶各位了解一些——關於製造方面的具體細節。

First, our in-house GMP manufacturing facility in San Diego successfully completed its FDA pre-license inspection or PLI, last year. All Form 483 observations were addressed. The facility is operational and positioned to support initial commercial launch for approximately 200 to 250 patients per year.

首先，我們位於聖地牙哥的自有 GMP 製造設施已於去年成功完成 FDA 的上市前查核（pre-license inspection，PLI）。所有 Form 483 的觀察事項均已完成改善。該設施已投入運作，並可支援初期商業上市，每年約 200 至 250 名病患。

Additionally, we are also well underway with our expansion to the second floor of that same facility, adding several additional clean rooms. At full capacity, this expansion will support treatment of approximately 2,000 to 2,500 patients per year, roughly 10,000 doses annually. We are staging the build-out deliberately. My goal is to have the facility fully validated and approved by the FDA in the first half of 2027. During the FDA review period, we will continue to produce material and increased capacity as we move through 2027.

此外，我們也正大幅推進同一設施二樓的擴建，新增數間潔淨室。在滿載產能下，此擴建可支援每年約 2,000 至 2,500 名病患的治療，約每年 10,000 劑。我們正有計畫地分階段進行擴建。我的目標是在 2027 年上半年讓該設施完成全面驗證並獲 FDA 核准。在 FDA 審查期間，我們將持續生產物料並在 2027 年推進過程中提升產能。

Finally, our primary manufacturing priority will begin stockpiling commercial doses once we have guidance on the label from the FDA. Since deramiocel is an ultra-cold chain product, it has to be labeled before it is frozen. FDA has been appraised of this and is working with us on a temporary label solution so that we can begin production of commercial doses. Based on current planning, we will have ample product to support our future launch, if approved.

最後，一旦我們從 FDA 獲得關於標籤（label）的指引，我們的首要製造重點將是開始囤積商業用劑量。由於 deramiocel 是超低溫冷鏈產品，必須在冷凍前完成貼標。FDA 已知悉此情況，並正與我們合作制定臨時標籤方案，以便我們能開始生產商業用劑量。依目前規劃，若獲核准，我們將有充足產品支援未來上市。

While the litigation against NS Pharma probably came as a surprise to many of you, we have become increasingly aware that not only was the deal structure impossible from an economic perspective, but they were seriously lagging behind in commercial planning, as I just mentioned. I have guided multiple times that we were building a small commercial team to provide support to NS when they were going to be the sole distributor.

雖然對 NS Pharma 提起訴訟可能讓各位多數人感到意外，但我們愈來愈清楚地意識到，不僅該交易架構在經濟角度上不可行，而且如我剛才提到的，他們在商業規劃上也嚴重落後。我曾多次說明，我們正在建立一個小型商業團隊，以在 NS 作為唯一經銷商時提供支援。

Now that we are gearing up to launch on our own, subject to the court's decision, we have accelerated those efforts and are in the process of building a fully functional commercial team. We already have a Senior Vice President of Market Access with significant rare disease experience who is actively preparing the market for deramiocel. In addition.

現在，若法院裁決允許我們自行推進上市，我們已加速這些工作，並正在建立一支功能完備的商業團隊。我們已擁有一位具備豐富罕見疾病經驗的市場准入資深副總裁，正積極為 deramiocel 進行市場準備。此外。

I am pleased to inform you that we have now secured a Chief Commercial Officer, who will join us in the next few weeks. This executive brings direct DMD and rare disease commercial experience and will be an integral member of our launch leadership team. We look forward to introducing him to the investment and DMD community shortly.

我很高興向各位報告，我們現已確定一位首席商務長（Chief Commercial Officer），他將在未來幾週加入我們。這位高階主管具備直接的 DMD 與罕見疾病商業化經驗，並將成為我們上市領導團隊的重要成員。我們期待不久後向投資人與 DMD 社群介紹他。

To that end, we are actively working towards approval and commercialization of deramiocel, and our current deliverables are to continue to work on hiring key individuals to support the successful commercialization of deramiocel to support patients and their caregivers.

為此，我們正積極推進 deramiocel 的核准與商業化，而我們目前的交付事項是持續聘用關鍵人才，以支持 deramiocel 的成功商業化，並支援病患及其照護者。

We are building to ensure commercial readiness and a product launch plan that considers all with a focus on seamless patient access across all payer segments. We are rapidly building the physician education and disease awareness infrastructure required to support responsible and rapid adoption, consistent with our expected labels in other rare disease patient populations. And lastly, the building of our patient support and distribution services is underway with a strong commitment to patients and their families while enabling access across all channels for as many eligible patients as possible. The DMD community is small, defined and engaged, and we know who the prescribers are. Now turning to our pipeline.

我們正在建置以確保商業就緒度，並制定一個兼顧各方、聚焦於在所有付款方區隔中實現無縫病患可近性的產品上市計畫。我們正快速建立醫師教育與疾病認知的基礎設施，以支援負責任且快速的採用，並與我們在其他罕見疾病病患族群中預期的標籤一致。最後，我們的病患支援與配送服務建置正在進行中；我們對病患及其家庭有堅定承諾，同時讓盡可能多的符合資格病患能透過所有通路取得治療。DMD 社群規模小、輪廓清晰且高度投入，我們也知道哪些醫師會開立處方。接下來談談我們的產品線。

Over many years, Capricor has been focused on translating the science of deramiocel into a potential commercial therapy, marshaling the majority of our resources to advance it from discovery to this pivotal stage. We have developed deep expertise in product development, manufacturing and clinical development.

多年來，Capricor 一直專注於將 deramiocel 的科學轉化為潛在的商業療法，並動員我們的大部分資源，推動其從發現階段走到這個關鍵里程碑。我們已在產品開發、製造與臨床開發方面建立深厚專業能力。

While we are proud of these accomplishments, pipeline expansion has not been our first priority. However, we have worked at a slow but steady pace in developing exosomes as therapeutic agents, taking advantage of their biological role as cellular delivery vehicles.

雖然我們為這些成就感到自豪，但擴展產品線並非我們的首要優先事項。然而，我們一直以緩慢但穩健的步伐開發外泌體作為治療載體，利用其作為細胞遞送載具的生物學角色。

We continue to explore and develop opportunities for pipeline expansion with CDC-based exosomes and our patented StealthX technology made from HEK293 cell exosomes and now being used in vaccine studies in collaboration with the National Institutes of Allergy and Infectious Disease, NIAID, for COVID prevention.

我們持續探索並開發以 CDC 為基礎的外泌體以及我們已取得專利的 StealthX 技術所帶來的產品線擴展機會；該技術源自 HEK293 細胞外泌體，目前正與美國國家過敏與傳染病研究所（NIAID）合作，用於疫苗研究以預防 COVID。

It is important to note that there has been a lot of instability in vaccine development over the past year. And so, this program has moved a bit more slowly than originally planned. However, the most important milestones for reach, which were manufacturing of clinical-grade vaccine exosomes and an approved CMC from FDA for large-scale manufacturing as well as showing that an exosome-based vaccine is safe.

需要注意的是，過去一年疫苗開發一直存在相當多的不穩定性。因此，這個計畫的進度比原先規劃稍微慢了一些。然而，就「觸及（reach）」而言，最重要的里程碑包括：製造臨床等級的疫苗外泌體、取得 FDA 核准用於大規模製造的 CMC（化學、製造與管制），以及證明以外泌體為基礎的疫苗是安全的。

We are moving away now from vaccine development at this time, and we'll focus on developing therapeutics with both classes of exosomes. We are actively presenting and publishing our rapidly expanding pipeline of exosome-based therapeutics and look forward to providing updates on the clinical development program as they become available throughout this year.

目前我們將暫時淡出疫苗開發，並把重心放在利用兩類外泌體來開發治療性產品。我們正積極發表並出版我們快速擴展的外泌體治療產品線，並期待在今年內隨著臨床開發計畫有進展時，適時提供最新資訊。

In terms of life cycle management of deramiocel, we have spent 20 years developing our therapy and now we'll be actively looking to expand into other indications. Our first target will likely be expansion to the younger DMD patients we are working on now. In addition, we plan to initiate the clinical pathway for Becker muscular dystrophy, which we plan to begin discussing with the FDA immediately following PDUFA for DMD.

就 deramiocel 的產品生命週期管理而言，我們花了 20 年開發這項療法，接下來將積極尋求擴展至其他適應症。我們的第一個目標很可能是擴展到我們目前正在推進的較年輕 DMD 病患族群。此外，我們也計畫啟動貝克型肌肉萎縮症（Becker muscular dystrophy）的臨床途徑，並打算在 DMD 的 PDUFA 之後立即與 FDA 展開討論。

Expansion of deramiocel outside the United States is also underway, and we expect to meet with the EMA and PMDA later this year. We believe deramiocel should be available to all patients worldwide, and that will be a goal that we actively work towards this year. In addition, we are evaluating opportunities in other rare neuromuscular diseases where inflammation and fibrosis are the primary pathologies.

deramiocel 在美國以外地區的擴展也正在進行中，我們預期今年稍晚將與 EMA 與 PMDA 會面。我們相信 deramiocel 應該能提供給全球所有病患，這也將是我們今年積極努力的目標。此外，我們也在評估其他罕見神經肌肉疾病的機會，這些疾病以發炎與纖維化為主要病理機制。

And now with that, I will turn the call over to AJ to review our financial results. AJ?

接下來，我將把電話會議交給 AJ，請他回顧我們的財務結果。AJ？

Thanks, Linda. As of March 31, 2026, we maintained a strong balance sheet with approximately $279 million in cash, cash equivalents and marketable securities. We believe our current capital is sufficient to fund anticipated operating expenses and capital expenditures into the fourth quarter of 2027. That expectation excludes potential product revenue or a priority review voucher monetization upon potential approval. There was no revenue recognized for the first quarter of 2026 or 2025.

謝謝，Linda。截至 2026 年 3 月 31 日，我們維持強健的資產負債表，現金、約當現金及有價證券約為 2.79 億美元。我們相信目前資本足以支應預期的營運費用與資本支出至 2027 年第四季。此一預期不包含潛在產品營收，或在潛在核准後優先審查憑證（priority review voucher）變現所帶來的資金。2026 年或 2025 年第一季皆未認列任何營收。

Our total operating expenses for the first quarter of 2026 were approximately $36.8 million compared to approximately $25 million for the first quarter of 2025. The increase was primarily driven by continued investment in clinical, regulatory and manufacturing activities as well as infrastructure expenditures supporting our Duchenne program.

2026 年第一季的總營運費用約為 3,680 萬美元，相較之下 2025 年第一季約為 2,500 萬美元。增加主要來自於持續投資臨床、法規與製造活動，以及支援我們杜興氏計畫的基礎設施支出。

Net loss for the first quarter of 2026 was approximately $33.9 million or $0.59 per share compared to a net loss of approximately $24.4 million or $0.53 per share for the first quarter of '25.

2026 年第一季淨損約為 3,390 萬美元，或每股 0.59 美元；相較之下，2025 年第一季淨損約為 2,440 萬美元，或每股 0.53 美元。

Our operating expense profile reflects a calibrated investment across our three priority areas of regulatory and clinical activities in support of our BLA, manufacturing capacity expansion and commercial infrastructure build-out, all aligned with our path towards potential approval and our balance sheet provides the runway for us to execute.

我們的營運費用結構反映出在三大優先領域上的審慎投資配置：支援 BLA 的法規與臨床活動、製造產能擴充，以及商業化基礎建置；這些都與我們邁向潛在核准的路徑一致，而我們的資產負債表也提供了足夠的資金跑道以利執行。

With that, operator, let's please open up the line for questions.

接下來，麻煩接線員，請開放提問。

(Operator Instructions)

（接線員指示）

Leland, Oppenheimer.

Oppenheimer 的 Leland。

Thank you. Linda, as we continue to digest the news on the lawsuit, wondering if you could share more color. Nippon Shinyaku, a large company with many products, but NS Pharma is a much more focused company with one product, which is for Duchenne and is complementary to deramiocel. So, they would seem to be very well set up to enter the market with your products and would be motivated to do so given the money is being made.

謝謝。Linda，隨著我們持續消化訴訟相關消息，想請你提供更多細節。Nippon Shinyaku 是一家產品眾多的大型公司，但 NS Pharma 則是一家更聚焦的公司，只有一項產品，且該產品用於杜興氏並與 deramiocel 互補。因此，他們看起來非常適合與你們的產品一起進入市場，而且在有利可圖的情況下也會有動機這麼做。

So, I'm just curious if you could share more color or insight on your discussions with NS Pharma as to their reluctance to move at all toward what could be a resolution that would enable deramiocel to be sold in such a way that the ASP-based pricing would work for the system. Again, given that NS Pharma would not be able to have any return on deramiocel until that's worked out. And a follow-up. Thank you.

所以我很好奇，你是否能就你們與 NS Pharma 的討論提供更多背景或洞見，說明他們為何遲遲不願朝向可能的解決方案前進；該解決方案將使 deramiocel 能以一種讓以 ASP 為基礎的定價在體系中可行的方式進行銷售。再說一次，因為在這件事解決之前，NS Pharma 將無法從 deramiocel 獲得任何回報。以及一個追問。謝謝。

Thank you, Leland. I think that was your longest question ever. So, I appreciate the clarity and the depth to which it's gone.

謝謝你，Leland。我想那可能是你問過最長的一個問題。所以我很感謝你表達得很清楚，也深入到相當的程度。

So, the answer is a little bit of a head scratcher for us as well. As I've been guiding since the filing of the suit on May 7, we actually entered negotiations with them regarding this very issue in March of 2025. So it's been over a year that we went back and forth and proposed a variety of solutions that we believe in several sets of lawyers, including some of the lawyers that represented NS Pharma believed would be a reasonable structure that would allow us to keep the economics essentially in place the way that they have been divided in the original agreement.

所以答案對我們來說也有點令人費解。正如我自 5 月 7 日提起訴訟以來一直所說，我們其實早在 2025 年 3 月就針對這個議題與他們展開協商。因此，我們已經來回協商超過一年，並提出多種解決方案；我們相信在多組律師的評估下（其中也包括曾代表 NS Pharma 的部分律師），這些方案會是合理的架構，能讓我們基本上維持原協議中既有的經濟分配方式。

We don't have a clear answer as to why they were so intransient on accepting any of these other opportunities. But what I can tell you is that within about one week before we filed the suit, they confirmed by e-mail that the structure that they proposed, which essentially eviscerated Capricor's brand and deramiocel and future indications was the only structure that they would accept. So, they really left us with no opportunity.

我們並不清楚他們為何如此強硬，拒絕接受任何其他選項。但我可以告訴你的是，在我們提起訴訟前大約一週，他們透過電子郵件確認，他們所提出的架構——本質上等同於徹底削弱 Capricor 的品牌、deramiocel 以及未來適應症——是他們唯一願意接受的架構。因此，他們確實讓我們別無選擇。

Now having said that, while we would never break an agreement that was being enacted upon appropriately, this gives a great opportunity for Capricor to become the distributor of deramiocel. And I think all of you know how passionate we are about deramiocel, spending more than 20 years developing it. We know the patients, we know the community, we know the doctors, we know the networks. And so, I'm excited by this opportunity as well as respectful of the terms of the original agreement, which are no longer applicable.

話雖如此，雖然我們絕不會在協議被適當履行的情況下違反協議，但這也為 Capricor 成為 deramiocel 的經銷商提供了一個很好的機會。我想各位都知道我們對 deramiocel 有多麼投入與熱情，花了超過 20 年來開發它。我們了解病患、了解社群、了解醫師、了解各種網絡。因此，我對這個機會感到興奮，同時也尊重原協議的條款——只是那些條款如今已不再適用。

In your statements, you said that Capricor determined that this would be a non-viable pricing structure. Do you have evidence or admission on NS Pharma's part or Nippon Shinyaku's part that they also did not -- they were not aware of the implications of this when they signed the agreement?

在你們的聲明中，你提到 Capricor 判定這會是一種不可行的定價架構。你是否有證據或對方的承認（無論是 NS Pharma 或 Nippon Shinyaku），顯示他們在簽署協議時也不知道——也就是說，他們並未意識到其中的影響？

Well, I'm assuming that they didn't know the implications of it because they signed the agreement. So, I think I don't know what was in their head or most people don't typically go into an agreement that is non-transactable if they know that there is sort of a killer clause in there.

嗯，我推測他們當時並不知道其影響，因為他們簽了協議。所以，我不知道他們心裡怎麼想；而且多數人通常不會在明知協議不可交易、裡面存在某種「致命條款」的情況下，還去簽署。

Having said that, we were all made aware of this problem based on the ASP at the exact same time by an independent consultant in the field. So, we know that they knew that this was a problem, as I said, starting from March of 2025. So, there's definitely not an ignorance-is-bliss kind of opportunity here.

話雖如此，我們是同一時間透過一位該領域的獨立顧問得知這個基於 ASP 的問題。因此，我們知道他們也知道這是個問題，正如我所說，從 2025 年 3 月開始就是如此。所以，這裡絕對不存在那種「不知者無罪」的空間。

All right. And lastly, in terms of your planning for commercial. So, you're getting going. You do have a few months until a presumed approval, but most companies would be well ahead of that in terms of time to market for these various preparations. Could you maybe share kind of where you expect to be assuming approval in August in terms of launch readiness relative to where you would ideally be to be launching a rare disease product to deramiocel? Thank you.

好的。最後，關於你們的商業化規劃。你們正在啟動相關工作。距離推定核准還有幾個月，但多數公司在上市準備方面通常會比這更超前，涵蓋各項上市前準備。你是否可以分享一下：假設 8 月獲得核准，你們預期在上市準備度上會處於什麼位置；相較於理想狀態下要推出 deramiocel 這類罕病產品，你們會落在什麼樣的水準？謝謝。

Yeah, so thanks, Leland. So, I think I stated pretty clearly that we were very disappointed with NS Pharma's approach to launch planning over the past few months, especially since the CRL. They had stated that they were putting pencils down. They didn't pick them back up theoretically again until March of this year, just a few months ago.

是的，所以謝謝你，Leland。所以，我想我已經相當清楚地表達，我們對 NS Pharma 在過去幾個月、尤其是在 CRL 之後的上市規劃作法感到非常失望。他們曾表示他們已經把鉛筆放下不做了。理論上直到今年三月、也就是幾個月前，他們才又把鉛筆拿起來。

They were egregiously behind in some of the very critical launch activities, which typically take place 12 to 18 months before and also pave the way whether or not you have responses from FDA. Having said that, we, at Capricor, knew launch was coming. I have been laying the groundwork quietly behind the scenes for many months now. We're building out not only a commercial team with a newly hired Chief Commercial Officer, but we've been working on market access, advocacy, planning, the patient journey, the product journey, getting a distributor, which we believe that we'll be able to secure very quickly. And so, I have high hopes for a very strong launch.

他們在一些非常關鍵的上市活動上嚴重落後；這些活動通常會在上市前 12 到 18 個月進行，而且不論你是否已收到 FDA 的回覆，都會為後續鋪路。話雖如此，我們 Capricor 知道上市即將到來。過去好幾個月我一直在幕後低調地打底。我們不僅在擴建商業團隊、並新聘了首席商務長（Chief Commercial Officer），也一直在推進市場准入、倡議、規劃、病患旅程、產品旅程、以及尋找經銷商；我們相信能很快敲定。因此，我對一次非常強勁的上市抱持高度期待。

Most importantly, we have nearly 100 open-label extension patients that we will be actively working to get over to commercial product and working with the payers to do so. And we remain committed to providing deramiocel to as many eligible patients as possible as soon as possible post-PDUFA.

最重要的是，我們有將近 100 位開放標籤延伸試驗（open-label extension）的病患，我們會積極協助他們轉換到商業化產品，並與付款方合作以完成這件事。我們也仍然承諾在 PDUFA 之後，盡快讓 deramiocel 提供給盡可能多的符合資格病患。

Thank you.

謝謝。

Ed, Piper Sandler.

Ed，Piper Sandler。

Great. Thank you very much, and I appreciate all the color on the priorities to get approval launch and then the lawsuit. I have a question just sort of what the steps are from here with respect to the lawsuit. I know you mentioned that you had requested an expedited lawsuit or I forget the exact word you used. How does that play out? And if it should go past PDUFA approval or the approval comes ahead of PDUFA, you're in a position where you're manufacturing the drug and you're putting your own sales force in place.

很好。非常感謝，也感謝你就取得核准、上市優先事項以及訴訟提供的所有補充說明。我有個問題，想請教接下來就訴訟而言的步驟大概會是什麼。我知道你提到你們已申請加速審理或我忘了你用的確切詞。那會如何進行？如果訴訟進度拖到 PDUFA 核准之後，或是核准在 PDUFA 之前就到來，你們會處在一個你們正在製造藥品、也在建立自有銷售團隊的狀態。

So, you're just going to launch it yourselves. Is that accurate?

所以，你們就會自己把它推上市。這樣理解正確嗎？

So our current plan is to launch deramiocel. Yes, we have the drug. Yes, we manufacture it, and that is what we're working towards here at Capricor. In terms of the timing, it is dependent upon the court for which we have very little -- in fact, we have no control. Having said that, the reason our lawyers filed a motion for preliminary injunction, courts typically act very quickly on those and will give us some guidance and hopefully will be heard on an expedited basis.

所以我們目前的計畫是自行推出 deramiocel。是的，我們有藥。是的，我們自行製造，而這正是我們在 Capricor 正在努力推進的方向。就時程而言，取決於法院，而我們幾乎——事實上我們完全無法控制。話雖如此，我們律師之所以提出初步禁制令（preliminary injunction）的動議，是因為法院通常會很快處理這類動議，並會給我們一些指引，也希望能以加速方式開庭審理。

We can't provide any other details on the litigation, but what we can tell you is that my plan now is to distribute deramiocel on time in a well thought out and strategic fashion while this is continuing either in the background or has been resolved.

我們無法提供訴訟的其他細節，但我們能告訴你的是：我現在的計畫是在此事仍在背景中持續進行、或已獲解決的同時，按時、以周全且具策略性的方式分銷 deramiocel。

Great. Well, I wish you all the luck because I do think this drug is best served to patients in your hands. So, thank you for that update.

很好。我祝你們一切順利，因為我確實認為這個藥由你們來推進，對病患是最好的。所以，謝謝你提供這個更新。

Thank you, Ted.

謝謝你，Ted。

Kristen, Cantor.

Kristen，Cantor。

Hi. Good afternoon, everybody. Thanks for the transparency today. So, when the press release first came out last week, I think some investors initially read it as they weren't preparing for a commercial launch appropriately because they didn't have conviction in an approval. But then on the other hand, myself and others read it as, well, if they truly believe that, they would happily just give deramiocel back to you and move on their way. I guess, can you just clarify that in no way is this, in your opinion, based on them lacking conviction in an approval?

嗨。各位下午好。謝謝你們今天的透明說明。所以，上週新聞稿剛出來時，我想有些投資人一開始解讀為：他們沒有適當準備商業化上市，是因為他們對能否獲批沒有信心。但另一方面，我自己和其他人也解讀為：嗯，如果他們真的這麼認為，他們應該會很樂意把 deramiocel 還給你們，然後就此離開。我想請你澄清一下：在你看來，這絕不是基於他們對獲批缺乏信心，對嗎？

Yeah, thanks. That's sort of icing on the cake in the sense that we were disappointed in the progress they were making. We believe that it shows a lack of belief in deramiocel from their perspective and the lack of focus on their part.

是的，謝謝。那有點像是錦上添花，因為我們對他們所取得的進展感到失望。我們認為，從他們的角度來看，這顯示他們對 deramiocel 缺乏信念，也顯示他們缺乏專注。

Having said all that, that would not have been the foundation nor the basis of an action at this point. We would have tried to work with them. And I think as I guided and have answered in a previous question, we would have shadowboxed behind them with our own commercial efforts, trying to support and help them get this to patients as quickly as possible. I think all of you know that we have great connections within the Duchenne space and relationships with patients and advocates and providers.

話雖如此，那並不會是我們在此時採取行動的基礎或根本原因。我們本來會嘗試與他們合作。而且我想如同我先前在另一個問題中所引導並回答的，我們會在他們背後以我們自己的商業化努力「影子作戰」，試著支持並協助他們，讓產品盡快到達病患手中。我想各位都知道，我們在杜興氏肌營養不良（Duchenne）領域有很強的人脈連結，並與病患、倡議者及醫療提供者維持關係。

So no, the foundation of the lawsuit is really this contractual basis, which really gives us good potential evidence for rescission, which is a decision that each party sort of went about the contract in good faith, it didn't work out and everybody goes back to square one and does their own thing, which would then allow us to have the rights back and we would be fully engaged as the sole distributor of deramiocel. But stay tuned. We don't have any answers from the court yet. Obviously, this is material, so we will provide updates as soon as they become available.

所以不是，這起訴訟的基礎其實是合約層面的依據；這讓我們有相當有力的潛在證據來主張撤銷（rescission），也就是認定雙方在簽約與履約時都是出於善意，但結果不如預期，於是大家回到原點、各自進行，這將使我們取回權利，並讓我們能以 deramiocel 的唯一分銷商身分全力投入。但請持續關注。我們目前還沒有收到法院的任何回覆。顯然這是重大事項，因此一旦有更新，我們會盡快提供。

Okay. And then maybe just on the review side, you made the comment that you're nearing potential labeling discussions. It seems a little bit quick. It's usually like a month or two before the PDUFA. I recognize we're three months out here.

好的。另外在審查方面，你提到你們接近可能的標示（labeling）討論。這看起來有點快。通常是在 PDUFA 前一到兩個月。我知道我們現在還有三個月。

So, is that just kind of based on the trajectory of the questions that you've been getting? Have they told you one way or another whether these labeling discussions are happening?

所以，這只是基於你們收到問題的走向所做的判斷嗎？他們有沒有以任何方式告訴你們，標示討論是否正在進行？

And then again, I appreciate the transparency around the information requests FDA has been giving you. Can you just provide more color? Is that on like understanding the data? Anything specific you can give us there? Thank you so.

另外，我也很感謝你們對 FDA 資訊要求的透明說明。你能否再多補充一些細節？例如是針對理解數據嗎？有沒有任何你能分享的具體內容？非常感謝。

Yeah. So, thanks, Kristen. It's always a pleasure to hear from you.

好的。謝謝你，Kristen。總是很高興聽到你的提問。

So, my regulatory lead advises us that information around labeling should be coming within the next few weeks. We are already in an interim label conversation with them because, as I mentioned in my prepared remarks, when you use an ultra-cold chain product, you have to put a label on before you freeze it. We want to start stockpiling for commercial. So, they know that this is something we're interested in and working on, and we look forward to providing updates as those labeling conversations come to fruition. In terms of the information request, they have been quite prolific coming from the CMC side.

所以，我們的法規主管告訴我們，關於標示的資訊應該會在接下來幾週內出來。我們已經在與他們進行一個臨時的標示對話，因為如我在事先準備的發言中提到的，當你使用超低溫冷鏈產品時，你必須在冷凍之前就把標籤貼上。我們希望開始為商業化備貨。因此，他們知道這是我們關注並正在推進的事項；我們也期待在標示討論有具體進展時提供更新。至於資訊要求，從 CMC 端提出的相當多。

Most of them sort of standard form types of things, asking for extra data or asking for extra clarification. On the clinical side and the statistical side as well, additional data clarification, when analyses were done, blinding protocols, that kind of information that you would expect FDA to be digging into at this time. So, we remain very optimistic about the review process for deramiocel.

其中多數算是標準格式類型的事項，例如要求補充額外數據或進一步釐清。在臨床端與統計端也有，像是補充數據釐清、分析是在何時完成、盲法（blinding）流程等，這些都是你會預期 FDA 在這個時間點會深入追問的資訊。因此，我們對 deramiocel 的審查流程仍然非常樂觀。

Thanks, Linda. All the best.

謝謝你，Linda。祝一切順利。

Thanks, Kristen. Take care.

謝謝你，Kristen。保重。

Madison, B. Riley Securities.

Madison，B. Riley Securities。

Hi, thanks for taking our question. Maybe a couple from us. Maybe could you help us understand any commercial readiness activities that you have either started or plan to start rather that were previously being conducted by your partner? For example, payer discussions, has Capricor engaged any third-party logistics distributors, things like that? And then maybe a follow-up afterwards.

嗨，謝謝讓我們提問。我們這邊可能有兩個問題。你們能否協助我們了解，任何你們已經開始或計畫開始的商業化就緒活動（而不是先前由你們的合作夥伴在進行的）有哪些？例如，與付款方（payer）的討論、Capricor 是否已接洽任何第三方物流（3PL）或經銷商之類的？之後可能還有一個追問。

Yeah, Madison, absolutely. Great to hear from you.

是的，Madison，當然。很高興聽到你的聲音。

Yes. So, as I mentioned, because we were concerned about the speed and efficiency at which NS was going about commercial planning, we were shadow boxing behind them the whole time. So yes, we have a 3PL identified. We could enter into an independent contract with them and that we're in the process of doing that at this time. Most of the other parameters of launch preparation were being either birddogged by Capricor or, in fact, led by Capricor.

是的。如我提到的，因為我們擔心 NS 在商業化規劃上的速度與效率，所以我們一直在他們背後同步跟進、做準備。所以是的，我們已經確定了一家 3PL。我們可以與他們簽訂獨立合約，而我們目前也正在進行這件事。上市準備的其他大多數要素，不是由 Capricor 在旁緊盯，就是其實由 Capricor 主導。

So, there's not really any areas right now that we've identified where NS had really taken the lead and we were sort of running behind or need to play some catch-up ball. My general hunch is that we're up to speed and moving as quickly as possible with PDUFA coming right up.

因此，目前我們並沒有發現任何領域是 NS 真正主導、而我們落後需要追趕的。我整體的直覺是，我們已經跟上進度，且在 PDUFA 日期迫近之際正以最快速度推進。

Got it. Understood. And then on the, I guess, internal label related to the cold storage. So, if you're able to manufacture around 250 doses per year currently, do you have a sense of how many you could complete between finalizing that interim label and the actual PDUFA date?

了解。明白。另外關於我想是內部標籤（label）與冷鏈儲存相關的部分。如果你們目前每年大約能製造 250 劑，你們是否能估算在完成那個臨時標籤到實際 PDUFA 日期之間，可以完成多少？

Yes, we haven't disclosed that number. We are actively working now starting to prepare for commercial manufacturing. The lag in time will be that each lot has to be reviewed and approved by FDA, and we expect to do that prior to and getting ready for launch.

是的，我們尚未披露那個數字。我們現在正積極開始為商業化製造做準備。時間上的落差在於，每一批（lot）都必須由 FDA 審查並核准，而我們預期會在上市前完成並為上市做好準備。

And so, what's most important is we will not delay dosing on any level of our open-label extension patients, and we will continue to make sure that we provide access to them and have no plans on delaying baseline commercial manufacturing. We're up to speed there. Just one quick correction. It's 200 to 250 patients who each received four doses. So, it will be 1,000 doses per year from our downstairs facility here in San Diego.

因此，最重要的是，我們不會在任何層面延遲開放標籤延伸試驗（open-label extension）的病患用藥，我們會持續確保他們能取得治療，也沒有計畫延後基礎的商業化製造。我們在這方面進度是跟得上的。另外快速更正一下。是 200 到 250 位病患，每位各接受四劑。所以，我們在聖地牙哥這裡樓下的設施每年將可生產 1,000 劑。

Got it. Thanks for that clarification.

了解。謝謝你澄清。

You're welcome.

不客氣。

Michael, Maxim Group.

Michael，Maxim Group。

Hey there. Thank you so much for taking my questions today. And I mean lots of interesting stuff going on here. I would like to see if you could just provide a bit more color on what the basis for a suspension or an injunction of the commercial agreement would be? And then if there's any sort of precedent for a court issuing an injunction to allow the manufacturer to distribute the drug while the IP or the some sort of licensing agreement is under dispute.

嗨。非常感謝今天回答我的問題。這裡確實有很多有趣的事情在進行。我想請你們再多提供一些細節：商業協議被暫停或被法院下達禁制令（injunction）的依據會是什麼？另外，是否有任何先例是法院在智慧財產權（IP）或某種授權協議仍在爭議中時，仍核發禁制令以允許製造商分銷該藥物？

Yeah, thanks. Those are really clear questions. I'm going to take the second one, and I might ask you to repeat the first one because the second one was kind of intense.

好的，謝謝。這些問題很清楚。我先回答第二個，然後我可能要請你再重複第一個，因為第二個有點強度。

So yes, so there are precedent cases of precision, not exactly similarly where you have a product, a drug product that is allowed to be manufactured and distributed while the other one is not able to. Part of the reason for that is the structure that NS proposed to us has literally never been done in US biopharma. It's only been done in generics once the drugs are off patent. So, a patented product has never been in this type of agreement that NS had proposed.

所以是的，確實有一些先例案例，雖然不完全相同，但大意是：在某些情況下，一個產品（藥品）可以被允許製造與分銷，而另一方則不能。其中一部分原因是，NS 向我們提出的架構在美國生物製藥領域從未真正做過。這種做法只在學名藥領域、且藥品專利到期後才做過一次。因此，具專利保護的產品從未以 NS 所提議的這種協議形式存在。

Our current plan is to continue to wait to hear the results of the injunction.

我們目前的計畫是繼續等待禁制令結果出爐。

Typically, in situations like this where the drug is potentially life-saving, life extending or certainly disease modifying, the lawyers think that the courts pretty much would not stop the distribution of the drug because of the need by patients, and we provided up to five affidavits with our lawsuit from patients and physicians stating that deramiocel should be available to patients regardless of who distributes it because of its importance to patients. So, we don't anticipate being shut down on that level, and we look forward to the results of the preliminary injunction and also to potential decision. That was your second question.

通常在這類情況下，如果該藥物可能是救命、延長生命，或至少能改變疾病進程，律師認為法院基本上不會因為這類爭議而停止藥物分銷，因為病患有迫切需求；而我們在訴訟中也提交了多達五份由病患與醫師提供的宣誓書（affidavits），表示 deramiocel 應該提供給病患，不論由誰來分銷，因為它對病患非常重要。因此，我們不預期會在那個層面被迫停擺；我們也期待初步禁制令（preliminary injunction）的結果，以及後續可能的裁決。這是你第二個問題的回答。

What was your first one again, Michael? I'm sorry, I forgot.

Michael，你第一個問題是什麼來著？抱歉，我忘了。

I'm not sure if you could hear me. I think my connection might be a little bit bad here. But I just -- I think you actually answered the first part of that question with the second part of the question. So, I think we're clear on that.

我不確定你是否聽得到我。我這邊連線可能有點不太好。不過我只是——我想你其實用第二個問題的回答也涵蓋了第一部分。所以我想我們這邊已經清楚了。

Okay. Well, I'll take it as a two-for-one special.

好的。那我就當作一次回答兩題的特惠。

Thank you so much.

非常感謝。

Catherine, Jones.

Catherine，Jones。

Hi, I guess -- can you expand on the definition of the private label distributor? What would that entail for Capricor, whether this would, in your view, constitute a breach of contract on their part?

嗨，我想——你能否進一步說明「自有品牌分銷商」（private label distributor）的定義？這對 Capricor 會意味著什麼？以及在你看來，這是否會構成他們違約？

So, we don't have a private label distributor agreement in place. We never did. The agreement that we have in place was a sales, marketing and distribution agreement. And the foundational aspect of it, which has led to this lawsuit is the transfer price that was originally built into the deal where NS Pharma would give Capricor a portion and just a portion, by the way, of the COGS in order to defray expenses and then NS pharma would take X number of doses, distribute them and then give us back our share of the royalties minus that small transfer price.

所以，我們並沒有任何自有品牌分銷商（PLD）協議。我們從來沒有。我們現有的協議是一份銷售、行銷與分銷協議。而其根本、也導致這起訴訟的關鍵在於交易中原先設計的移轉價格（transfer price）：NS Pharma 會支付給 Capricor 一部分——而且只是 COGS（銷貨成本）的一小部分——用以分攤費用，然後 NS Pharma 會取得 X 數量的劑量、進行分銷，接著再把我們應得的權利金（royalties）份額返還給我們，並扣除那個小額移轉價格。

So pretty clear opportunity, seem like a rational deal structure at the time, except this transfer price would then set the average sale price, which would be disastrous, as I've said many times. So, there is no PLD relationship or agreement in place, and therefore, there's no breach. The problem is that we can't agree on the structure. They want a PLD and we would have liked a co-commercial agreement.

所以當時看起來是一個相當明確的機會、也像是合理的交易架構，但問題在於這個移轉價格會設定平均銷售價格，而那將會是災難性的——我已經多次提過。因此，目前並不存在任何 PLD 關係或協議，也就談不上違約。真正的問題是我們無法就架構達成一致。他們想要 PLD，而我們原本希望的是共同商業化（co-commercial）協議。

Okay. Got it. And then I recall there were some milestone payments that they were or owed to you on approval. If they're unwilling to walk away from the contract, are they still required to pay those milestones? What happens if they choose not to if the lawsuit is going on?

好的。了解。另外我記得在核准時他們有一些里程碑款項需要支付給你們或是欠你們的。如果他們不願意退出合約，他們是否仍必須支付那些里程碑款？如果訴訟正在進行中而他們選擇不支付，會發生什麼事？

So. Well, and I'm not a lawyer. So, I will say that please take my answer not from the perspective of a lawyer, but from a CEO guiding the company through this very challenging but exciting time. And so, what we have been made to understand from the lawyers is that the agreement as it stands is non-transactable. That means everything in the agreement is, at this point, not able to be acted upon. So, we are assuming that they're not going to be paying the milestones.

所以。嗯，而且我不是律師。因此，我要說，請不要從律師的角度來看我的回答，而是從一位在這個非常艱難但也令人振奮的時期帶領公司前進的執行長角度來看。因此，根據律師讓我們理解的內容，目前這份協議在現狀下是不可執行、不可交易的。這表示協議中的所有事項，在此刻都無法付諸行動。所以，我們假設他們不會支付里程碑款。

We're not expecting them to pay the milestones.

我們不預期他們會支付里程碑款。

And in fact, one of the things that we have actively considered is if there is rescission, we will probably pay them back the $50 million that they've paid us in initial upfront payments and then the two milestone payments. So, we're ready to do that. Happy to do that, actually, if that becomes necessary, and we're not expecting any future payments from them at this time.

事實上，我們積極考量的一件事是：如果發生撤銷（rescission），我們很可能會把他們已支付給我們的5,000萬美元初始預付款，以及之後的兩筆里程碑款退還給他們。所以，我們已準備好這麼做。如果有必要的話，我們其實也樂於這麼做，而且目前我們不預期他們會再有任何未來付款。

Got it. Thanks.

了解。謝謝。

Matthew, AGP Global Partners.

Matthew，AGP Global Partners。

Hey, guys. Thank you for taking my questions. So, first, is $60 million kind of a fair estimate of what would need to be paid back to NS Pharma in the event that both of you guys just kind of shake hands, as you put it, divorce and walk away? Or would it maybe be a little bit less? I know they've already paid you the $50 million. So, the color on that would be great.

嗨，各位。謝謝你們回答我的問題。首先，如果你們雙方如你所說「握手」分道揚鑣、解除合作並各走各路，那麼需要退還給NS Pharma的金額，用6,000萬美元來估算算是合理嗎？還是可能會少一點？我知道他們已經付了你們5,000萬美元。如果能就此提供一些說明就太好了。

We're expecting that if we have to pay something back, it would be the $50 million. But we don't know. We don't know what agreement we're going to come to. We don't know what the court is going to say. We stand ready to do so, certainly if that becomes part of the ruling in the case.

我們預期如果必須退還款項，金額會是那5,000萬美元。但我們不確定。我們不知道最終會達成什麼協議。我們也不知道法院會怎麼說。如果案件裁決的一部分要求我們這麼做，我們當然已準備好退還。

Understood. And then just pivoting a little bit to the opportunity in Becker. If you could give us just an idea of the market size, what your work has already been in reaching out, maybe to patients or providers for Becker, and the sevasemten, it's in Phase III clinical studies right now for Becker. Would deramiocel be an additive onto this potentially approvable drug? And has that been something that you guys have been looking into in Becker?

了解。接著稍微轉到Becker的機會。能否請你們大概談一下市場規模、你們目前在接觸Becker患者或醫療提供者方面已做了哪些工作？另外，sevasemten目前正在針對Becker進行第三期臨床研究。deramiocel是否可能作為一種加成療法，搭配這個潛在可獲核准的藥物？你們是否也在Becker領域評估過這點？

Yeah, so we're very much looking forward to the opportunity in Becker muscular dystrophy. I've been kind of eyeballing it for a bit. The pathophysiology of Becker is literally identical to DMD. It's just a slower progressor.

是的，我們非常期待Becker肌肉失養症的機會。我已經觀察這個領域一段時間了。Becker的病理生理機轉與DMD基本上完全相同。只是疾病進展較慢。

The primary cause of death in those men with Becker is cardiomyopathy. And our cardiology key opinion leaders tell us that if you give them an MRI of a Becker patient and of a Duchenne patient, you can't tell them apart. So, we have a drug that treats the cardio as well as the skeletal muscle myopathy, and therefore, think it would be ideal for Becker.

Becker男性患者的主要死因是心肌病變。而我們的心臟科關鍵意見領袖告訴我們，如果給他們一張Becker患者與Duchenne患者的MRI，他們是分不出差別的。因此，我們有一個同時治療心臟以及骨骼肌肌病變的藥物，所以我們認為它非常適合用於Becker。

The market size in the US is about 5,000 patients per year. So, I think it's considered an ultra-rare group, and it's a very slow progressor. So, I have some idea of what I'd like to go after with the FDA. One of my first goals post-PDUFA will be to meet with them on Becker.

美國的市場規模大約是每年5,000名患者。因此我認為這被視為超罕見族群，而且疾病進展非常緩慢。所以我對於想向FDA爭取什麼已有一些想法。在PDUFA之後，我的首要目標之一就是與他們就Becker進行會談。

The reason that I'm waiting until after PDUFA is strategic because we qualify for a PRV and for a PRV, a priority review voucher, you have to be treating solely a pediatric condition. So, I don't want to sort of cloud it with an adult-based disease until we have clarity on our PDUFA for Duchenne and get our PRV and are able to use that to provide a source of non-dilutive capital to the company.

我之所以等到PDUFA之後，是出於策略考量，因為我們符合PRV資格；而要取得PRV（優先審查憑證，priority review voucher），你必須僅治療一個兒科疾病。所以在我們針對Duchenne的PDUFA有明確結果、取得PRV，並能利用它為公司帶來非稀釋性資金來源之前，我不想因為成人疾病而讓情況變得複雜。

Great. And just on the sevasemten question, very quickly. Is that something you guys are looking at? Or is that part of the plan right now?

很好。再很快追問一下sevasemten的問題。這是你們正在評估的方向嗎？或這目前是計畫的一部分嗎？

So any -- yeah, we feel like deramiocel is additive to literally any of the other products that are on the market right now or could be on the market in the clinical evaluation stage, given its activity, bioactivity, and efficacy in the heart. And that's in patients, by the way, with a diagnosed cardiomyopathy, not just healthy hearts.

所以任何——是的，我們認為deramiocel幾乎可以與目前市面上任何其他產品，或任何仍在臨床評估階段、未來可能上市的產品形成加成效果，因為它在心臟方面的活性、生物活性與療效。順帶一提，這是在已被診斷為心肌病變的患者身上，而不只是健康心臟。

And then, in addition to that, the ability to control inflammation and fibrosis can only help those other therapies be more effective. So, we look forward to partnering with the other drugs that will become part of the polypharmacy to treat DMD and, of course, Becker in the future.

此外，控制發炎與纖維化的能力，只會讓其他療法更有效。因此，我們期待與其他藥物合作，這些藥物將成為治療DMD的多重用藥（polypharmacy）的一部分，當然未來也包括Becker。

Great. Thank you guys for taking my questions, and best of luck going forward.

很好。謝謝你們回答我的問題，也祝你們接下來一切順利。

Thanks, Matt, and have a good day.

謝謝，Matt，祝你今天愉快。

Thank you.

謝謝。

Boobalan, Roth Capital Partners.

Boobalan，Roth Capital Partners。

Hi, good afternoon and thank you for taking my questions. So maybe a couple of us. Firstly, I wanted to start or maybe delve a little deeper between MSP and ASP. So, maybe can you provide some context in terms of what the difference is between average selling price and the maximum sale price for a drug like deramiocel or maybe for rare disease drugs in general?

嗨，午安，謝謝你們回答我的問題。我可能有幾個問題。首先，我想從MSP與ASP之間開始，或再深入一點。能否提供一些背景，說明像deramiocel這類藥物，或一般罕見疾病藥物的平均銷售價格（ASP）與最高銷售價格（MSP）之間有何差異？

Yeah, we haven't really been disclosing any of that information yet. We're still working on gathering that information. As I said, we are working actively now to build our commercial planning strategies. And so please stay tuned for more information on ASP, MSP, that kind of thing, as it becomes available.

是的，我們目前還沒有真正揭露這方面的任何資訊。我們仍在蒐集相關資訊。如我所說，我們正在積極建立商業化規劃策略。因此，關於ASP、MSP之類的資訊，待可提供時請持續關注。

All right. And then I think somewhere in your press release, it states that NS Pharma demanded that Capricor give up the control of regulatory relationships. So, my understanding is that NS Pharma was brought in to sell, market, or distribute deramiocel in the hospital setting. So why are they heavy-handed on the regulatory relationship? Because it looks like -- I mean, I don't know whether they just woke up one day and decided to meddle in regulatory aspects of deramiocel as well.

好。另外，我想你們在某處新聞稿中提到，NS Pharma要求Capricor放棄對法規監管關係的控制權。我的理解是，NS Pharma被引入是為了在醫院端銷售、行銷或經銷deramiocel。那為什麼他們會在法規監管關係上如此強勢？因為看起來——我的意思是，我不知道他們是不是某天突然醒來，就決定也要插手deramiocel的法規監管面向。

So maybe any color on that?

所以這部分能否提供一些說明？

Yes. So, it was really a situation where -- AJ can take this question.

可以。所以這其實是一種情況——AJ可以回答這個問題。

Yeah, I think they were focused, thanks, Boobalan, on with respect to the PLD that they were looking to propose for us, some of the regulatory relationships would fall under the guise of that potential agreement, which, of course, we're not comfortable with. So that's kind of the root of your question, I believe. And so that's fundamentally one of the reasons why, of course, it didn't work for us.

是的，我想他們的重點是——謝謝你，Boobalan——就他們打算向我們提出的PLD而言，部分法規監管關係會被納入那個潛在協議的範疇之下，而這當然是我們無法接受的。所以我相信這就是你問題的根源。因此，這從根本上也是為什麼那樣的安排對我們行不通的原因之一。

And then maybe one last. I think somewhere in the 8-K, it says that although the distribution agreement is no longer binding, blah, blah, blah, then the distributor may interfere with Capricor's relationship with third parties. So, I just feel that this, it appears NS Pharma has been in the driver's seat towards the process, and Capricor is playing a defensive game, even though Capricor is the innovator of the drug. So curious why, if the distribution agreement is no longer binding, what is the concern of interference coming from?

然後也許最後一個問題。我想在 8-K 的某處有提到，雖然該分銷協議已不再具有約束力，諸如此類，但分銷商仍可能干擾 Capricor 與第三方的關係。所以我只是覺得，看起來 NS Pharma 在這個流程中一直處於主導位置，而 Capricor 在打防守戰，儘管 Capricor 才是這款藥物的創新者。所以我很好奇，若分銷協議已不再具有約束力，那麼對於可能來自干擾的擔憂是什麼？

Yeah, first of all, we don't feel like we're on the defensive. We feel like we're equal-weighted partners who are actively interested in commercializing deramiocel together, each of us playing to our individual strengths. Unfortunately, that relationship is no longer possible based on the existing agreement, and we're prepared now to launch on our own.

是的，首先，我們不覺得自己是在防守。我們覺得我們是權重相當的合作夥伴，雙方都積極有興趣一起推動 deramiocel 的商業化，各自發揮自身的優勢。不幸的是，基於現有協議，這種合作關係已不再可能，而我們現在已準備好自行上市。

In terms of interaction with vendors, of course, in an 8-K, it's incumbent upon the company to put forth all of the potential risk factors. Boots on the ground, we haven't really had any problems with any vendors. In fact, they like working with us. We're efficient, we're smart. We're well-informed and strategic.

就與供應商的互動而言，當然，在 8-K 中，公司有責任提出所有潛在的風險因素。就實際執行情況來看，我們確實沒有遇到任何供應商方面的問題。事實上，他們喜歡與我們合作。我們效率高、很聰明。我們資訊充分、且具策略性。

So, in practice, we're not seeing any problems with vendors. In theory, if one occurred, we would just find a different vendor to work with. There are lots of different vendors out there to take advantage of for commercializing products.

所以在實務上，我們沒有看到任何供應商方面的問題。理論上，如果真的發生，我們就會找另一家供應商合作。市面上有很多不同的供應商可供選擇，用於產品商業化。

All right. Thank you.

好的。謝謝。

Have a great day.

祝你有美好的一天。

Michael, Maxim Group.

Michael，Maxim Group。

Hey there. Sorry, I got bumped off there before. I just had one follow-up I wanted to ask. You seem to be making great progress on prepping the commercial force, but there's a pretty quick time window that you have before the PDUFA date.

你好。抱歉，我剛才被斷線了。我還有一個追問想請教。你們在籌備商業團隊方面似乎進展很大，但在 PDUFA 日期之前你們的時間窗口相當短。

So, my question is, first, are you expecting that you will be able to hit the ground running, assuming this gets approved right on time and launches shortly thereafter? And then what gives you confidence that you will be able to build a sales force capable of addressing this market?

所以我的問題是，第一，你們是否預期在假設如期獲批、並在其後不久上市的情況下，能夠立刻展開行動？第二，什麼讓你們有信心能建立一支足以覆蓋這個市場的銷售團隊？

I'll take the second part first. The sales force becomes a relatively easy question to answer. In a rare disease, especially like Duchenne, with a very connected set of patients, physicians, and providers, it really becomes less about getting a big sales force, and we're not going after adult heart disease. But what we are going after is efficiency. And so, I have a lot of confidence that we'll be able to build a good sales force.

我先回答第二部分。銷售團隊其實相對容易回答。在罕見疾病領域，尤其像杜興氏這樣，病患、醫師與照護提供者之間的連結非常緊密，重點其實不在於建立一支龐大的銷售隊伍，而且我們也不是在做成人心臟病市場。我們追求的是效率。因此，我非常有信心我們能建立一支優秀的銷售團隊。

We have a good reputation in the community and get it out there.

我們在社群中有良好的聲譽，也能把訊息傳遞出去。

In terms of the distribution of deramiocel, we are the ones who have been getting ready to do so. We expect that we will launch in a time-efficient manner. We hadn't announced a date for launch. And by the way, neither had been. So, we're not gearing or guiding towards an actual launch date as yet.

就 deramiocel 的分銷而言，一直是我們在做準備。我們預期能以高效率的方式上市。我們尚未宣布上市日期。順帶一提，對方也沒有。因此，我們目前還沒有針對實際上市日期進行部署或提供指引。

We're working towards PDUFA. We're guaranteeing drug in an uninterrupted format to the open-label extension patients and perhaps other patients as the information becomes available, and we'll update the Street on our potential launch date as we get some clarity there.

我們正朝著 PDUFA 推進。我們將以不中斷的方式確保藥物供應給開放標籤延伸試驗的病患，並且隨著資訊逐步明朗，可能也會提供給其他病患；一旦我們對潛在上市日期有更清楚的掌握，我們會向市場更新。

All right. Thank you very much and best of luck. I think it's abundantly clear to anyone who's seen the data just how important getting access to this as quickly as possible.

好的。非常感謝，祝你們好運。我想對任何看過數據的人來說都非常清楚，讓大家盡快取得這項治療有多重要。

Thank you so much, Michael. That means a lot. We feel the same way.

非常感謝你，Michael。這對我們意義重大。我們也有同樣的感受。

Thank you. I will now turn the call back to Capricor management for final thoughts.

謝謝。我現在把電話交回 Capricor 管理層作最後結語。

Yeah, thank you again for joining us today and for your continued support.

好的，再次感謝各位今天加入我們，也感謝各位持續的支持。

Again, for the boys and young men living with Duchenne and their families who have waited alongside them, we are eagerly anticipating the approval of daramiocel to treat DMD. The science is strong. The data are strong and before the FDA, and we are wholly focused on ensuring that when approved, deramiocel will be made available to every eligible patient as quickly and as broadly as possible.

再次強調，對於與杜興氏共存的男孩與年輕男性，以及一路陪伴等待的家人，我們正熱切期待 daramiocel 獲批用於治療 DMD。科學基礎扎實。數據也很強，且已提交 FDA 審查；我們全力以赴確保一旦獲批，deramiocel 能以最快、最廣的方式提供給每一位符合資格的病患。

For our investors, the thesis is straightforward: a potential first-in-class approval in defined rare disease populations, proprietary in-house manufacturing, a growing pipeline, and a leadership team with the conviction and capital to execute. We believe that we are building something that will create meaningful value for patients, for this company, and for shareholders who have believed in this mission. We look forward to a transformational summer. Thank you. You may now disconnect.

對投資人而言，我們的投資論點很直接：在明確界定的罕見疾病族群中，具備潛在同類首創（first-in-class）的核准機會；自有專有的內部製造能力；持續成長的產品線；以及一支具備信念與資本、能夠落實執行的領導團隊。我們相信，我們正在打造的事業將為病患、為公司，以及為相信這項使命的股東創造有意義的價值。我們期待一個具轉型意義的夏天。謝謝。您現在可以掛線。

Ladies and gentlemen, this concludes today's conference call. Thank you for participating. You may now disconnect.

各位女士先生，今天的電話會議到此結束。感謝各位參與。您現在可以掛線。