Capricor Therapeutics Inc (CAPR) 2025 Q1 法說會逐字稿

Good afternoon ladies and gentlemen and welcome to the Capricor Therapeutics first quarter 2025 earnings call. (Operator Instructions). I would now like to turn the conference over to our CFO, AJ Bergmann, for the forward-looking statement. Please go ahead.

女士們、先生們下午好，歡迎參加 Capricor Therapeutics 2025 年第一季財報電話會議。（操作員指令）。現在，我想將會議交給我們的財務長 AJ Bergmann 來發表前瞻性聲明。請繼續。

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, our future R&D plans, including our anticipated conduct and timing of pre-clinical and clinical studies, our enrollment of patients in clinical studies, our plans to present or report additional data, our plans regarding regulatory filings, potential regulatory development.

謝謝大家，下午好。在我們開始之前，我想聲明一下，我們將在今天的演講中做出一些前瞻性的陳述。這些聲明可能包括關於我們候選產品的功效、安全性和預期用途、我們未來的研發計劃（包括我們預期進行的臨床前和臨床研究及其時間表）、我們在臨床研究中招募的患者、我們展示或報告額外數據的計劃、我們關於監管備案的計劃、潛在的監管發展等。

Involving our product candidates, potential regulatory inspections, revenue and reimbursement estimates, projected terms of definitive agreements, manufacturing capabilities, potential milestone payments, our financial position, and our possible uses of existing cash and investment resources. These forward-looking statements are based on 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.

涉及我們的產品候選、潛在的監管檢查、收入和報銷估計、最終協議的預計條款、製造能力、潛在的里程碑付款、我們的財務狀況以及我們對現有現金和投資資源的可能用途。這些前瞻性陳述是基於當前資訊、假設和預期，這些資訊、假設和預期可能會發生變化，並涉及許多風險和不確定性，可能導致實際結果與前瞻性陳述中的結果有重大差異。

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

我們向聯邦通訊委員會 (FCC) 提交的定期文件中描述了這些風險和其他風險，包括我們的季度和年度報告。我們提醒不要過度依賴這些前瞻性陳述。我們不承擔更新此類聲明的任何義務。說完這些，我將把電話轉給執行長琳達馬班 (Linda Marban)。

Thanks, AJ. Good afternoon, everyone, and thank you for joining today's first quarter conference call. Today I will review the latest updates on the progress of our BLA seeking full approval for deramiocel and treating Duchenne muscular dystrophy, cardiomyopathy, as well as a brief update on our pipeline programs. While we understand that there have been some changes at the FDA, we remain confident that the strength of our data, which I will highlight in a few minutes, combined with the unmet need in treating DMD cardiomyopathy, will potentially lead to approval.

謝謝，AJ。大家下午好，感謝大家參加今天的第一季電話會議。今天，我將回顧我們的 BLA 的最新進展，該 BLA 尋求對 deramiocel 的全面批准以及治療杜氏肌肉營養不良症和心肌病，以及我們的管道計劃的簡要更新。雖然我們知道 FDA 已經做出了一些改變，但我們仍然相信，憑藉我們數據的優勢（我將在幾分鐘內重點介紹），再加上治療 DMD 心肌病變方面尚未滿足的需求，我們的藥物有可能獲得批准。

Our path with FDA to this point has been smooth, and FDA has not fallen behind in any way. Our objectives, deliverables, and timelines remain on track. There has also been concern expressed over the announcement of an FDA advisory committee meeting for Capricor.

到目前為止，我們與 FDA 的合作一直很順利，FDA 也沒有落後。我們的目標、交付成果和時間表均按計劃進行。人們也對 FDA 宣布召開 Capricor 諮詢委員會會議表示擔憂。

I want to highlight that having the opportunity to participate in an [AdCom] is a positive step for Capricor, for deramiocel, and for the program as a whole because it gives us the opportunity to showcase the strong scientific and clinical data that is the basis of our BLA.

我想強調的是，有機會參加 [AdCom] 對 Capricor、deramiocel 以及整個計畫來說都是積極的一步，因為它讓我們有機會展示作為我們 BLA 基礎的強大的科學和臨床數據。

We do not believe nor have FDA signaled that the determination to hold an [AdCom] had anything to do with weaknesses in the application, but rather, we believe the nature of a first in class therapy for a new indication warrants additional feedback from subject matter experts in the field, as well as giving the advocacy and patient community an opportunity to voice their opinion on deramiocel.

我們既不相信，FDA 也沒有表示召開 [AdCom] 的決定與申請中的弱點有關，相反，我們認為針對新適應症的一流療法的性質值得該領域的主題專家提供更多反饋，同時也讓倡導者和患者社區有機會表達他們對 deramiocel 的意見。

The [AdCom] also affords us the opportunity to highlight that deramiocel cell has a strong safety record demonstrated in over 700 infusions, treating over 250 patients with some subjects receiving deramiocel infusions for almost five years. We are asking for approval for a therapy that has been shown to be generally safe and effective for the treatment of DMD cardiomyopathy, for which there are no approved therapies.

[AdCom] 也為我們提供了機會強調，deramiocel 細胞在超過 700 次輸注中具有良好的安全記錄，治療了超過 250 名患者，其中一些受試者接受 deramiocel 輸注的時間長達近五年。我們正在請求批准一種已被證明對治療 DMD 心肌病變總體上是安全有效的療法，目前尚無針對該疾病的核准療法。

For those new to the story, deramiocel is a cellular therapy. It is a biologic comprised of a rare population of cardiac cells that reside in the heart. We have spent almost 20 years developing, characterizing, and harnessing their potential. Our clinical focus for the past approximately eight years has been Duchenne muscular dystrophy, a rare ex-linked neurodegenerative disease with no cure, afflicting approximately 15,000 boys and young men across the United States and over 150,000 across the world.

對於那些不熟悉這個故事的人來說，deramiocel 是一種細胞療法。它是一種生物製劑，由位於心臟內的稀有心臟細胞群組成。我們花了近20年的時間來開發、描述和利用它們的潛力。過去約八年來，我們的臨床重點一直是杜氏肌肉營養不良症，這是一種罕見的、無法治癒的神經退化性疾病，困擾著美國約 15,000 名男孩和年輕男性以及全球超過 150,000 名患者。

These young men aim to live life as any strong-willed young adolescent boy would desire to, but throughout the course of their life they suffer from devastating symptoms ranging from loss of ambulation, leading to full or part-time wheelchair use, deterioration of their upper limb function, leading to assistance needed to accomplish daily tasks such as feeding themselves or taking a drink of water. Followed typically by the use of ventilator support for breathing, and ultimately DMD takes their life.

這些年輕人的目標是像任何意志堅強的青春期男孩一樣生活，但在他們的一生中，他們遭受毀滅性的症狀，從失去行走能力，導致全職或部分時間使用輪椅，上肢功能惡化，導致需要幫助才能完成日常任務，例如自己進食或喝水。隨後通常需要使用呼吸器來支持呼吸，最終 DMD 奪走他們的生命。

I highlight these things to paint the picture of what these young men experience in their daily lives and why it is so important to develop therapies to attenuate the disease process.

我強調這些事情是為了描繪這些年輕人在日常生活中的經歷，以及為什麼開發治療方法來減輕疾病進程如此重要。

Now what is our indication exactly? It is the heart disease that affects every patient with DMD at some point in their lives. Every day, silently, the hearts of the DMD boys are being damaged, and no standard cardiac medication is good enough to combat that process.

那我們的指示到底是什麼呢？這種心臟病會在每位 DMD 患者一生中的某個階段影響他們。每天，患有杜氏肌肉營養不良症 (DMD) 的男孩的心臟都在默默地受到損害，而且沒有任何標準的心臟藥物能夠有效對抗這一過程。

DMD cardiomyopathy is now the leading cause of death in DMD, and deramiocel is the only therapeutic that has been shown to be effective in slowing the decline in ejection fraction, which is a measure of how the heart is meeting the needs of the body.

DMD 心肌病變現在是 DMD 患者死亡的主要原因，而德拉米奧塞爾是唯一被證明能夠有效減緩射血分數下降的治療方法，射血分數是衡量心臟如何滿足身體需求的指標。

While there has been some progress made in treating the dystrophinopathy, with several drug therapies now on the market in the US, specific to the genetic mutation associated with the disease, there are no therapies approved or on the market that aim to specifically treat the cardiomyopathy associated with GMT.

儘管在治療肌肉營養不良症方面取得了一些進展，目前美國市場上有幾種針對與該疾病相關的基因突變的藥物療法，但尚未有針對治療與 GMT 相關的心肌病變的療法獲得批准或上市。

Deramiocel's mechanism of action, which is immunomodulatory and anti-fibrotic, is directly targeted to treat the secondary effects of DMD.

Deramiocel 的作用機轉是免疫調節和抗纖維化，直接針對治療 DMD 的副作用。

And we believe can be used with other therapeutics which are currently approved or in development to treat the genetic mutation.

我們相信它可以與目前已批准或正在開發的其​​他療法一起用於治療基因突變。

Deramiocel is delivered by a simple intravenous infusion once a quarter at a dose of 150 million cells.

Deramiocel 透過每季一次的簡單靜脈輸注輸送，劑量為 1.5 億個細胞。

I would now like to discuss the data that supports our BLA.

我現在想討論支持我們的 BLA 的數據。

The filing is based on our blinded, randomized, and placebo-controlled HOPE-2 study, and also by the HOPE-2 open label extension study compared to a robust FDA and NHLBI funded natural history data set.

該文件基於我們的盲法、隨機和安慰劑對照的 HOPE-2 研究，以及與 FDA 和 NHLBI 資助的強大自然歷史資料集進行比較的 HOPE-2 開放標籤擴展研究。

While sample sizes are small, what is most relevant is not the size of the data set, but that the statistically and clinically significant differences are highly unlikely to be due to chance.

雖然樣本量很小，但最相關的不是資料集的大小，而是統計和臨床上的顯著差異極不可能是由於偶然造成的。

We have worked with multiple internal and external statisticians, presented the data at meetings and to KOLs, and what we have heard, seen, and acted upon was that the likelihood is extremely low that the impact on the heart, or for that matter the skeletal muscle, is due to chance.

我們與多位內部和外部統計學家合作，在會議和關鍵意見領袖 (KOL) 上展示數據，我們所聽到、看到並採取的行動是，對心臟或骨骼肌的影響是偶然發生的可能性極低。

We have three clinical files and approximately four years of open label extension data that supports that premise. There has also been an emphasis and written guidance from FDA encouraging the use of real world evidence to support clinical trial data, especially in rare diseases. Deramiocel is a perfect case for using this type of data to validate the efficacy of a drug product.

我們有三個臨床文件和大約四年的開放標籤擴展數據支持該前提。FDA 也強調並發出書面指導，鼓勵使用真實世界證據來支持臨床試驗數據，尤其是在罕見疾病方面。Deramiocel 是使用此類數據來驗證藥品功效的完美案例。

Turning to the HOPE-3 trial, our Phase 3 study, which is ongoing and fully enrolled in the United States, I want to be clear that at this time FDA has not requested the efficacy data from the HOPE-3 study to support our BLA application, although FDA has reviewed and will continue to review the safety data from the study.

談到 HOPE-3 試驗，這是我們正在進行的 3 期研究，該研究在美國已經全面展開，我想明確指出的是，目前 FDA 尚未要求提供 HOPE-3 研究的療效數據來支持我們的 BLA 申請，儘管 FDA 已經審查了該研究的安全性數據，並將繼續審查。

Our current plan is to use this data in the future for potential label expansion and are actively evaluating plans for HOPE-3 to be expanded internationally. We will provide more updates on this program as they become available.

我們目前的計劃是將來使用這些數據進行潛在的標籤擴展，並正在積極評估 HOPE-3 在國際上擴展的計劃。我們將隨時提供有關該計劃的更多更新資訊。

Now as we are transitioning Capricor from a translational medicine company into a commercial stage entity, we continue to actively work with our commercial partner NS Pharma on launch readiness for the United States.

現在，我們正在將 Capricor 從轉化醫學公司轉型為商業階段實體，我們將繼續積極與我們的商業合作夥伴 NS Pharma 合作，為在美國推出產品做好準備。

As we announced earlier today, we also have appointed Dr. Michael Binks as our new Chief Medical Officer. I am extremely proud that Dr. Binks joined our team. He has over 25 years of experience leading global clinical development, translational research efforts across the industry, most recently as Vice President and head of rare disease, clinical and translational research, worldwide research development and medical at Pfizer, and prior to that at GSK, where he was instrumental in advancing multiple first in class therapies in early and late-stage developments.

正如我們今天早些時候宣布的那樣，我們還任命邁克爾·賓克斯博士為我們的新首席醫療官。我非常自豪賓克斯博士加入我們的團隊。他擁有超過 25 年領導全球臨床開發和整個行業轉化研究工作的經驗，最近擔任輝瑞公司副總裁兼罕見疾病、臨床和轉化研究、全球研究開發和醫療負責人，之前在葛蘭素史克公司工作，在推動早期和後期開發中多種一流療法方面發揮了重要作用。

Based on our current plans, we aim to have over 100 patients transition from clinical to commercial products following potential BLA approval.

根據我們目前的計劃，我們的目標是在獲得 BLA 批准後讓超過 100 名患者從臨床產品過渡到商業產品。

Let me remind you that we have been providing Deramiocel to all open label extension patients for over three years. Nearly all HOPE-3 patients are in open label extension now and will transition to commercial products if it is their desire to continue Deramiocel.

讓我提醒您，我們已經向所有開放標籤擴展患者提供 Deramiocel 三年多了。目前，幾乎所有 HOPE-3 患者都處於開放標籤擴展階段，如果他們希望繼續使用 Deramiocel，他們將轉向商業產品。

We, along with NS Pharma, are now working with physicians to assist them in preparing to prescribe Deramiocel for DMG cardiomyopathy.

我們目前正與 NS Pharma 合作，與醫生合作，協助他們準備為 DMG 心肌病變開出 Deramiocel 處方。

We know it will be a partnership between treating neurologists and cardiologists and prescribing Deramiocel, and this is part of the reason we are enhancing our medical leadership with Dr. Binks, who will guide the physicians through the prescribing process.

我們知道這將是神經科醫生和心臟科醫生與 Deramiocel 處方之間的合作，這也是我們與 Binks 博士一起加強醫療領導力的原因之一，他將指導醫生完成處方過程。

Please remember that Deramiocel is not designed to compete with the medicines that address the dystrophinopathy, such as gene or exile skipping therapies, but rather to address the secondary aspects of the disease, which is inflammation and fibrosis, both in the heart and skeletal muscle and again has a very strong safety profile. It's important to note that a naturally derived cell therapy does not have the safety risk that is in any way similar to the gene therapies which do involve viral vectors.

請記住，Deramiocel 的設計目的並非與治療肌肉營養不良症的藥物（如基因療法或流放跳躍療法）競爭，而是解決該疾病的次要方面，即心臟和骨骼肌的炎症和纖維化，並且具有非常強的安全性。值得注意的是，天然細胞療法並沒有與涉及病毒載體的基因療法類似的安全風險。

Now for an update on our current commercial manufacturing preparations, as we built our San Diego GMP manufacturing facility for the purpose of commercial manufacturing, so I have a high degree of confidence in our processes, procedures, and facilities.

現在介紹一下我們目前的商業生產準備情況，由於我們為商業生產目的建造了聖地牙哥 GMP 生產設施，因此我對我們的流程、程序和設施非常有信心。

To remind you, our San Diego GMP facility is fully staffed and operational and is currently producing doses of Deramiocel.

提醒您，我們的聖地牙哥 GMP 工廠人員配備齊全，正常運轉，目前正在生產 Deramiocel 劑量。

In addition, we are underway with our previously announced manufacturing expansion to build additional clean rooms in the same facility. We plan on the expansion to be operational mid to late 2026, allowing us to bolster supply of the product to meet potential demands.

此外，我們正在進行先前宣布的生產擴建計劃，將在同一工廠內建造更多的無塵室。我們計劃在 2026 年中後期進行擴建，以加強產品供應，滿足潛在需求。

Turning to an update on our European partnering opportunities, we remain in negotiations with Nippon Shinyaku with respect to the potential distribution of Deramiocel in the European region and have extended the period of negotiation of the definitive agreement to the end of the second quarter.

談到我們在歐洲的合作機會的最新情況，我們仍在與日本新藥公司就 Deramiocel 在歐洲地區的潛在分銷進行談判，並已將最終協議的談判期延長至第二季度末。

Our strategy is emerging in regards to [ex USA] markets, and we will continue to explore opportunities for our technology in other areas globally. We will add additional color as our strategy for Europe continues to unfold.

我們的策略正在針對（美國以外）市場展開，我們將繼續在全球其他地區探索我們的技術機會。隨著我們歐洲戰略的不斷展開，我們將增添更多色彩。

And finally, for an update on our exosomes programs, we continue to develop our StealthX exosome platform technology as part of a next generation drug delivery platform. While this program has taken a backseat appropriately to Deramiocel, we are still working to develop exosomes as cellular delivery vehicles. We believe strongly in this opportunity and the exosome's ability to change the way we get biologics across the cell membrane.

最後，為了更新我們的外泌體計劃，我們將繼續開發 StealthX 外泌體平台技術，作為下一代藥物傳輸平台的一部分。雖然這個計畫已經適當地讓位給 Deramiocel，但我們仍在致力於開發外泌體作為細胞遞送載體。我們堅信這個機會和外泌體改變生物製劑穿過細胞膜的方式的能力。

Our exosome development team is focused on advancing an efficient and cost-effective way to manufacture them for scale for therapeutic utilization.

我們的外泌體開發團隊致力於開發一種高效且經濟的方法來大規模生產外泌體以供治療使用。

Despite all the concerns regarding vaccines based on the newer ethos of the FDA, I am pleased to inform you that a program under Project NextGen, which aims to test vaccine candidates for COVID-19 prevention and to prepare for future pandemics, remains underway. Our vaccine is very important because it is natural, made from the native proteins and contains no adjuvants, which has been one of the main concerns of the new HHS secretary.

儘管基於 FDA 的新理念，人們對疫苗存在許多擔憂，但我很高興地通知大家，下一代計畫 (Project NextGen) 下的一個計畫仍在進行中，該計畫旨在測試用於預防 COVID-19 的候選疫苗，並為未來的大流行做好準備。我們的疫苗非常重要，因為它是天然的，由天然蛋白質製成，不含佐劑，這是新任衛生與公共服務部部長主要關注的問題之一。

We continue to work in conjunction with the National Institute of Allergy and Infectious Disease, otherwise known as NIAD, which will conduct a clinical trial and provide the data to us. This vaccine could potentially be game changing as it meets all the criteria set forth by the US government of future vaccine technology. Phase 1 of the trial is set to start in Q3, and we will provide updates on this program as they become available.

我們將繼續與美國國家過敏和傳染病研究所（簡稱 NIAD）合作，該研究所將進行臨床試驗並向我們提供數據。這種疫苗可能會改變遊戲規則，因為它符合美國政府為未來疫苗技術設定的所有標準。試驗的第一階段將於第三季開始，我們將在該計劃發布最新更新後提供相關資訊。

In conclusion, our program remains strong both with our path to Deramiocel potential approval and our exosome platform. Our cash balance totals approximately $145 million with our current runway taking us into 2027 with no additional infusions of cash.

總而言之，我們的計劃仍然強勁，無論是在獲得 Deramiocel 潛在批准的道路上，還是在外泌體平台上。我們的現金餘額總計約為 1.45 億美元，目前的狀況是，我們無需額外注入現金即可維持到 2027 年。

If we receive FDA approval, we will be slated to receive an $80 million milestone payment from Nippon Shinyaku, and we will also receive a priority review voucher which we have the full rights to sell. These non-diluted cash infusions could potentially total well over $200 million. This would allow us to enhance our therapeutic pipeline for Deramiocel, as well as expand other areas of our pipeline in an effort to deliver value for patients and for our shareholders.

如果我們獲得 FDA 批准，我們將從日本新藥公司獲得 8000 萬美元的里程碑付款，並且我們還將獲得我們擁有完全銷售權的優先審查券。這些未稀釋的現金注入總額可能遠超過 2 億美元。這將使我們能夠增強 Deramiocel 的治療產品線，並擴展我們產品線的其他領域，從而為患者和股東創造價值。

With over 250 publications on the CDCs, including the mechanism of action Deramiocel and multiple statistically significant and clinically relevant clinical trials demonstrating Deramiocel's impact on patients, we look forward to presenting our data to the advisory committee. At this time, we don't know the specific date for the meeting, but we will alert the market when a date is set. I want to thank you for joining today's call. We truly appreciate your continued support. I will now turn the call over to AJ to run through our finances.

我們擁有超過 250 篇關於 CDC 的出版物，包括 Deramiocel 的作用機制以及多項具有統計學意義和臨床相關的臨床試驗，證明了 Deramiocel 對患者的影響，我們期待向諮詢委員會提交我們的數據。目前，我們還不知道會議的具體日期，但日期確定後我們會通知市場。感謝您參加今天的電話會議。我們衷心感謝您一直以來的支持。現在我將把電話轉給 AJ 來討論我們的財務狀況。

Thanks, Linda.

謝謝，琳達。

This afternoon's press release provided a summary of our first quarter, 2025 financials on a GAAP basis, and you may also refer to our quarterly report on Form 10Q, which we expect to become available shortly and will be accessible on the SEC website, as well as the financial section of our website. Let me start with our cash position. As of March 31, 2025, our cash equivalents, and marketable securities totaled approximately $144.8 million.

今天下午的新聞稿提供了我們 2025 年第一季基於 GAAP 的財務狀況摘要，您也可以參考我們的 10Q 表季度報告，該報告預計很快就會發布，可在美國證券交易委員會 (SEC) 網站以及我們網站的財務部分查閱。讓我先從我們的現金狀況開始。截至 2025 年 3 月 31 日，我們的現金等價物和有價證券總額約為 1.448 億美元。

Turning briefly to the financials, revenues for the first quarter of 2025 were zero compared to approximately $4.9 million for the first quarter of 2024. I'd like to point out that the source of our revenue for the first quarter of 24 was the radical recognition of the $40 million we received under our US distribution agreement with Nippon Shinyaku, which at this point has now been fully recognized as of December 31, 2024.

簡單回顧一下財務狀況，2025 年第一季的營收為零，而 2024 年第一季的營收約為 490 萬美元。我想指出的是，我們 24 年第一季的收入來源是我們與日本新藥株式會社簽訂的美國分銷協議所獲得的 4000 萬美元的徹底確認，截至 2024 年 12 月 31 日，這筆收入現已完全確認。

Moving to our operating expenses for the first quarter of 2025, excluding stock based compensation, our research and development expense for approximately $16.2 million compared to approximately $10.1 million in Q1 2024.

轉到 2025 年第一季的營運費用（不含股票薪酬），我們的研發費用約為 1,620 萬美元，而 2024 年第一季約為 1,010 萬美元。

Again, excluding stock-based compensation, our general and administrative expense was approximately $3.1 million in Q1 2025, approximately $1.8 million in Q1 2024. Net loss for the first quarter of 2025 was approximately $24.4 million compared to a net loss of approximately $9.8 million for the first quarter of 2024.

同樣，不包括股票薪酬，我們的一般和行政費用在 2025 年第一季約為 310 萬美元，2024 年第一季約為 180 萬美元。2025 年第一季淨虧損約 2,440 萬美元，而 2024 年第一季淨虧損約 980 萬美元。

We will now open the line up for questions.

我們現在開始回答問題。

Thank you, ladies and gentlemen, we will now begin the question-and-answer session. (Operator Instructions).

謝謝女士們、先生們，我們現在開始問答環節。（操作員指令）。

[Edward A. Tenthoff] from Piper's Sandler. Your line is open.

[愛德華·A.出自 Piper's Sandler 樂團的《Tenthoff》。您的線路已開通。

Great thank you very much can you hear me, okay?

太好了，非常感謝，你聽得到我說話嗎，好嗎？

We can hear you fine, thanks, [Ed]. How are you?

我們聽得很清楚，謝謝，[編輯]你好嗎？

Very well thanks. So, I just want to pick up on some of the comments that you have. Have you guys had your, site inspection yet in San Diego? And if not, can you tell us when that's scheduled for? And then, just with respect to preparation for the [AdCom], walk us through maybe what you consider to be key features and anything along the lines in terms of particular prep.

非常好，謝謝。因此，我只是想談談你們的一些評論。你們在聖地牙哥進行過現場檢查了嗎？如果沒有，您能告訴我們具體安排在什麼時候嗎？然後，關於 [AdCom] 的準備工作，請向我們介紹您認為的關鍵特點以及特定準備工作方面的內容。

Thank you.

謝謝。

Thanks [Ed]. Yeah, we haven't had our pre licensing inspection. It's coming up this quarter, within the next few weeks and so we'll update you guys once it's completed. I will tell you this place is a buzz with preparation and we feel really good about it and we're looking forward to having that inspection done, reminder which I said in my prepared remarks.

謝謝[編輯]。是的，我們還沒有進行許可前檢查。它將在本季度的未來幾週內推出，因此一旦完成，我們就會向大家更新。我會告訴你，這個地方正忙著準備，我們對此感覺非常好，我們期待著完成那次檢查，提醒一下，我在準備好的發言中說過。

But this facility was built in anticipation of commercial manufacturing and so we feel pretty ready for it. In terms of [AdCom] prep we are actively working on that we had anticipated, as we have said multiple times now that it was going to be requested by the agency typically as I also just stated. When there's a new indication or a new therapy first in class, they almost always conduct an [AdCom].

但該設施是為了商業製造而建造的，所以我們覺得已經做好了充分的準備。在 [AdCom] 準備方面，我們正在積極地進行我們預期的工作，正如我們多次說過的那樣，這通常是由機構提出的要求，正如我剛才所說的那樣。當出現新的適應症或新療法時，他們幾乎總是進行[廣告委員會]。

We actually think it's an incredibly good sign that they've asked for an [AdCom] one it shows that the agency is really moving forward with our application and I will tell you with, I think we're over 20 information requests now, a follow up meeting with them, as most recently as tomorrow, and several other meetings along the way they're actively in our file reviewing our file and working with us. They have told us in mid-cycle review that there were no substantive issues, which gave us good confidence in that in terms of AdCom prep we've already had two mock ad coms and without speaking, about that because of confidentiality, I can say that we passed with flying colors, and the data looks really great. So, we're looking forward to the future. We're waiting for our [produce] a date and things here are hopping along as we get ready for our approval.

我們實際上認為，他們要求 [AdCom] 是一個非常好的跡象，這表明該機構正在真正推進我們的申請，我會告訴你，我認為我們現在有超過 20 個資訊請求，與他們進行了一次後續會議，最近一次是在明天，以及在此過程中的其他幾次會議，他們正在積極審查我們的文件並與我們合作。他們在中期審查中告訴我們，沒有實質問題，這給了我們很大的信心，在 AdCom 準備方面，我們已經進行了兩次模擬廣告會議，由於保密性，我不會談論這件事，我可以說我們以優異的成績通過了，數據看起來真的很棒。因此，我們對未來充滿期待。我們正在等待我們的[生產]日期，這裡的事情進展順利，我們準備獲得批准。

Great thank you so much.

太好了非常感謝。

Leland Gershell, Oppenheimer. Your line is open.

利蘭·格謝爾，奧本海默。您的線路已開通。

Good afternoon and thanks for taking our questions. Just two from us, Linda, first, just want to ask with respect to Nippon Shinyaku, you had signed kind of that that, letter of intent or what have you back in the fall, and I know you'd updated us that you've extended that negotiation period.

下午好，感謝您回答我們的問題。琳達，我們只想問兩個問題，關於日本新藥，你們在秋天簽署了那種意向書或什麼的，我知道你已經告訴我們你已經延長了談判期。

Just wondering if we could also maybe read into that. That you may be considering an alternative mechanism for launching in Europe perhaps on your own, which could be more lucrative to Capricor in terms of economic preservation, as you've seen rare disease therapy is often to be even better over in Europe than in the states. And the second question is with respect to and that's in the states. If you could just share to the extent, you can kind of how they're set up here in terms of their footprint, for marketing Deramiocel presenting approval. Thanks.

我只是想知道我們是否也可以讀到這一點。您可能正在考慮自行在歐洲推出替代機制，這對 Capricor 的經濟保護而言可能更有利可圖，正如您所見，罕見疾病治療在歐洲通常比在美國更好。第二個問題與美國有關。如果您可以分享一下，您可以大致了解他們在這裡是如何建立的，就他們的足跡而言，為了行銷 Deramiocel 而提出的批准。謝謝。

Frankly, it's always great to hear your voice. So, addressing your first question about our LOI with NS and the EU opportunity, we have had really great experiences building towards commercialization with NS in the United States. We think that they're a great partner in the US and we're excited for the launch, and I'll get into some of that color in your second question. In terms of Europe, we've moved forward very rapidly in the United States.

坦白說，聽到你的聲音總是很高興。因此，回答您關於我們與 NS 的意向書和歐盟機會的第一個問題，我們在美國與 NS 合作實現商業化方面擁有非常豐富的經驗。我們認為他們是美國的優秀合作夥伴，我們對此次發布感到非常興奮，我將在您的第二個問題中深入探討這一點。就歐洲而言，我們在美國取得了非常迅速的進展。

We were told and have now filed a BLA for Deramiocel in the US. We have a great plan in the US, and we believe that can be enacted in Europe, as you correctly stated. Therapies for rare diseases have a different path in Europe, and there's nothing currently improved in Europe for GMD, and our data is very strong. And meet the AMA criteria. We are working directly with the European authorities ourselves right now as we prepare for moving into Europe and so we are evaluating on a regular basis the opportunity. We certainly remain in active negotiations with Nippon Shinyaku and we'll provide more updates on that program as our thoughts and theirs evolve.

我們已獲悉此事並已在美國提交了 Deramiocel 的 BLA。正如您正確指出的那樣，我們在美國有一個偉大的計劃，我們相信這個計劃也可以在歐洲實施。歐洲對罕見疾病的治療有不同的路徑，目前歐洲對 GMD 的治療沒有任何改進，而且我們的數據非常強勁。並滿足AMA標準。我們目前正在與歐洲當局直接合作，為進入歐洲做準備，因此我們會定期評估這個機會。我們當然仍在與日本新藥進行積極的談判，隨著我們的想法和他們的想法不斷發展，我們將提供有關該計劃的更多更新。

In terms of their opportunity in the states, they've done a really nice job of building a sales and marketing distribution team for Viltepso, which is their exon skipper, and so these seasoned executives who are US based and have spent a lot of time in the [Duchennes] space are preparing to launch deramiocel. They have 125 FTEs. We're told that nearly all of them are focusing on deramiocel at this point. We work with their leadership. Within the states and also in Japan so that we remain aligned on the path forward. We are enhancing our own management team, as you heard today, we appointed Dr. Michael Binks as Chief Medical Officer.

就他們在美國的機會而言，他們已經為 Viltepso 建立了一個銷售和營銷分銷團隊，Viltepso 是他們的外顯子船長，因此這些在美國並且在 [Duchennes] 領域花費了大量時間的經驗豐富的高管正在準備推出 deramiocel。他們有 125 名全職員工。我們得知，目前幾乎所有公司都將注意力集中在 deramiocel 上。我們與他們的領導階層合作。在美國國內以及日本，我們都能保持一致的前進方向。我們正在加強我們自己的管理團隊，正如您今天所聽到的，我們任命邁克爾·賓克斯博士為首席醫療官。

Dr. Binks is going to build some Medifare support behind him, which we'll be talking about over the next month or two, and we will continue to support Nippon Shinyaku in their education and commercialization for deramiocel to physicians in the community.

Binks 博士將爭取一些醫療保險支持，我們將在接下來的一兩個月內討論這個問題，我們將繼續支持日本新藥公司 (Nippon Shinyaku) 對社區醫生進行 deramiocel 的教育和商業化。

Thanks very much.

非常感謝。

Thank Leland.

謝謝 Leland。

Kristen Kluska, Cantor Fitzgerald. Your line is open.

克里斯汀·克魯斯卡，坎托·費茲傑拉。您的線路已開通。

Hi, good afternoon, everyone. Thanks for taking the questions, and I look forward to meeting and working with Dr. Binks on your team. So, you made a comment that when you talk to several key leaders, they truly point out that these effects can't be by chance, and that's something that we often hear as well when we speak to thought leaders. I wanted to ask what are the biggest key drivers that these physicians say is that proof, and I know the FDA certainly is no stranger to the natural history work having funded it themselves, but maybe can you talk about what they're viewing as truly the strongest signals when they look at that data set.

大家好，下午好。感謝您回答這些問題，我期待與您的團隊中的 Binks 博士會面並合作。所以，您評論說，當您與幾位關鍵領導人交談時，他們確實指出這些影響不可能是偶然的，這也是我們在與思想領袖交談時經常聽到的。我想問一下，這些醫生所說的最重要的關鍵驅動因素是什麼，我知道 FDA 對自然歷史研究並不陌生，他們自己也資助過這項工作，但您能否談談，當他們看到該數據集時，他們認為什麼是真正最強烈的信號。

Yeah Kristen, thanks. So, you have just hit on the most important part of our application, and I want to highlight that. The relevance, even though theoretically in terms of new numbers of patients, the numbers are small, the sample size is small. In reality, the reason that we are able to say very confidently that there is very little chance that the data is due to chance is because of the statistical significance.

是的，克里斯汀，謝謝。所以，您剛剛觸及了我們應用程式中最重要的部分，我想強調這一點。相關性，儘管從理論上講，就新患者數量而言，數量很少，樣本量也小。實際上，我們之所以能夠非常自信地說資料是偶然產生的可能性很小，是因為其具有統計顯著性。

Now the statistical. Significance is actually a number that says how much likelihood is your data due to chance, and what we are able to look at here is that MRI, cardiac MRI, which is an objective measure of cardiac function, you can't wish your heart better. You can't volitionally think, [gee], I'm going to perform better in the MRI today, and you can't do anything really to sustain or improve cardiac function in any relevant way in a short-term basis.

現在進行統計。重要性實際上是一個數字，它表示您的數據有多少可能性是由偶然因素造成的，我們在這裡可以看到 MRI，即心臟 MRI，它是心臟功能的客觀衡量標準，您不能希望您的心臟變得更好。你不能隨意地想，[哎呀]，我今天的核磁共振檢查結果會更好，而且你也無法真正做任何事情來在短期內以任何相關的方式維持或改善心臟功能。

And that is why MRI is such a clever and easy way of determining disease progression. So, Dr. Jonathan Saslow, who also is the author, lead author on the paper for the Natural History Study and is funded, as I mentioned by FDA and NHLBI to collect that data, has spoken very eloquently and pointed to literature. That shows that very few patients are actually necessary to discern the treatment effect using cardiac MRI. So we are confident in the data.

這就是為什麼 MRI 是一種如此聰明且簡單的確定病情進展的方法。因此，喬納森·薩斯洛博士（他也是《自然歷史研究》論文的作者和主要作者，並且得到了 FDA 和 NHLBI 的資助，正如我提到的那樣，他收集了這些數據，他講得非常有說服力，並引用了文獻。這表明實際上需要很少的患者才能透過心臟MRI來辨別治療效果。所以我們對數據很有信心。

We are hopeful that the FDA will be confident in the data and that the outcome will also support the data because, as I mentioned, taken together mathematically as well as in an individual patient basis, the data is a very strong suggestion of not only improvement and ejection fraction, which is a measure of how the heart meets the needs of the body, but also the long term stabilization and even improvement in cardiac function. Our open label extension guys are out to four years now, and some of them have dropped literally no ejection fraction points, which in a non-ambulant later stage descent patient is almost unheard of.

我們希望 FDA 對這些數據有信心，並且結果也將支持這些數據，因為正如我所提到的，從數學角度以及從個別患者的角度綜合考慮，這些數據不僅非常有力地表明了射血分數的改善（這是衡量心臟如何滿足身體需要的標準），而且還表明了心臟功能的長期穩定甚至改善。我們的開放標籤擴展人員現在已經工作了四年，其中一些人的射血分數幾乎沒有下降，這在無法行走的晚期下降患者中幾乎是聞所未聞的。

Thank you for that and yeah, that was literally going to be my next question. Last summer is when you presented the three year data and well I know we don't have a date yet for this ad con I'm wondering if you will have any four year data to share at that point with the agency or if you've been disclosing early data as you've had these ongoing review meetings.

謝謝你，是的，這確實是我的下一個問題。去年夏天，您展示了三年的數據，我知道我們還沒有確定這次廣告會議的日期，我想知道您是否有四年的數據可以與該機構分享，或者您是否在進行這些持續的審查會議時披露了早期數據。

Yeah, we plan on presenting the four year data at the PPMG meeting in June, and you know I'm delighted to say that the four year data is looking very promising. I'll give you that little bit of a preview, but it is really quite exciting to see this long-term stabilization, as I mentioned, not only in the disease process, but remember our guys are the older guys. These are the. That are in the later stage of the disease that they are losing function in a measurable and very almost reliable sad to say way and so the fact that there's long term stabilization in cardiac as well as performance of the upper limb function is really important and data we plan to highlight at PPMG.

是的，我們計劃在 6 月的 PPMG 會議上展示四年的數據，我很高興地說，四年的數據看起來非常有希望。我會給你一點預覽，但看到這種長期穩定確實非常令人興奮，正如我所提到的，不僅在疾病過程中，而且記住我們的人都是年紀較大的人。這些是。處於疾病後期的患者正在以可測量且幾乎可靠的方式喪失功能，這令人悲傷，因此，心臟和上肢功能表現的長期穩定非常重要，我們計劃在 PPMG 上重點介紹這些數據。

Okay thanks and then the last question that I had is obviously there's been a lot of changes at the administration so can you just talk from a high level if the people that you've been speaking with over the course of the year and beyond when you first presented these data to the FDA has there been a lot of changes within that or for the most part are most of the people that you're speaking and working with the same people that have been part of the process. Thanks again.

好的，謝謝。我的最後一個問題是，顯然政府內部發生了很多變化，因此您能否從高層次談談，在您第一次向 FDA 提交這些數據時，您在過去一年及以後與之交談過的人是否發生了很多變化，或者在大多數情況下，與您交談和合作的人仍然是參與該過程的同一批人。再次感謝。

Everyone.

每個人。

Thanks, Kristen. It's funny. I get asked that question now. I don't know, 8 times to 10 times per day by investors, and I think it's really important to say that we see the FDA really starting to calm down and stabilize the appointment of Dr. Makari, the appointment of Dr. Prasad, both of which have spoken about their commitment to rare disease, their commitment to moving therapies forward, and their commitment to making sure that medicines get to patients as they need them based on good quality data, which Capricor certainly has given us great confidence, taken together, the reviewers that we've worked with over time for the most part, most of them are there we believe that [Nicole Verdun] is still there and she has been working on our file really since the beginning of 2024 when we started having high visibility with the agency and. All of the other reviewers I don't want to call out their names are definitely still there, definitely engaged and as I mentioned in my answer to my question to [Ted], we are getting literally bombarded with questions and opportunities to continue the conversation with FDA on a weekly basis so we know they're actively in our file.

謝謝，克里斯汀。很有趣。現在有人問我這個問題。我不知道，投資者每天會諮詢 8 到 10 次，我認為非常重要的是，我們看到 FDA 真的開始冷靜下來並穩定 Makari 博士的任命和 Prasad 博士的任命，兩人都談到了他們對罕見疾病的承諾、他們對推動療法發展的承諾以及他們致力於確保藥物根據高質量數據及時送達患者的承諾，Capricor 無疑給了我們那裡的信心Verdun] 仍然在那裡，她從 2024 年初我們開始在該機構獲得高度關注以來就一直在處理我們的文件。我不想點名的所有其他審閱者肯定還在那裡，肯定在參與，正如我在回答 [Ted] 的問題時提到的那樣，我們每週都會收到大量問題和繼續與 FDA 對話的機會，因此我們知道他們在我們的檔案中很活躍。

Catherine Novack, Jones Research. Your line is open.

凱瑟琳‧諾瓦克，瓊斯研究公司。您的線路已開通。

Hi. Good afternoon. Thanks for taking my questions. I'm just wondering strategically, suppose that the FDA issued a CRL for efficacy in August. What's the plan? Would you then read out HOPE-3 and submit for DMD skeletal muscle function, where do you see going from there?

你好。午安.感謝您回答我的問題。我只是從策略角度考慮，假設 FDA 在 8 月發布了關於療效的 CRL。有什麼計劃？那麼，您會讀出 HOPE-3 並提交 DMD 骨骼肌功能嗎？您認為接下來會怎麼發展？

Yeah, that's exactly right. Hi, nice to chat with you again. So, we are in a really unique and quite favorable position in that way. We have a fully enrolled phase 3 trial, the indication for which is skeletal muscle dysfunction, particularly in the non-ambulant patients, is measured by the performance of the upper limb. So a different indication on the cardiomyopathy.

是的，完全正確。你好，很高興再次和你聊天。因此，從這個角度來看，我們處於真正獨特且非常有利的地位。我們有一個已完全招募的 3 期試驗，其適應症是骨骼肌功能障礙，特別是對於無法行走的患者，是透過上肢的表現來衡量的。因此對心肌病變有不同的指徵。

If in the unexpected circumstance they are, they issue a CR for whatever reason in August we just turn around and submit the data for the HOPE-3 trial which we expect to be positive based on three positive clinical trials proceeding and just go after. Skeletal muscle indication and the secondary endpoints for HOPE-3 are the same cardiac ones that we've applied for. So, we would just apply for cardiac and skeletal based on that. It's a randomized double-blind placebo-controlled trial and certainly would support any of the findings that we've already submitted on.

如果在意外情況下，他們在 8 月出於任何原因發布了 CR，我們只需轉身提交 HOPE-3 試驗的數據，根據正在進行的三項積極的臨床試驗結果，我們預計該試驗將獲得積極結果，然後繼續進行。HOPE-3 的骨骼肌適應症和次要終點與我們申請的心臟適應症相同。因此，我們僅以此為基礎申請心臟和骨骼治療。這是一項隨機雙盲安慰劑對照試驗，肯定會支持我們已經提交的任何研究結果。

Got it. So, you would intend to submit for both if you were filing with HOPE-3 data.

知道了。因此，如果您使用 HOPE-3 資料進行歸檔，那麼您打算同時提交這兩項資料。

Yeah, I mean, I'm expecting that the data would be reflective of what we've seen so far. We have done blinded assessments of the HOPE-3 data compared to the HOPE-2 data, and the distribution charts look almost identical. They can overlay each other, so I feel very confident. That we would see similar results in HOPE-3 that we saw on HOPE-2.

是的，我的意思是，我希望數據能反映我們目前所看到的情況。我們對 HOPE-3 資料和 HOPE-2 資料進行了盲法評估，發現分佈圖幾乎完全相同。它們可以相互疊加，所以我感覺非常有信心。我們會在 HOPE-3 中看到與 HOPE-2 類似的結果。

And I haven't seen, there's no way to preview, blinded data for cardiac and so, we expect that to be positive as well based on the MRI data we've seen in all our trials.

我還沒有看到，沒有辦法預覽心臟的盲法數據，因此，根據我們在所有試驗中看到的 MRI 數據，我們預計這也是正面的。

Great. And then, if you can share any specific feedback [FDA] has given you about LVF as a surrogate endpoint, what have they said about this endpoint, the ability to predict cardiac outcomes? How much of that are you supporting with your OLE data? Thanks.

偉大的。然後，如果您可以分享 [FDA] 給您的任何關於 LVF 作為替代終點的具體回饋，他們對這個終點、預測心臟結果的能力有何評價？您的 OLE 數據支援多少這樣的情況？謝謝。

Yeah. So, I actually read some of this in one of your notes, so I think it's an important point to highlight. FDA has stated that they're not looking at ejection fraction as a surrogate endpoint in this specific situation, rare disease DMD. That's why they funded the study with John [Sosso] by the Office of Orphan Products co-funded with the NHLBI, was to determine what measures they could conclude to be outcome measures in a rare disease.

是的。所以，我實際上在你的一篇筆記中讀到了其中的一些內容，所以我認為這是需要強調的重要一點。FDA 表示，在這種特殊情況下，即罕見疾病 DMD，他們不會將射血分數作為替代終點。這就是他們與約翰 [索索] 共同資助由孤兒藥產品辦公室和 NHLBI 共同資助的研究的原因，目的是確定他們可以得出哪些指標作為罕見疾病的結果指標。

In a rare [pediatric] disease, you cannot do an outcome measure like mortality. It's not going to happen. So, you have to be able to find other measures that are analogous to those outcomes of mortality or some other type of hospitalization, other types of standard cardiac measurements of progression of disease and use those. And so [Dr. Sosso] published in the journal Circulation Heart Failure in 2023, this beautiful study with the national history data that we were then. Able to use on a patient level in a propensity matched way to show that the ejection fraction in treated patients with deramiocel was significantly improved over what would be expected in actual history.

對於罕見的[兒科]疾病，你無法進行死亡率等結果測量。這不會發生。因此，您必須能夠找到與死亡率或其他類型住院率的結果類似的其他測量方法、其他類型的疾病進展標準心臟測量方法並使用它們。所以[Dr. Sosso 於 2023 年在《循環心臟衰竭》雜誌上發表了這項精彩的研究，採用了當時的國家歷史數據。能夠以傾向匹配的方式在患者層面使用，以表明使用 deramiocel 治療的患者的射血分數比實際病史預期的有顯著改善。

Real world evidence has been stated by FDA and it's been put forth in guidance as well as in a new office in CIR that emphasizes looking at the actual progression of a disease, real world evidence, and able to use that. Analogous to a clinical trial data set to make sure that efficacy is effectively analyzed, so we are in the really nice position of having good efficacy data of our own that we can compare to a natural history data set in an objective measure which is cardiac MRI of disease progression. And so overall we don't look at ejection fraction, nor does the FDA in this situation as a surrogate measure but rather an outcome measure.

FDA 已聲明真實世界證據，並將其納入指南以及 CIR 的新辦公室中，該辦公室強調關注疾病的實際進展、真實世界證據並能夠利用這些證據。類似於臨床試驗數據集，以確保有效分析療效，因此我們處於非常好的位置，擁有我們自己的良好療效數據，我們可以將其與客觀測量的自然歷史數據集進行比較，即疾病進展的心臟 MRI。因此，整體而言，我們不會將射血分數作為替代指標，FDA 在這種情況下也不會將其作為結果指標。

Got it, thanks for clarifying that for me that was very helpful.

明白了，謝謝你的澄清，這對我非常有幫助。

No problem.

沒問題。

Aydin Huseynov, Ladenburg. Your line is open.

拉登堡的艾登‧胡賽諾夫 (Aydin Huseynov)。您的線路已開通。

Hi, good afternoon, Linda. Congrats for the quarter.

你好，下午好，琳達。恭喜本季。

Thank you for taking questions. I got a couple.

謝謝您的提問。我有一對。

So given the sort of general pressure on all gene and cell therapy companies recently and especially in those sort of treatments that were approved conditionally for almost a decade, never got confirmatory studies, do you think a full approval or my cell would put it into some kind of, Specific position when it can actually be utilized more than [exosome skipper] or AV gene therapies.

因此，考慮到最近所有基因和細胞治療公司面臨的普遍壓力，特別是那些近十年有條件批准、從未得到確認性研究的治療方法，您是否認為完全批准或我的細胞療法會使其處於某種特定的位置，而這種位置實際上可以比 [外泌體跳躍者] 或 AV 基因療法得到更多的利用。

Yeah, so I really can't comment on that specifically. We certainly would be in a good position with full approval. We have highlighted in our biologic license application and the community is well aware that we see both skeletal and cardiac benefit, so it's possible that physicians would use it with both in mind.

是的，所以我真的無法對此發表具體評論。如果獲得全面批准，我們肯定會處於有利地位。我們在生物製品許可申請中已經強調了這一點，業界也清楚地知道我們看到了骨骼和心臟方面的益處，因此醫生可能會同時考慮這兩種益處來使用它。

The patient population that typically have cardiac dysfunction that would be starting out on deramiocel probably already have implications of skeletal muscle dysfunction that would make them eligible to benefit from deramiocel. So, you know that remains to be seen how FDA puts forth the label where we have applied for full approval, and we'll provide updates when we have our label discussions later this summer. They're already scheduled.

開始使用德拉米奧塞爾的患者通常患有心臟功能障礙，他們可能已經患有骨骼肌功能障礙，這使得他們有資格從德拉米奧塞爾中受益。因此，您知道，FDA 如何推出我們已經申請全面批准的標籤還有待觀察，我們將在今年夏天晚些時候進行標籤討論時提供更新資訊。他們已經安排好了。

Appreciate that. And for the whole data, could you, remind us again when do you plan to disclose that, I mean, irrespective of what's going to happen in August, and if you get approved in based on HOPE-2, how do you think the label is going to change hypothetically once you disclose HOPE-3 data and add for a label expansion?

非常感謝。對於整個數據，您能否再次提醒我們，您計劃何時披露這些數據，我的意思是，無論 8 月份發生什麼，如果您根據 HOPE-2 獲得批准，您認為一旦您披露 HOPE-3 數據並添加標籤擴展，標籤將如何假設變化？

Yeah, so we're in a really great position, as I said a minute ago with HOPE-3. Our current plans are somewhat fluid. We're making sure that everything proceeds as planned with this application for the cardiomyopathy.

是的，正如我剛才在 HOPE-3 中所說的那樣，我們處於非常好的位置。我們目前的計劃有些不確定。我們正在確保這項針對心肌病變的應用一切按計劃進行。

If it does, which we fully expect it to, then our plans are to take HOPE-3 to Europe and potentially to Japan to be able to expand our label globally as we've talked about. That would mean that we would keep HOPE-3 blind. A more patients from the appropriate geographic area and unblinded when those patients had reached their timeline for their primary efficacy endpoint, which would be a year after enrolling into the study. So that would change the unveiling of the HOPE-3 data if for some reason, as the previous questioner just asked, there was some reason that we needed to move very quickly towards unblinding the HOPE-3 data.

如果確實如此，我們完全期待它會發生，那麼我們的計劃是將 HOPE-3 帶到歐洲，並可能帶到日本，以便能夠在全球範圍內擴展我們的品牌，正如我們所討論的那樣。這意味著我們將對 HOPE-3 保持盲目態度。來自適當地理區域的更多患者，並在這些患者達到其主要療效終點的時間線時揭盲，這將是參與研究一年後。因此，如果出於某種原因，正如前一位提問者剛才問到的，我們需要非常迅速地揭開 HOPE-3 數據的神秘面紗，那麼這將改變 HOPE-3 數據的公開。

It would be likely in the third quarter, early fourth quarter of this year, and we would then, be able to expand the label in that way either in terms of skeletal muscle implications. Right now, the primary efficacy endpoint is the performance of the upper limb 2.0. We have had. Preliminary discussions with the FDA, although they were in 2024, that we would ask for approval for full DMD as opposed to all skeletal muscle myopathies down to diagnosis, but we have not had full labeling discussions with them, nor have we finalized our plans on how that indication would look, but it would be for skeletal muscle myopathy.

這很可能在今年第三季或第四季初實現，屆時我們將能夠透過這種方式擴大標籤，無論是在骨骼肌影響方面。目前，主要療效終點是上肢 2.0 的表現。我們有過。儘管是在 2024 年，我們與 FDA 進行了初步討論，但我們將請求批准用於治療全部 DMD，而不是所有診斷出的骨骼肌肌病，但我們尚未與他們進行全面的標籤討論，也沒有最終確定該適應症的計劃，但它將用於治療骨骼肌肌病。

Thank you, Linda. Very helpful. The final last question is regarding PRV voucher that you may be eligible to receive in August or September. So, would you plan to sell it right away and get whatever the last price is, I think $155 million, or would you like to keep it for possibly using for some indications on your own, such as [Duchenne muscular dystrophy]?

謝謝你，琳達。非常有幫助。最後一個問題是關於您可能有資格在八月或九月收到的 PRV 代金券。那麼，您打算立即出售它並獲得最終價格，我認為是 1.55 億美元，還是想保留它以供自己使用，例如[杜氏肌肉營養不良症]？

Yeah, our current plan is to sell it. It will really strengthen our balance sheet, and we feel that any indications that have come along, we probably wouldn't need a PRV. We'd get probably priority review anyway for a rare disease, so I don't think that would be as helpful to us as the dollars in our bank account.

是的，我們目前的計劃是出售它。它將真正加強我們的資產負債表，而且我們認為，根據出現的任何跡象，我們可能不需要 PRV。無論如何，我們可能會因為一種罕見疾病而獲得優先審查，所以我認為這對我們的幫助不如我們銀行帳戶裡的錢大。

Okay, thank you, thanks so much.

好的，謝謝，非常感謝。

Thanks, Aydin.

謝謝，艾登。

(Operator Instructions). Boobalan Pachaiyappan, Roth Capital. Your line is open.

（操作員指令）。Boobalan Pachaiyappan，羅斯資本。您的線路已開通。

Hi, my name is Manasa, and I'm dialing in for Boobalan. So, congrats on your progress team, and we have a few questions. First, in your pay, market research studies, have the participants indicated the type of profile that you know would make their own best in indication?

你好，我叫 Manasa，我正在為 Boobalan 撥號。所以，恭喜你們的進步團隊，我們有幾個問題。首先，在您的薪資市場調查研究中，參與者是否顯示了您認為最適合自己的類型？

I'm sorry, so are you asking which patients would do best under my cell?

抱歉，您是想問哪些病人在我的牢房裡表現最好？

Yeah, like, have the participants indicated the type of, the profile that would like what that preference would be, yeah, that's what I'm trying to ask.

是的，例如，參與者是否表明了他們喜歡的類型，他們喜歡的偏好是什麼，是的，這就是我想問的。

Yeah, so right now what we are looking for in the label is either the presence of LGE, which is la gadolinium enhancement, or scar in the heart as measured by MRI. Sometimes you can get that very young in age, and the kids don't know it. Their sort of ignorance is bliss in terms of the damage to their heart, but they need to start on deramiocel.

是的，所以現在我們在標籤中尋找的是 LGE 的存在，即钆增強，或是透過 MRI 測量的心臟疤痕。有時你可能年紀很小，但孩子不知道。就對心臟的損害而言，他們的無知是一種福氣，但他們需要開始使用 deramiocel。

So that we can attenuate that progression and or the presence of cardiac dysfunction, which would be measured by an ejection fraction of 55% or less, which could be measured in any way, whether by MRI or by echo, so the presence of either or both of those situations would allow for the prescribing of deramiocel, at least in its first iterations.

這樣，我們就可以減緩病情的進展和/或心臟功能障礙的存在，這可以透過射血分數 55% 或更低來衡量，可以透過任何方式測量，無論是透過 MRI 還是透過超音波檢查，因此，其中一種或兩種情況的存在都可以開出德拉米奧塞爾處方，至少在其第一次迭代中是如此。

Okay, and the next question is like, can you provide some color on the progress achieved with the Japanese regulators?

好的，下一個問題是，您能否介紹一下與日本監管機構的進展？

Yeah, so we're just working with the Japanese regulators now. We've actually been in conversations with them for a couple of years. They knew that we were waiting for FDA approval before proceeding in Japan. We have some upcoming meetings with the CRO that we're working with in Japan. It's a very well-known one called CMIC, and then we're planning to have PMDA meetings either later this year or early next year as we plan on propelling deramiocel forward in Japan.

是的，我們現在正在與日本監管機構合作。事實上我們已經與他們交談了幾年。他們知道我們正在等待 FDA 批准才能在日本開展業務。我們即將與日本合作的 CRO 舉行一些會議。這是一個非常有名的組織，叫做 CMIC，我們計劃在今年稍後或明年年初召開 PMDA 會議，因為我們計劃在日本推動 deramiocel 的發展。

Well we have some ideas of what's going to be required, which does not look like it's going to be too difficult to achieve. I don't have specific information to disclose yet on Japan, but we're actively working on that now.

嗯，我們對需要做的事情有一些想法，看起來實現起來並不太難。關於日本，我目前還沒有具體的資訊可以透露，但我們現在正在積極努力。

Okay, thank you. And the last question is, can you kind of, speak about the [AdCom] like can you speak to the [AdCom] committee composition and expertise and do you regard the [AdCom] as a positive indicator?

好的，謝謝。最後一個問題是，您能否談談 [AdCom]，例如您能否談談 [AdCom] 委員會的組成和專業知識，您是否認為 [AdCom] 是一個積極的指標？

Yeah, so the components or the participants or panels of the [AdCom] is available online. I don't have it in front of me right now, but you can find it. It's the cell and gene therapy Advisory Committee board. They select from there and then they're allowed to put [AdCom] members onto the ad com. Obviously we don't know who those are yet. We don't have a. Get for [AdCom], but we are practicing regularly. The company that we're working with brings in panelists that have curriculum details or resumes that are very similar to the panelists so that they can ask and answer questions for us and with us so that we are well prepared and we feel really good about it.

是的，[AdCom] 的組成部分、參與者或小組都可以在線上查閱。我現在沒有它，但你可以找到它。這是細胞和基因治療諮詢委員會。他們從那裡進行選擇，然後將 [AdCom] 成員放入廣告委員會中。顯然我們還不知道他們是誰。我們沒有。爭取[AdCom]，但我們正在定期練習。與我們合作的公司會邀請一些與小組成員有著非常相似課程細節或簡歷的小組成員，以便他們可以為我們提問和回答問題，讓我們做好充分的準備，我們對此感覺非常好。

Okay.

好的。

Thank you so much and congratulations again on.

非常感謝，並再次恭喜。

Your progress.

你的進步。

Thank you so much.

太感謝了。

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

現在我將把電話轉回給 Capricor 管理層，徵求他們的最終意見。

Thank you so much for joining today's quarterly call. We look forward to updating you on our progress. Obviously, there's going to be a lot of it over the coming months, and we will definitely keep you informed as we move forward, operator, you may now disconnect.

非常感謝您參加今天的季度電話會議。我們期待向您通報我們的進展。顯然，在接下來的幾個月裡，這種情況會很多，我們一定會隨時向您通報進度情況，接線員，您現在可以斷開連接了。

This concludes today's conference.

今天的會議到此結束。

Thank you for participating in my now disconnect.

感謝您參與我現在的斷開連接。