Capricor Therapeutics Inc (CAPR) 2024 Q2 法說會逐字稿

Good afternoon, ladies and gentlemen, and welcome to the Capricor Therapeutics Second Quarter 2024 earnings call.

女士們、先生們，下午好，歡迎參加 Capricor Therapeutics 2024 年第二季財報電話會議。

(Operator Instruction) Also know that this call is being recorded on Wednesday, August 7, 2024. And I would like to turn the call over to our CFO, AJ Bergmann, for the forward-looking statements.

（操作員說明）另請注意，此通話的錄音時間為 2024 年 8 月 7 日星期三。我想將電話轉給我們的財務長 AJ Bergmann，以獲取前瞻性陳述。

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.

謝謝大家，大家下午好。在開始之前，我想聲明，我們將在今天的演示中做出某些前瞻性陳述。

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 preclinical and clinical studies, enrollment of patients in our clinical studies, our plans to present or report additional data, our plans regarding regulatory filings, potential regulatory developments involving our product candidates, revenue and reimbursement estimates, manufacturing capabilities, potential milestone payments, our financial position and our possible uses of existing cash and investment resources.

這些聲明可能包括有關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的聲明，包括我們預期的臨床前和臨床研究的進行和時間表、我們臨床研究中的患者入組、我們的計劃提供或報告其他數據、我們有關監管備案的計劃、涉及我們候選產品的潛在監管發展、收入和報銷估算、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使用。

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 the actual results to differ materially from those contained in the forward-looking statements.

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包含的結果有重大差異。

These and other risks are described in our periodic filings made 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.

這些風險和其他風險在我們向美國證券交易委員會提交的定期文件中進行了描述，包括我們的季度和年度報告。請您注意不要過度依賴這些前瞻性陳述，我們不承擔更新此類陳述的義務。

With that, I'll turn the call over to Linda Marbán, CEO.

接下來，我會將電話轉給執行長琳達·馬班 (Linda Marbán)。

Thanks, AJ. Good afternoon, and thank you for joining today's second quarter conference call. Now over halfway through 2024, Capricor continued to make significant progress on bringing our lead asset, Deramiocel, which was previously referred to as CAP-1002, closer to the submission of a biologics license application or BLA, for the treatment of Duchenne muscular dystrophy as well as developing our pipeline of exosome products for clinical development. Our published results and two independently conducted clinical trials and the recent long-term open-label extension results, we believe are some of the strongest data seen in the space. As Deramiocel addresses the inflammation and fibrosis caused by the cell death due to the lack of dystrophin, which typically protects muscle cells from damage.

謝謝，阿傑。下午好，感謝您參加今天的第二季電話會議。2024 年已過半，Capricor 在將我們的主要資產 Deramiocel（之前稱為 CAP-1002）方面繼續取得重大進展，更接近於提交生物製劑許可申請或 BLA，用於治療杜氏肌肉營養不良症以及開髮用於臨床開發的外泌體產品管道。我們發布的結果、兩項獨立進行的臨床試驗以及最近的長期開放標籤擴展結果，我們相信是該領域看到的最有力的數據。Deramiocel 解決了缺乏肌肉營養不良蛋白而導致的細胞死亡引起的發炎和纖維化，而肌肉營養不良蛋白通常可以保護肌肉細胞免受損傷。

We believe it can serve as a potential backbone therapy for all boys and young men afflicted with DMD, with the product's ability to significantly slow disease progression from both a skeletal and cardiac standpoint. Additionally, as the KOLs across the DMD fields have continued to message, DMD will require a multidrug paradigm to address all of the pathological consequences of this disease.

我們相信，它可以作為所有患有 DMD 的男孩和年輕男性的潛在骨幹療法，從骨骼和心臟的角度來看，該產品能夠顯著減緩疾病進展。此外，隨著 DMD 領域的 KOL 不斷發出訊息，DMD 將需要多藥物範例來解決疾病的所有病理後果。

So I want to remind everyone that as Deramiocel move towards potential approval and commercialization, our United States distribution agreement with Nippon Shinyaku provides for approximately $700 million in potential developmental and sales milestones that may become due to Capricor, which would enable us to invest in our commercial launch and give us the resources to strategically expand our focus to other diseases of inflammation and fibrosis.

因此，我想提醒大家，隨著Deramiocel 走向潛在的批准和商業化，我們與Nippon Shinyaku 的美國分銷協議規定了大約7 億美元的潛在開發和銷售里程碑，這可能會歸功於Capricor，這將使我們能夠投資我們的產品商業發布，並為我們提供資源，策略性地將我們的重點擴展到其他發炎和纖維化疾病。

In fact, we are preparing to discuss opportunities to potentially expand Deramiocel into Becker muscular dystrophy with the FDA later this year. We believe in Deramiocel's potential to become a transformational treatment for DMD and beyond.

事實上，我們正​​準備在今年稍後與 FDA 討論將 Deramiocel 擴展到貝克爾肌肉營養不良症的機會。我們相信 Deramiocel 有潛力成為 DMD 及其他疾病的變革性治療方法。

And we are positioning Capricor to become a world-class commercial and R&D operation with a robust pipeline of opportunities for growth, including the expansion of our StealthX exosome-based platform. My goal is to position the organization to deliver for that transformation, and I continue to be proud of the progress that we have made to date.

我們將 Capricor 定位為世界一流的商業和研發企業，擁有強大的成長機會，包括擴展我們基於 StealthX 外泌體的平台。我的目標是讓組織能夠實現這一轉型，我仍然為我們迄今為止所取得的進展感到自豪。

I'd like to take a moment to update you on our recent three-year HOPE-2 Open-Label Extension or OLE study results, which were presented at this year's PPMD Annual Meeting. Treated subjects showed a statistically significant benefit with a p-value of 0.001 and an improvement in 3.7 PUL points on the PUL 2.0 when compared to an external comparator dataset of similar DMD patients.

我想花點時間向您介紹我們最近三年的 HOPE-2 開放標籤擴展或 OLE 研究結果，這些結果已在今年的 PPMD​​ 年會上公佈。與類似 DMD 患者的外部比較資料集相比，接受治療的受試者顯示出統計上顯著的益處，p 值為 0.001，PUL 2.0 提高了 3.7 個 PUL 點。

This translates into an approximate 50% delay in disease progression in treated patients versus the external comparator. Data also showed improvements in multiple cardiac measures, including left ventricular ejection fraction, as well as indexed left ventricular and left ventricular end systolic volume and just solid diastolic volumes.

這意味著與外部比較者相比，接受治療的患者的疾病進展延遲了約 50%。數據也顯示多項心臟指標的改善，包括左心室射血分數、指數左心室和左心室收縮末期容積以及固體舒張期容積。

These are measures of cardiac function and are considered highly relevant in terms of predicting long-term outcomes. In addition, greater improvements in cardiac function were observed in those patients that had ejection fractions greater than 45% at the beginning of the HOPE-2 randomized clinical trial. Currently, as you know, there are no approved treatments specifically for DMD cardiomyopathy.

這些是心臟功能的衡量標準，被認為與預測長期結果高度相關。此外，在 HOPE-2 隨機臨床試驗開始時，射血分數大於 45% 的患者的心臟功能得到了更大的改善。如您所知，目前尚無批准的專門針對 DMD 心肌病變的治療方法。

Our HOPE-2 Open Label Extension study continues to show a favorable safety profile, and we believe these three results continue to underscore the potential long-term and consistent benefits of Deramiocel for the treatment of DMD. Based on the favorable data from our clinical trials, I want to reiterate that our primary goal is to file a BLA as soon as possible, and we are actively working with FDA to accomplish that goal. Deramiocel is the only therapeutic to our knowledge that has shown statistically significant improvements in cardiac function in DMD associated cardiomyopathy, which is the leading cause of death for those with DMD.

我們的 HOPE-2 開放標籤擴展研究繼續顯示出良好的安全性，我們相信這三項結果繼續強調了 Deramiocel 對於治療 DMD 的潛在長期和一致益處。基於我們臨床試驗的有利數據，我想重申，我們的首要目標是盡快提交 BLA，我們正在積極與 FDA 合作以實現這一目標。據我們所知，Deramiocel 是唯一一種能夠顯著改善 DMD 相關心肌病變心臟功能的治療方法，而心肌病變是 DMD 患者死亡的主要原因。

FDA is aware of the strength of Capricor's cardiac data and may allow for its inclusion in the label for Deramiocel, which FDA acknowledges as important to the DMD patient community. We have just met with FDA in a very positive pre-BLA meeting.

FDA 意識到 Capricor 心臟數據的優勢，並可能允許將其納入 Deramiocel 的標籤中，FDA 承認 Deramiocel 對 DMD 患者群體很重要。我們剛剛與 FDA 舉行了一次非常積極的 BLA 前會議。

And while we are waiting final minutes from the meeting, I can share that we are discussing alternative paths to our BLA filing. One pathway would be filing our BLA with currently available data from the HOPE-2 and the HOPE-2 OLE studies to support an accelerated approval pathway with confirmatory data to be provided at a later date.

當我們等待會議最後幾分鐘時，我可以告訴大家，我們正在討論 BLA 備案的替代途徑。一種途徑是使用 HOPE-2 和 HOPE-2 OLE 研究中目前可用的資料提交 BLA，以支援加速審批途徑，並在稍後提供驗證性資料。

Another option is a traditional full approval. We are pleased to announce FDA's acceptance of our rolling BLA submission and based on that, Capricor intends to initiate the filing of our BLA shortly.

另一種選擇是傳統的完全批准。我們很高興地宣布 FDA 接受我們的滾動 BLA 提交，基於此，Capricor 打算很快開始提交我們的 BLA。

On the CMC front, as we reported last quarter, our San Diego manufacturing facility is fully operational and actively preparing for commercial runs necessary for the filing. Additionally, we have accelerated the pace of our preparations for commercial market entry. Our partner, Nippon Shinyaku, is focused on their role in getting Deramiocel ready for launch. We and they expect a rapid adoption of Deramiocel by payers and patients with DMD.

在 CMC 方面，正如我們上季度報告的那樣，我們的聖地亞哥製造工廠已全面投入運營，並積極為備案所需的商業運行做準備。此外，我們也加快了商業市場准入的準備步伐。我們的合作夥伴 Nippon Shinyaku 專注於為 Deramiocel 的推出做好準備。我們和他們預期 Deramiocel 會被付款人和 DMD 患者迅速採用。

We are actively engaged with them to ensure a smooth and successful launch. Capircor is focused on the areas of preparation for market access, reimbursement, as well as opportunities for expanded label usage. Now for an update on our exosome program. While our primary focus is on advancing Deramiocel, we are committed to our StealthX exosome platform technology as part of our next-generation drug delivery platform.

我們正在積極與他們合作，以確保順利、成功地啟動。Capircor 專注於市場准入、報銷以及擴大標籤使用機會的準備領域。現在我們的外泌體計劃的最新情況。雖然我們的主要重點是推動 Deramiocel，但我們致力於將 StealthX 外泌體平台技術作為我們下一代藥物傳輸平台的一部分。

We have been planning for the success of Deramiocel for several years and to that end has built a pipeline of engineered exosome products based on our foundational cell therapy experience. The goal has been to take advantage of our experience of scaling up and out cell therapies as well as to continue to refine cell-based products such as exosomes, in terms of their ability to be quantified based on mechanism of action as well as targeting them for more strategic treatment paradigm.

多年來，我們一直在計劃 Deramiocel 的成功，並為此根據我們的基礎細胞治療經驗建立了一系列工程外泌體產品。我們的目標是利用我們擴大和縮小細胞療法的經驗，並繼續完善外泌體等基於細胞的產品，使其能夠根據作用機制進行量化以及靶向它們以獲得更具策略性的治療範例。

Our exosome platform focuses on the use of our proprietary StealthX technology to develop therapeutics by harnessing exosomes as delivery vehicles. This program requires the loading of specific cargoes into the exosomes and then attaching targeting moieties on the outside of the exosome to direct it where to go. We are currently ramping up our business development efforts with our exosome platform technology as we continue to develop the therapeutic opportunities of the platform.

我們的外泌體平台專注於使用我們專有的 StealthX 技術，透過利用外泌體作為遞送載體來開發治療藥物。該程序需要將特定的貨物裝載到外泌體中，然後將靶向部分附著在外泌體的外部以引導其去向。隨著我們繼續開發該平台的治療機會，我們目前正在利用我們的外泌體平台技術加強業務開發。

Regarding our vaccine candidate, we are actively collaborating with the United States government's Project NextGen, which aims to test vaccine candidate for COVID-19 prevention and also to prepare for future pandemics. Currently, our vaccine candidate is in the manufacturing phase with plans to deliver to the National Institutes of Allergy and Infectious Diseases by the end of 2024. Then NIAID will conduct and fully fund a Phase 1 clinical trial.

關於我們的候選疫苗，我們正在與美國政府的 NextGen 計畫積極合作，該計畫旨在測試預防 COVID-19 的候選疫苗，並為未來的大流行做好準備。目前，我們的候選疫苗正處於生產階段，並計劃於 2024 年底交付給美國國家過敏和傳染病研究所。然後 NIAID 將進行並全額資助一期臨床試驗。

On the therapeutic front, we have recently made notable advancements with our exosome platform, which is in preclinical development.

在治療方面，我們的外泌體平台最近取得了顯著進展，該平台正處於臨床前開發階段。

We presented data at the International Society for Cell & Gene Therapy in Vancouver, where we were selected for oral presentation. Our preclinical data highlighted an exosome based approach for targeted delivery to skeletal muscle in the mouse lower limb. Subsequently, our research demonstrated that using an in-house developed proprietary exogenous loading technique, we can not only target exosomes to muscle cells, but also deliver antisense oligonucleotide, one of our payloads, with exosomes carrying a muscle targeting moiety.

我們在溫哥華國際細胞與基因治療學會上展示了數據，並被選中進行口頭展示。我們的臨床前數據強調了一種基於外泌體的方法，用於靶向遞送至小鼠下肢的骨骼肌。隨後，我們的研究表明，使用內部開發的專有外源加載技術，我們不僅可以將外泌體靶向肌肉細胞，還可以傳遞反義寡核苷酸（我們的有效負載之一），外泌體攜帶肌肉標靶部分。

This work was featured in a poster presentation at the International Society for Extracellular Vesicles in Melbourne, Australia. The data from both conferences strongly suggests that exosomes are targeting moeities can direct delivery to skeletal muscle and the mouse lower limbs, presenting potential for targeted delivery applications, including the treatment of Duchenne muscular dystrophy, combined with early data on Arginase-1 deficiency, which is being developed as a potential enzyme replacement therapy. These findings reinforce the potential of our platform.

這項工作在澳洲墨爾本國際細胞外囊泡學會的海報展示中得到了展示。兩次會議的數據強烈表明，外泌體的靶向部分可以直接遞送至骨骼肌和小鼠下肢，這呈現出靶向遞送應用的潛力，包括治療杜氏肌肉營養不良症，結合精氨酸酶- 1缺乏症的早期數據，正在開發作為一種潛在的酵素替代療法。這些發現增強了我們平台的潛力。

We believe there are significant opportunities for broad applications, and our data from our StealthX program continues to support this belief. I would like to take a few moments to provide our current corporate updates and priorities as we progress through 2024. Based on our significant progress over the last quarter and now a potential expedited path to BLA.

我們相信，存在廣泛應用的重大機會，而我們來自 StealthX 計劃的數據也繼續支持這一信念。我想花一些時間介紹一下我們在 2024 年取得進展時當前的公司最新情況和優先事項。基於我們在上個季度取得的重大進展以及現在可能加快 BLA 的路徑。

This clarity gives us confidence that we are well-positioned to bring additional capital into Capricor, to strengthen our balance sheet and extend our cash runway While there are various ways to accomplish this goal, our primary mission is to secure capital in the best interest of our shareholders.

這種清晰度使我們相信，我們有能力為Capricor 帶來更多資本，以加強我們的資產負債表並擴大我們的現金跑道。確保資本符合Capricor 的最佳利益。

Therefore, we are pursuing multiple different business opportunity, business development opportunities. And we are in active discussions with several parties looking to potentially distribute Deramiocel in Europe for the treatment of DMD. We have been judicious with respect to partnering for the rights and the EMA as we knew the asset would garner greater value as we progress through clinical development and achieved clarity with FDA. I am very pleased to inform you that we are late stage discussions for these rights and we will share updates as they become available.

因此，我們正在追求多個不同的商機、業務發展機會。我們正在與一些希望在歐洲銷售 Deramiocel 來治療 DMD 的各方進行積極討論。我們在權利和 EMA 的合作方面一直非常明智，因為我們知道，隨著我們在臨床開發中取得進展並與 FDA 達成澄清，該資產將獲得更大的價值。我很高興地通知您，我們正在對這些權利進行後期討論，我們將在更新可用時分享。

We believe that Deramiocel is a highly valuable asset in the EU as well as other regions around the world, and our partnering efforts remain focused on securing these relationships.

我們相信 Deramiocel 在歐盟以及世界其他地區是一項非常有價值的資產，我們的合作努力仍然集中在確保這些關係上。

Additionally, our U.S. agreement with Nippon Shinyaku, as I mentioned a moment ago, has approximately $700 million in potential milestone payments payable to Capricor, over $90 million of which is up to the time of approval and are triggered upon certain regulatory based achievements, some of them to be expected in the near term.

此外，正如我剛才提到的，我們與Nippon Shinyaku 的美國協議有大約7 億美元的潛在里程碑付款將支付給Capricor，其中超過9,000 萬美元在批准時會被觸發，並且是在某些基於監管的成就時觸發的，有些其中預計在短期內。

We believe that we can achieve these milestones as they will continue to support our balance sheet and further extend our cash runway. Furthermore, if we receive approval for Deramiocel, we will be eligible to receive a Priority Review Voucher, a PRV based on our pediatric disease designation to which we retain full rights. And finally, we were pleased to announce earlier this quarter to be added to the Russell 2000 and Russell 3,000 indices, which should bring added visibility to our company as we continue to remain focused on bringing more institutional buy and sell-side support to our story.

我們相信我們能夠實現這些里程碑，因為它們將繼續支持我們的資產負債表並進一步擴大我們的現金跑道。此外，如果我們獲得 Deramiocel 的批准，我們將有資格獲得優先審查券，這是基於我們的兒科疾病指定的 PRV，我們保留對此的全部權利。最後，我們很高興地在本季早些時候宣布將其納入羅素2000 和羅素3,000 指數，這將為我們公司帶來更高的知名度，因為我們將繼續專注於為我們的故事帶來更多機構買方和賣方支持。

In conclusion, this has been an extraordinary year for Capircor thus far, we have made significant strides in our regulatory pathway, getting closer to potential approval with each FDA meeting. Patients and families are fully supportive of our efforts and leave a lasting power of Deramiocel and helping those of DMD to feel and function better.

總而言之，到目前為止，今年對 Capircor 來說是不平凡的一年，我們在監管途徑上取得了重大進展，在每次 FDA 會議上都更接近潛在的批准。患者和家屬全力支持我們的努力，並留下 Deramiocel 的持久力量，幫助 DMD 患者感覺更好，功能更好。

We have built, equipped and are now producing doses from our manufacturing facility in San Diego for a fraction of the costs of other biotechnology companies. We have some of the strongest and most consistent data in terms of efficacy and safety in the DMD community with the hope to and hope to open label extension data sets.

我們已經在聖地牙哥的製造工廠建造、裝備並生產劑量，而成本只是其他生技公司的一小部分。我們在 DMD 社群的功效和安全性方面擁有一些最強大和最一致的數據，希望並希望開放標籤擴展資料集。

We have a strong pipeline in our engineered exosome technology, supported by StealthX. Capricor's goal is to continue to meet its milestones and deliverables as we have set forth. As we continue to focus our efforts on bringing Deramiocel towards potential commercialization and are investing judiciously across the organization to prepare for that endeavor. Over the next several months, we will be presenting at various medical scientific and industrial related conferences, details of which will be shared as they become available.

我們在工程外泌體技術方面擁有強大的研發管線，並得到 StealthX 的支援。Capricor 的目標是繼續實現我們設定的里程碑和可交付成果。我們將繼續專注於將 Deramiocel 推向潛在的商業化，並在整個組織中進行明智的投資，為這項努力做好準備。在接下來的幾個月中，我們將在各種醫學科學和工業相關會議上發表演講，詳細資訊將在發布後分享。

And of course, finally, I want to thank the patients, their families, and all of our investors for their continued support. Later this quarter, we plan to announce further details from our pre-BLA meeting, at which time we will be able to articulate more formal guidance on the upcoming pivotal milestones of our DMD program. I will now turn the call over to AJ to run through our financials.

當然，最後，我要感謝患者、他們的家人以及我們所有投資者的持續支持。本季度晚些時候，我們計劃宣布 BLA 前會議的更多細節，屆時我們將能夠就 DMD 計劃即將到來的關鍵里程碑提出更正式的指導。我現在將把電話轉給 AJ 來了解我們的財務狀況。

Thanks, Linda. This afternoon's press release provided a summary of our second quarter 2024 financials on a GAAP basis. You may also refer to our quarterly report on Form 10 Q, which we expect to become available shortly and will be accessible on the SEC website as well as the financial section of our website.

謝謝，琳達。今天下午的新聞稿提供了我們按 GAAP 計算的 2024 年第二季財務狀況摘要。您也可以參考我們的 10 Q 表格季度報告，我們預計很快就會提供該報告，您可以在 SEC 網站以及我們網站的財務部分存取該報告。

Let me start with our cash position. As of June 30, 2024, the company's cash, cash equivalents and marketable securities totaled approximately $29.5 million compared to approximately $39.5 million on December 31st, 2023.

讓我從我們的現金狀況開始。截至2024年6月30日，該公司的現金、現金等價物及有價證券總額約為2,950萬美元，而2023年12月31日約為3,950萬美元。

Based on our current operating plan and projections, company's cash runway is expected to be sufficient to support operations into the first quarter of 2025. This expectation excludes any potential additional milestone payments under our commercialization and distribution agreements with Nippon Shinyaku.

根據我們目前的營運計劃和預測，該公司的現金跑道預計將足以支持 2025 年第一季的營運。這一預期不包括我們與 Nippon Shinyaku 的商業化和分銷協議下任何潛在的額外里程碑付款。

Turning now to the financials. Revenue for the second quarter of 2024 for approximately $4 million compared with approximately $3.9 million for the second quarter of 2023. Source of revenue is the ratable recognition of the $40 million we have received from our US exclusive commercialization and distribution agreement with Nippon Shinyaku.

現在轉向財務狀況。2024 年第二季的營收約為 400 萬美元，而 2023 年第二季的營收約為 390 萬美元。收入來源是我們從與 Nippon Shinyaku 的美國獨家商業化和分銷協議中獲得的 4,000 萬美元的應得認可。

Moving to our operating expenses for the second quarter of 2024, excluding stock-based compensation, our R&D expense was approximately $11.7 million compared to approximately $8.4 million in Q2 2023.

說到 2024 年第二季的營運費用，不包括股票薪酬，我們的研發費用約為 1,170 萬美元，而 2023 年第二季約為 840 萬美元。

The increase in expenses of $3.3 million was primarily due to increased clinical and manufacturing costs associated with our Duchenne muscular dystrophy program. Again, excluding stock-based compensation, our general and administrative expense was approximately$ 1.8 million in Q2 2024 and approximately $1.7 million in Q2 2023.

費用增加 330 萬美元主要是由於與杜氏肌肉營養不良計畫相關的臨床和製造成本增加。同樣，不包括股票薪酬，我們 2024 年第二季度的一般和管理費用約為 180 萬美元，2023 年第二季度約為 170 萬美元。

Net loss for the second quarter 2024 was approximately $11 million, compared to a net loss of approximately $7.4 million for the second quarter of 2023. And net loss for the first half of 2024 was approximately $20.8 million compared to a net loss of approximately $15.1 million for the first half of 2023. We will now open the line up for questions.

2024 年第二季的淨虧損約為 1,100 萬美元，而 2023 年第二季的淨虧損約為 740 萬美元。2024 年上半年的淨虧損約為 2,080 萬美元，而 2023 年上半年的淨虧損約為 1,510 萬美元。我們現在開始排隊提問。

(Operator Instructions)

（操作員說明）

And your first question will be from Joseph Pantginis of H.C. Wainwright. Please go ahead.

你的第一個問題將來自 H.C. 的 Joseph Pantginis。溫賴特。請繼續。

Good afternoon, Linda and AJ, and thanks for taking the questions. Two questions, if you don't mind. So Linda, in your prepared comments, you're talking about you just met with the FDA and it sounds like the key aspects were deciding on the two alternate path for filing.

下午好，Linda 和 AJ，感謝您提出問題。如果你不介意的話，有兩個問題。琳達，在您準備好的評論中，您談論的是您剛剛與 FDA 會面，聽起來關鍵方面是決定兩種替代申請路徑。

I guess I wanted to get the latest update. And of course, I know this is before you get the minutes is to what extent did the cardiovascular play into this latest meeting? And what do you consider the key outstanding questions or issues still?

我想我想獲得最新的更新。當然，我知道在你拿到會議記錄之前，心血管在這次最近的會議中扮演了多大的角色？您認為仍有哪些關鍵的懸而未決的問題或問題？

Yes, Joe, thank you and thank you for your support over these many years. I really think we're going to get this across the line, and I'm super excited. The cardiovascular aspect of the program is finally getting the attention that it deserves from the FDA. I think that's really due to the combination of efforts of the KOLs and addition advocacy community that I've highlighted that this is the number one cause of death in these boys and young men.

是的，喬，謝謝你，謝謝你多年來的支持。我真的認為我們會成功，我非常興奮。該計劃的心血管方面終於得到了 FDA 應有的關注。我認為這實際上要歸功於 KOL 和其他倡導社區的共同努力，我強調這是這些男孩和年輕人的頭號死因。

And yet frankly, all the therapies that are available to them are ones that might go to, you know, some person has adult based cardiac disease that kind of joking, something your brand I might get this would be the first therapeutic to treat the cardiomyopathy disease associate with Duchenne muscular dystrophy, which pathogenesis is extraordinarily different from others.

但坦白說，他們可以使用的所有療法都可能會用到，你知道，有些人患有成人心臟病，就像開玩笑一樣，你的品牌我可能會得到這將是第一個治療心肌病的療法此疾病與杜氏肌肉營養不良症相關，其發病機制與其他疾病截然不同。

So yes, it has become a key focus of the program. They are willing to consider Deramiocel as part of the label for cardiac.

所以，是的，它已成為該計劃的重點。他們願意將 Deramiocel 視為心臟藥物標籤的一部分。

They're also at least in writing thus far willing to consider expanding the label to all patients diagnosed with Duchenne muscular dystrophy. And so we're really excited to see the results of these minutes and continue to push this towards BLA very quickly.

到目前為止，他們還至少以書面形式願意考慮將標籤擴大到所有診斷為杜氏肌肉營養不良症的患者。因此，我們非常高興看到這幾分鐘的結果，並繼續快速推動 BLA 的發展。

That's very helpful. Thanks. And if I could just switch swiftly to the exosome program. Obviously it's a platform that has a broad-reaching capability.

這非常有幫助。謝謝。如果我能迅速切換到外泌體計畫就好了。顯然，這是一個具有廣泛功能的平台。

So you mentioned your BD efforts. I guess how would you describe sort of the proportion of, say, inbound versus outbound from reaching with regard to BD?

所以你提到了你的BD工作。我想您會如何描述 BD 的入站與出站比例？

Yes. So the exosome program is really coming to light. I think, you know and others that follow our story know that my goal is to launch Deramiocel and expand that into other indications as appropriate. But because of our long experience with cell therapy and making cells and using cells, expanding into an Exosome based program was critical.

是的。因此，外泌體計劃真正曝光了。我想，您和其他關注我們故事的人都知道，我的目標是推出 Deramiocel 並將其擴展到其他適當的適應症。但由於我們在細胞治療、製造細胞和使用細胞方面的長期經驗，擴展到基於外泌體的計畫至關重要。

And the first thing that we had to do was make sure that we could make exosomes in large numbers easily and relatively cheaply because their main competitor would be a lipid nanoparticle.

我們要做的第一件事是確保我們能夠輕鬆且相對便宜地大量製造外泌體，因為它們的主要競爭對手是脂質奈米顆粒。

Now exosomes are way better than lipid nanoparticles because you can target them and load them. And so there's a lot more opportunities, plus they're very safe and not clogging up your liver and so that was our first goal.

現在，外泌體比脂質奈米顆粒要好得多，因為您可以靶向它們並加載它們。所以有更多的機會，而且它們非常安全，不會堵塞你的肝臟，所以這是我們的第一個目標。

We achieved that and or people thought we were quiet for a long time. Now that we're getting out and about with some of these opportunities including potentially loading, for instance, a Duchenne-based antisense and exon skipper into the exosome, targeting it to skeletal muscle using our targeting moiety.

我們實現了這一目標，或者人們認為我們已經安靜了很長一段時間。現在我們正在探索其中的一些機會，包括可能將基於杜興的反義和外顯子跳躍者加載到外泌體中，使用我們的靶向部分將其靶向骨骼肌。

This becomes now a very tangible product. And so yes, we have more incoming interest than we've had before. We're also planning in 25, a pretty extensive business development external, reaching effort.

現在這已成為一種非常有形的產品。所以，是的，我們比以前有更多的興趣。我們也計劃在25年，進行相當廣泛的外部業務開發，以達成努力。

Thank you very much for the color, Linda.

非常感謝你的顏色，琳達。

Absolutely, Joe. Look forward to seeing you soon.

絕對可以，喬。期待很快見到您。

Thank you.

謝謝。

Next question will be from Leland Gershell of Oppenheimer. Please go ahead.

下一個問題將由奧本海默的利蘭·格謝爾提出。請繼續。

Yes, hi.

是的，嗨。

Thanks for taking the questions.

感謝您提出問題。

Just hoping to get some clarity on the timelines here. You mentioned the pre-BLA meeting and quorum plans that will be coming up from around the data and thereafter, I want to understand you know, have you begun any aspects of the rolling submission? If not, when do you expect to begin that and how long after that top line data from the HOPE-3 study we expect to complete the rolling BLA submission?

只是希望能在這裡弄清楚時間表。您提到了 BLA 前會議和法定人數計劃，這些計劃將從數據中提出，此後，我想了解您是否已經開始滾動提交的任何方面？如果沒有，您預計什麼時候開始？

Yes. So our current plan is to begin submitting the rolling BLA within the next 60 days. FDA has seen our timelines and have approved it as part of our communication plan in our most recent meeting. Shoule the HOPE-3 data would be necessary for the BLA, we would anticipate the submission to be complete at the end of the first quarter. We are also exploring, however, with the agency the opportunity to complete the file without the HOPE-3 data. And then that would obviously accelerate submission to the end of 2024.

是的。因此，我們目前的計劃是在未來 60 天內開始提交滾動 BLA。FDA 已經看到了我們的時間表，並在最近的會議上批准了它作為我們溝通計劃的一部分。如果 BLA 需要 HOPE-3 數據，我們預計提交將在第一季末完成。不過，我們也正在與該機構探討是否有機會在沒有 HOPE-3 數據的情況下完成文件。那麼這顯然會加速到 2024 年底提交。

Okay, thank you.

好的，謝謝。

And then a question on the on the ex-US potential partnership. Wondering if there's any, based on where you may be in your discussions, if you can make any comment as to on the the view of having one or more partnerships in place by year end, just sort of want to understand kind of what the evolution of those discussions will lead to perform from here.

然後是關於美國以外的潛在夥伴關係的問題。想知道根據您在討論中的情況，您是否可以對年底前建立一個或多個合作夥伴關係的觀點發表任何評論，只是想了解一下演變情況這些討論將導致從這裡開始執行。

Our goal is to finalize those conversations before the end of this year. As I said, I know people have sort of wondered, you know, we've been talking to EU partners for a while. The clarity was FDA has been critical and also beginning conversations with regulatory authorities and consultants in Europe.

我們的目標是在今年年底前完成這些對話。正如我所說，我知道人們有點想知道，我們已經與歐盟夥伴交談了一段時間。明確的是，FDA 一直至關重要，並開始與歐洲監管機構和顧問進行對話。

We now have a much cleaner picture of where it's going to take to get it across the line there. We think it's going to be actually much easier than we originally anticipated. Our partners think so also, so the goal would be to close these deal before the end of this calendar year to strengthen the balance sheet and to support the approval and expansion for Deramiocel.

我們現在對如何跨越界線有了更清晰的了解。我們認為這實際上比我們最初預期的要容易得多。我們的合作夥伴也這麼認為，因此我們的目標是在今年年底前完成這些交易，以加強資產負債表並支持 Deramiocel 的批准和擴張。

Thank you.

謝謝。

Yes, great talking to you always, Leland.

是的，總是很高興與你交談，利蘭。

Thank you.

謝謝。

Next question will be from Aydin Huseynov at Ladenburg. Please go ahead.

下一個問題將由拉登堡的 Aydin Huseynov 提出。請繼續。

Hi.

你好。

Good afternoon, everyone, and congrats with the progress. A positive FDA meeting. Discussion and finding another name for CAP-1002 with Deramiocel. So a couple of questions regarding the FDA meeting. So you mentioned that you had some discussions about expansion of the indication. And I was just curious to see if there is any preliminary feedback or reaction of FDA to potential Becker dystrophy expansion and any thoughts that they have, any hypothetical DMD trial design?

大家下午好，祝賀取得的進展。一次積極的 FDA 會議。與 Deramiocel 討論並找到 CAP-1002 的另一個名稱。關於 FDA 會議的幾個問題。所以你提到你們對擴大適應症進行了一些討論。我只是好奇 FDA 對潛在的 Becker 營養不良擴張是否有任何初步反饋或反應，以及他們有什麼想法，有任何假設的 DMD 試驗設計嗎？

Yes, Aydin, thank you so much for the questions. I know you've been really a big supporter of us expanding into DMD for awhile. So thank you for that guidance.

是的，艾丁，非常感謝你的提問。我知道一段時間以來，您一直是我們向 DMD 擴張的堅定支持者。所以感謝您的指導。

We have mentioned that as part of our conversations with FDA. Obviously, it hasn't been a main focus on the meeting. So really exciting part for us right now is that FDA is beginning to understand the impact of cardiomyopathies in these muscular dystrophies, it really gives us a window into how we can move forward.

作為我們與 FDA 對話的一部分，我們已經提到了這一點。顯然，這並不是這次會議的主要焦點。現在對我們來說真正令人興奮的是 FDA 開始了解心肌病變對這些肌肉營養不良症的影響，這確實為我們提供了一個了解如何前進的窗口。

And for instance, Becker where the cardiomyopathy is also the leading cause of death. We haven't really talked about trial design yet because I want to get through this stage with FDA, see where it comes down, cardiac implications and then strategically build a program. We plan to meet with FDA on DMD before the end of the year so that we can get their guidance and we're putting together sort of our KOL panels and plans as we speak. So yes, this is a great opportunity, obviously, for the therapy, but also for the patients.

例如，貝克爾的心肌病變也是導致死亡的主要原因。我們還沒有真正討論過試驗設計，因為我想與 FDA 一起度過這個階段，看看它會發生什麼，對心臟的影響，然後策略性地制定一個計劃。我們計劃在今年年底前與 FDA 就 DMD 問題進行會面，以便獲得他們的指導，並且我們正在整理我們的 KOL 小組和計劃。所以，是的，這顯然對於治療來說是一個很好的機會，對患者來說也是如此。

And I still think that this is helpful for full potential DMD label for very myself. How we should think about the label, do you think it would be pretty broad label that would include Pinon on DMD patients are similar to the ones you recruited and hoped to and hopefully trial? Or do you think the FDA may sort of specify patients with cardiomyopathy only? And how to how do you think the FDA will approach?

我仍然認為這對於我自己的 DMD 標籤的全部潛力很有幫助。我們應該如何考慮這個標籤，您認為這是一個相當廣泛的標籤，其中包括皮農 DMD 患者，與您招募、希望和希望試驗的患者相似嗎？或者您認為 FDA 可能只指定心肌病變的患者？您認為 FDA 將如何處理？

Yes. So the cardiomyopathy is going to be and or? So that's how we positioned it. I think that's how the label will read. It will be for those with skeletal muscle and cardiac myopathy and or for skeletal muscle or cardiomyopathy because as you know, and has been well published, the diseases are not progressed similarly, both in cardiac and skeletal muscle. So some kids might have a bad heart and skeletal muscle or vice versa. So we will be an opportunity for stabilization of disease, both in skeletal and cardiac. In terms of labeling, our goal is all those diagnose attrition, muscular dystrophy.

是的。那麼心肌病變將是和或？這就是我們的定位。我想這就是標籤的讀法。它將適用於那些患有骨骼肌和心肌病變和/或骨骼肌或心肌病變的人，因為正如您所知，並且已經廣泛發表，這些疾病在心肌和骨骼肌中的進展並不相似。因此，有些孩子的心臟和骨骼肌可能較差，反之亦然。因此，我們將成為穩定骨骼和心臟疾病的機會。在標籤方面，我們的目標是所有診斷消耗、肌肉萎縮症的人。

Our preliminary feedback from FDA suggested they are open to that opportunity and we will certainly push that as hard as we can.

FDA 的初步回饋表明他們對這個機會持開放態度，我們肯定會盡最大努力推動這一機會。

Certainly the precedent that has been set by some of the others that have gotten approval in the space with very broad labels gives us a door open into that opportunity as well, which could double our market opportunity at launch,

當然，其他一些在該領域獲得批准的先例也為我們打開了通往這一機會的大門，這可能會使我們在推出時的市場機會加倍，

Alright. Great, makes sense. Regarding the -- sort of advanced discussions regarding partnerships in Europe, so would you like to pursue a partnership deal before the top line readout in the fourth quarter 2024 or after the readout? So this is purely economic question, is that cash now or more cash later?

好吧。太好了，有道理。關於歐洲合作夥伴關係的高級討論，您想在 2024 年第四季營收公佈之前還是在公佈之後尋求合作協議？所以這純粹是經濟問題，現在是現金還是以後更多現金？

Well, you know, it's an interesting question. We're talking to partners right now. We feel that the offers that are coming our way are reasonable and appropriate, almost assuming approval of the BLA. So Europe has another regulatory structure, and we're very confident now based on some of our conversations with authorities over there that we will meet their criteria either way small clinical trial or no clinical trial. We're not sure yet and so I guess the short answer to your question is we'll close the deal when we have reached the best economics to support our balance sheet to drive the company forward and also to give credit to the asset as it is due.

嗯，你知道，這是一個有趣的問題。我們現在正在與合作夥伴交談。我們認為，我們收到的報價是合理且適當的，幾乎假設 BLA 已獲得批准。因此，歐洲有另一個監管結構，根據我們與當地當局的一些對話，我們現在非常有信心，我們將透過小型臨床試驗或不進行臨床試驗來滿足他們的標準。我們還不確定，所以我想對你的問題的簡短回答是，當我們達到最佳的經濟效益來支持我們的資產負債表以推動公司前進並給予資產信用時，我們將完成交易到期了。

Got it. And the last question I have is regarding the potential milestones from Nippon Shinyaku. So we know there's $90 million of waiting up until BLA approval, but are you -- should we expect anything before the FDA approval, should we expect anything based on the top line readout at the end of this year?

知道了。我的最後一個問題是關於日本新藥的潛在里程碑。因此，我們知道在 BLA 批准之前還有 9000 萬美元的等待時間，但是您是否 - 在 FDA 批准之前我們是否應該期待任何事情，我們是否應該根據今年年底的頂線讀數期待任何事情？

So as you know, we're not able to disclose the actual cadence of the milestone payments. We built them into our personal balance sheet estimations.

如您所知，我們無法透露里程碑付款的實際節奏。我們將它們納入我們的個人資產負債表估算中。

And so we're aware of those milestones come in. And that's really all I can say right now, but there's $90 million coming up to and including approval of the BLA.

所以我們知道這些里程碑的到來。我現在能說的就是這些，但 BLA 的批准還需要 9,000 萬美元。

Got it. Understood. All right. Thank you so much for taking my questions and congrats with the progress.

知道了。明白了。好的。非常感謝您回答我的問題並祝賀我的進展。

Thank you.

謝謝。

Look forward to seeing you soon.

期待很快見到您。

Thank you. (Operator Instructions)

謝謝。（操作員說明）

And your next question will be from Kristen Kluska at Cantor. Please go ahead.

您的下一個問題將來自 Cantor 的 Kristen Kluska。請繼續。

Hello.

你好。

This is Rick Miller on for Christian. Thanks for taking our questions.

這是克里斯蒂安的里克·米勒。感謝您回答我們的問題。

We've got two for you here.

我們這裡有兩個給你。

What are you hearing from physicians and caregivers about the potential for non-ambulant DMD patients now receiving gene therapy on an accelerated basis? How confident is the space in this, especially as it relates to these cardiac issues that you were talking about?

您從醫生和護理人員那裡得知，非流動性 DMD 患者現在加速接受基因治療的潛力如何？對此的信心有多大，特別是因為它與您所談論的這些心臟問題有關？

Yes. Really good question. So yes, I mean, everybody is laser focused on the implications of gene therapy for the non-ambulant and whether a will actually work, you know, are you knocking on a closed door that muscle is a dead muscle or are you going to really you know, preserve what's there. And I certainly hope for those guys with DMD, it preserves what's there.

是的。真是個好問題。所以，是的，我的意思是，每個人都高度關注基因療法對非行走者的影響，以及基因療法是否真的有效，你知道，你是在敲一扇關閉的門，肌肉是死肌肉，還是你真的會這麼做？我當然希望對於那些患有 DMD 的人來說，它可以保留那裡的東西。

We actually presented to FDA a comparison of Deramiocel and Elevidys and its pathogenesis or delay in the pathogenesis of the disease. And we were able to show that Deramiocel performs at least as well as gene therapy in year one and better than gene therapy in year two when compared with non-ambulant patients.

我們實際上向 FDA 提交了 Deramiocel 和 Elevidys 及其發病機製或延遲疾病發病機制的比較。我們能夠證明，與非流動患者相比，Deramiocel 在第一年的效果至少與基因療法一樣好，在第二年的效果優於基因治療。

The cardiomyopathy becomes a real problem with post-gene therapy patients for multiple reasons. One, physicians are concerned about potential inflammatory processes as part of the gene therapy and could that have negative implications on the heart. Two, have these guys are more active as I can put more strain on their hearts and will their hearts fill faster.

由於多種原因，心肌病變成為基因治療後患者的一個真正問題。第一，醫生擔心基因治療中潛在的發炎過程可能會對心臟產生負面影響。第二，讓這些人更活躍，因為我可以給他們的心臟更大的壓力，讓他們的心臟更快地充滿。

And three, there seems to be sort of a correlation between gene therapy and and somewhat worsening in the cardiomyopathy. So I think this is a great opportunity for Deramiocel, we have shown preclinically that there's no negative impact of Deramiocel in treatment conjunction with our microdystrophin treatment. And so we believe that there will be great partners together with the gene therapy, restore the muscle, and let Deramiocel protect it.

第三，基因治療和心肌病變的惡化之間似乎存在某種相關性。所以我認為這對 Deramiocel 來說是一個很好的機會，我們已經在臨床前證明 Deramiocel 與我們的微肌營養不良蛋白治療結合使用不會產生負面影響。所以我們相信，將會有偉大的合作夥伴與基因療法一起，恢復肌肉，並讓 Deramiocel 保護它。

Okay.

好的。

Maybe just one more question then from us. Switching to Becker, can you help us kind of frame the cardiomyopathy opportunity that you're thinking about here. I think sometimes the field thinks sort of naively about Becker as being a less severe form of DMD. So how does the kind of cardiac burden and Becker break down as it relates to DMD? Is it sort of more proportional or more severe? Thoughts there?

也許我們還有一個問題。轉向貝克爾，你能幫助我們建構你在這裡考慮的心肌病變機會嗎？我認為有時該領域天真地將貝克爾視為一種不太嚴重的 DMD 形式。那麼，與 DMD 相關的心臟負擔和 Becker 是如何分解的呢？是比例更大還是更嚴重？想法在那裡？

Yes, great question. And certainly, one that the KOLs, cardiologists basically laser focusing on according to some of the key opinion leaders that we've talked to.

是的，很好的問題。當然，根據我們採訪過的一些關鍵意見領袖的說法，KOL、心臟科醫生基本上都高度關注這個問題。

We participate actively and PPMDs cardiomyopathy meetings, which they hold once a year for both Becker and Duchenne. The Becker cardiomyopathy actually begins at about age 14, not so on similar, I guess similar to the Duchenne muscular dystrophy cardiomyopathy, the progression of skeletal muscle myopathy is slower.

我們積極參加 PPMD​​ 的心肌病變會議，他們每年為 Becker 和 Duchenne 舉辦一次會議。貝克爾心肌病變其實是在14歲左右開始的，不是那麼相似，我猜和杜氏肌肉營養不良症心肌病變類似，骨骼肌肌病變的進展速度較慢。

These guys end up with a pretty severe cardiomyopathy heart failure paradigm and most of them die in their 40s and 50s as a result of the heart disease. I think now that the whole field is becoming more aware of the cardiac dysfunction as a mediator of potential death, it'll open the door. As I mentioned in a question earlier to treating the cardiomyopathy associated with Becker.

這些人最終患上了相當嚴重的心肌病變心臟衰竭，他們中的大多數人在 40 多歲和 50 多歲的時候就死於心臟病。我認為現在整個領域都越來越意識到心臟功能障礙是潛在死亡的中介因素，這將為我們打開大門。正如我之前在一個問題中提到的治療與貝克爾相關的心肌病變。

And yes, you know, well, it's less severe in terms of dying at 20 versus dying of 50. Nobody wants to die at 50 either, so let's keep those guys go into.

是的，你知道，20 歲死亡比 50 歲死亡輕。也沒有人願意在 50 歲時死去，所以讓我們讓這些人繼續研究。

Okay.

好的。

Thank you so much.

太感謝了。

Thank you so much.

太感謝了。

Thank you. And at this time, I would like to turn the call back over to Capricor management for final thoughts.

謝謝。此時，我想將電話轉回 Capricor 管理層以徵求最終想法。

Thank you very much for attending our earnings call today, and thank you for your thoughtful questions, and I look forward to updating all of you on our programs as we continue through 2024. Have a nice evening and we'll talk to you soon.

非常感謝您參加今天的財報電話會議，也感謝您提出深思熟慮的問題，我期待著在 2024 年繼續進行的過程中向大家通報我們計劃的最新情況。祝您有個愉快的夜晚，我們很快就會與您聯繫。

Thank you.

謝謝。

Ladies and gentlemen, this does indeed conclude your conference call for today. Once again, thank you for attending and at this time, we do ask that you please disconnect your lines.

女士們、先生們，今天的電話會議到此結束。再次感謝您的出席，此時，我們懇請您斷開線路。