Capricor Therapeutics, Inc. (CAPR) 2026 Q2 法說會逐字稿

Good afternoon ladies and gentlemen and welcome to the Capricor Therapeutics second quarter 2026 Conference Call. (Operator Instructions) The call is being recorded on Thursday, August 13, 2026.

各位女士、各位先生，下午好，歡迎參加 Capricor Therapeutics 2026 年第二季電話會議。(接線員指示) 本次電話會議將於 2026 年 8 月 13 日（星期四）錄音。

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

現在我想把會議交給財務長 AJ Bergmann 進行前瞻性聲明。請開始。

Thank you very much. Before we begin, I'd like to remind you that any statements made during today's call that are not historical are considered to be forward-looking statements. Actual results may differ materially from those indicated by these statements as a result of various important factors, including those discussed in the risk factors section of our company's most recent annual report on Form 10-K and our most recent quarterly reports on Form 10-Q, as well as other reports filed with the SEC. Any forward-looking statements may represent our views as of today, August 13, 2026. An audio replay of the call will be available on our website following its completion.

非常感謝。在開始之前，我想提醒各位，今天電話會議中所作的任何非歷史性陳述，均屬前瞻性陳述。由於各種重要因素，實際結果可能與這些陳述所示存在重大差異，包括我們公司最近一期 Form 10-K 年度報告及最近一期 Form 10-Q 季度報告之風險因素章節所討論者，以及向美國證券交易委員會（SEC）提交的其他報告。任何前瞻性陳述僅代表我們截至今日（2026 年 8 月 13 日）的觀點。本次電話會議結束後，將可於我們網站收聽錄音回放。

With that, I will turn the call over to Linda Marban, CEO.

接下來，我將把電話會議交給執行長 Linda Marban。

Good afternoon everyone and thank you for joining Capricor's second quarter 2026 earnings call. Our BLA for deramiocel remains under review with the FDA with a current PDUFA target action date of August 22. Because that review is ongoing, there is a limit to what I can say about our interactions with the agency, but wanted to provide an update across three main topics: our regulatory status, pathway for deramiocel, our commercial and manufacturing readiness, and our dispute with NS Pharma. I will then briefly address our pipeline programs before turning it back to AJ.

各位下午好，感謝各位參加 Capricor 2026 年第二季財報電話會議。我們針對 deramiocel 的 BLA 仍在 FDA 審查中，目前的 PDUFA 目標行動日期為 8 月 22 日。由於審查仍在進行中，我能就我們與主管機關互動所能說明的內容有所限制，但我想就三個主要主題提供最新進展：我們的法規審查狀態、deramiocel 的推進路徑、我們的商業化與製造準備情況，以及我們與 NS Pharma 的爭議。之後我會簡要說明我們的研發管線計畫，再把時間交回給 AJ。

On July 29, 2026, the FDA convened the Cellular, Tissue, and Gene Therapies Advisory Committee to review our BLA. The committee was presented with a single voting question, does the available evidence provide substantial evidence of effectiveness of deramiocel for the treatment of cardiomyopathy in patients with DMD?

2026 年 7 月 29 日，FDA 召開「細胞、組織與基因治療諮詢委員會」審查我們的 BLA。委員會收到一個投票問題：現有證據是否為 deramiocel 用於治療 DMD 患者之心肌病變提供充分的有效性證據？

The vote was three in favor, nine against, with no abstentions. That is not the outcome we had planned for, and we are, of course, disappointed, but we remain committed to working with the FDA on the next steps for this program. Our priority is, and always has been, to get deramiocel to those who need it most.

投票結果為贊成 3 票、反對 9 票，無棄權。這並非我們原先預期的結果，我們當然感到失望，但我們仍致力於與 FDA 合作，推進此計畫的後續步驟。我們的首要任務一直都是，讓 deramiocel 送達最需要它的人。

I would like to provide some color in our perspective about why we continue to believe in the potential of deramiocel in DMD patients. First, the indication we originally requested in the BLA going back to 2024 was the treatment of cardiomyopathy in DMD. Therefore, the focus of the FDA and the advisory committee was on whether deramiocel should be approved to treat cardiomyopathy.

我想補充一些背景，說明為何我們仍相信 deramiocel 在 DMD 患者中的潛力。首先，我們在 BLA 中最初於 2024 年提出的適應症，是治療 DMD 的心肌病變。因此，FDA 與諮詢委員會的重點在於 deramiocel 是否應獲核准用於治療心肌病變。

However, the measurement of deramiocel's effect on cardiomyopathy was a key secondary endpoint rather than the primary endpoint of the HOPE-3 study. And it measured change in ejection fraction across the full DMD population, rather than in patients with established cardiomyopathy, the population the proposed indication addresses.

然而，deramiocel 對心肌病變影響的衡量指標，在 HOPE-3 研究中屬於關鍵次要終點，而非主要終點。此外，它衡量的是整體 DMD 族群的射出分率變化，而非在已確立心肌病變的患者中評估；而後者才是所提適應症所針對的族群。

By contrast, HOPE-3 was actually designed with a skeletal functional primary endpoint and with power to assess efficacy in upper limb function. Importantly, the advisory committee was not asked to vote on whether they believe the data on the HOPE-3 primary efficacy endpoint could support approval of the product, nor whether the overall benefit-risk profile of deramiocel was favorable.

相較之下，HOPE-3 的設計其實是以骨骼肌功能作為主要終點，並具備評估上肢功能療效的統計檢定力。重要的是，諮詢委員會並未被要求就 HOPE-3 主要療效終點的數據是否可支持產品核准進行投票，也未就 deramiocel 的整體效益風險概況是否有利進行投票。

We continue to believe that the data on the primary, as well as multiple other endpoints, support a finding of effectiveness on these measures. It is worth noting that in a separate discussion on upper limb function during the ADCOM, the committee's feedback was directionally supportive of the clinical evidence for the primary endpoint in upper limb function. The discussion was substantive and the full record is public for anyone who wants to review it.

我們仍相信主要終點以及多個其他終點的數據，支持在這些衡量指標上具有有效性的結論。值得注意的是，在 ADCOM 期間針對上肢功能的另一段討論中，委員會的回饋方向上支持主要終點（上肢功能）的臨床證據。該討論內容相當深入，完整紀錄已公開，任何想查閱的人都可取得。

Now, this brings me to an update that I am very pleased to share. We are continuing to work closely with FDA on a potential path forward for deramiocel focused on an upper limb skeletal muscle indication reflected in the primary efficacy endpoint of HOPE-3.

接下來，我要分享一項讓我非常高興的最新進展。我們正持續與 FDA 密切合作，為 deramiocel 探索一條可能的前進路徑，聚焦於 HOPE-3 主要療效終點所反映的上肢骨骼肌適應症。

To that end, following discussions with the agency, subsequent to our Advisory Committee meeting, we plan to submit an amendment to our BLA that includes the 24-month open-label extension data from the HOPE-3 study, along with additional analyses on the existing data package, in order to support a refined indication focused on the primary endpoint.

為此，在諮詢委員會會議之後，並在與主管機關討論後，我們計畫提交 BLA 修正案，納入 HOPE-3 研究 24 個月開放標籤延伸期數據，以及對既有數據套件的額外分析，以支持一個聚焦於主要終點的精煉適應症。

The FDA has indicated it is willing to review this amendment and upon receipt to extend the PDUFA action date accordingly. We are finalizing the timing of that submission and will provide an update as appropriate. We appreciate the FDA's engagement throughout this process and its shared commitment to addressing the major unmet need for Duchenne muscular dystrophy.

FDA 已表示願意審查此修正案，並在收到後相應延長 PDUFA 行動日期。我們正在敲定提交時程，並將在適當時候提供更新。我們感謝 FDA 在整個過程中的投入，以及其與我們共同致力於解決杜興氏肌肉失養症重大未被滿足醫療需求的承諾。

Now there were two other developments in the review this quarter. In July, we were proud to report that the results of the HOPE-3 clinical trial were published in The Lancet following extensive and independent peer review.

本季審查過程中還有另外兩項進展。7 月，我們很榮幸宣布 HOPE-3 臨床試驗結果在經過廣泛且獨立的同儕審查後，已發表於《The Lancet》。

The first publication of the full Phase 3 dataset, an important milestone for this program and for the field. The publication highlights the efficacy of deramiocel and the supplement highlights the mechanism of action as well as the individual patient-level data.

這是首次發表完整第三期數據集，對本計畫及整個領域而言都是重要里程碑。該論文強調 deramiocel 的療效，而補充資料則強調其作用機轉以及個別患者層級的數據。

There's a lot of information available publicly, and we are confident that this highly regarded publication will help support continued progress for our deramiocel program. In connection with that peer review and as part of our dialogue with the FDA and The Lancet, we identified an issue with the statistical model in the clinical study report and reverted back to the statistical analysis plan version 3.0 put in place prior to unblinding.

目前已有大量資訊公開可得，我們相信這篇備受推崇的發表將有助於支持 deramiocel 計畫的持續推進。在該同儕審查過程中，並作為我們與 FDA 及《The Lancet》對話的一部分，我們發現臨床研究報告中的統計模型存在一項問題，因此回復採用在揭盲前即已制定的統計分析計畫第 3.0 版。

That model, the one underlying our top-line release, included an interaction term combining two independent variables, age and baseline, which were part of the pre-specified plan. The only endpoint directly impacted was left ventricular ejection fraction in all patients, the key secondary endpoint of the HOPE-3 study.

該模型（亦即我們發布主要結果時所依據的模型）包含一個交互作用項，將兩個獨立變數——年齡與基線值——結合在一起，而這些皆屬於預先指定計畫的一部分。唯一受到直接影響的終點是所有患者的左心室射出分率，亦即 HOPE-3 研究的關鍵次要終點。

At top line, we reported a 2.4 percentage point treatment difference with a p-value of 0.04. As published in The Lancet under the pre-specified model, the measure of left ventricular ejection fraction in all patients was a 1.8 percentage point treatment difference with a p-value of 0.09.

在主要結果發布時，我們報告治療差異為 2.4 個百分點，p 值為 0.04。依《The Lancet》所發表之預先指定模型，所有患者的左心室射出分率衡量結果為 1.8 個百分點的治療差異，p 值為 0.09。

We took the most conservative approach available to us in the publication and in follow-up interactions with FDA. Nothing else changed in the data or its analysis. We remind you in the pre-specified cardiomyopathy subgroup, the result was unchanged at p equals 0.02 with a 2.8 percentage point treatment difference. The endpoints below left ventricular ejection fraction in the testing hierarchy are characterized now as nominally significant with treatment effect unchanged.

在論文發表以及後續與 FDA 的互動中，我們採取了我們所能採取的最保守作法。數據本身或其分析的其他部分均未改變。我們也提醒各位，在預先指定的心肌病變亞組中，結果維持不變：p 值為 0.02，治療差異為 2.8 個百分點。在檢定階層中位於左心室射出分率之下的終點，現被描述為名目上顯著（nominally significant），且治療效果不變。

Now, let me be clear that the HOPE-3 primary endpoint was unaffected and is significant both statistically and clinically. Deramiocel demonstrated a statistically significant slowing of upper limb disease progression as measured by PUL 2.0 with a mean difference of 4.55% in favor of deramiocel with a p-value of 0.029, which corresponds to a 1.2 point absolute change in [total full point of].

現在，讓我清楚說明：HOPE-3 的主要終點未受影響，且在統計與臨床上皆具顯著性。Deramiocel 顯示可在統計上顯著減緩上肢疾病進展；以 PUL 2.0 衡量，deramiocel 組相較對照組的平均差異為 4.55%，p 值為 0.029，這相當於[總分滿分的] 1.2 分絕對變化。

We believe the efficacy and safety data supporting the potential for deramiocel is strong. We have administered approximately 1,300 intravenous infusions across our clinical program to over 200 patients with DMD in three separate clinical trials. More than 80 patients are in our collective open-label extension studies, with some receiving continuous infusions for more than five years, and the long-term safety profile is consistent and well-characterized.

我們相信，支持 deramiocel 潛力的療效與安全性數據相當強勁。在我們的臨床計畫中，我們已於三項獨立臨床試驗中，對超過 200 名 DMD 患者施打約 1,300 次靜脈輸注。在我們合併的開放標籤延伸研究中有超過 80 名患者，其中部分患者已連續輸注超過五年，而其長期安全性概況一致且已充分表徵。

The open public hearing part of the advisory committee included testimony from patients, families, and clinicians living with Duchenne muscular dystrophy. We were grateful that their experience is part of the record, and we look forward to continuing with the FDA on a path forward for deramiocel.

諮詢委員會的公開聽證環節包含了來自杜興氏肌肉失養症患者、家屬與臨床醫師的證詞。我們很感謝他們的經驗已納入紀錄，並期待與 FDA 持續合作，推進 deramiocel 的後續路徑。

Also in July, as part of the review process, the FDA conducted a bioresearch monitoring inspection, or BIMO, and issued a Form 483 citing one observation. We have submitted our responses and are currently awaiting feedback.

另外在 7 月，作為審查流程的一部分，FDA 進行了生物研究監測稽查（bioresearch monitoring inspection，或 BIMO），並發布 Form 483，指出一項觀察事項。我們已提交回覆，目前正等待回饋。

Second, let me talk a little bit about our commercial readiness and manufacturing. We are continuing our commercial readiness activities, but at a slower pace until we have further regulatory clarity. And although the scope and timing of some of them may change, depending on the outcome of the review, we are controlling our cash against this.

第二，我想簡要談談我們的商業化準備與製造。我們持續推進商業化準備工作，但在獲得更進一步的監管明確性之前，將以較慢的節奏進行。此外，儘管其中部分工作的範圍與時程可能會依審查結果而改變，我們也正據此控管現金支出。

Our in-house GMP manufacturing facility in San Diego is operational and positioned to support an initial commercial launch if approved. The expansion to the second floor of that same facility continues, and our goal remains full validation and FDA approval of the expanded space estimated to be in 2027. The space is ideal for early commercialization and allows for the most flexibility as we continue to scale our CMC capacity to account for potential demand.

我們位於聖地牙哥的自有 GMP 製造設施已投入運作，若獲核准，已具備支援初期商業上市的條件。同一設施二樓的擴建工程仍在進行中，我們的目標仍是於 2027 年完成擴建空間的全面驗證並取得 FDA 核准。該空間非常適合早期商業化，並在我們持續擴充 CMC 產能以因應潛在需求時，提供最大的彈性。

On the commercial side, Michael Moore joined us as our Chief Commercial Officer, bringing direct DMD and rare disease commercial experience. And he has judiciously been building out the launch organization alongside our market access leadership.

在商業端方面，Michael Moore 加入我們擔任首席商務官，帶來直接的 DMD 與罕見疾病商業化經驗。他也在我們市場准入領導團隊的配合下，審慎地建置上市組織。

Now let me talk for a minute about our dispute with NS Pharma. The state court was scheduled to hear our motion for preliminary injunction on August 10, ahead of the FDA's expected PDUFA date. However, we determined that resolving this contractual dispute in arbitration following the agency's decision would give the parties a more complete regulatory record to work from. Therefore, we withdrew the motion without prejudice. In terms of timeline, we estimate the arbitration process to begin this fall to address the contract dispute while continuing to pursue commercial readiness activities for deramiocel.

接下來我想花一點時間談談我們與 NS Pharma 的爭議。州法院原定於 8 月 10 日、在 FDA 預期的 PDUFA 日期之前，審理我們提出的初步禁制令動議。然而，我們判斷在主管機關作出決定後，於仲裁中解決此合約爭議，將使各方能基於更完整的監管紀錄來處理。因此，我們在不影響日後再提的情況下撤回該動議。就時程而言，我們預估仲裁程序將於今年秋季開始，以處理合約爭議，同時也會持續推進 deramiocel 的商業化準備工作。

Now, let me be clear that our position on the underlying dispute has not changed. We continue to believe that the pricing structure in the US agreement is fundamentally flawed in a way that would impede patient access and we continue to seek rescission. What changed is the current process by which we are pursuing a remedy. Our view of the merits of the case has not changed.

我現在要明確說明，我們對於爭議核心問題的立場並未改變。我們仍然認為，美國協議中的定價結構在根本上存在缺陷，將阻礙患者取得治療，我們也持續尋求撤銷該協議。改變的是我們目前尋求救濟的程序。我們對本案實體理由的看法並未改變。

Now, very quickly turning to our pipeline, I would like to state that all pipeline work that is not directly related to deramiocel is on hold right now until we have further regulatory clarity. Having said that, in terms of life cycle management of deramiocel, we have initiated regulatory engagement in Europe and Japan.

接著很快談到我們的產品線（pipeline），我想說明，目前所有與 deramiocel 無直接關聯的產品線工作都暫停，直到我們獲得更進一步的監管明確性。不過，就 deramiocel 的產品生命週期管理而言，我們已在歐洲與日本啟動監管互動。

Our expansion plans, including those for younger DMD patients and for Becker muscular dystrophy, remain priorities, and the timing of those clinical trial initiations will be stage-gated by the timeline of our regulatory pathway for deramiocel in the US to treat those with Duchenne muscular dystrophy later stage.

我們的擴展計畫（包括針對更年幼的 DMD 患者以及貝克型肌肉失養症）仍為優先事項，而這些臨床試驗啟動的時點，將依我們在美國針對較晚期杜興氏肌肉失養症患者之 deramiocel 監管路徑時程進行分階段決策（stage-gated）。

With that, I will now turn the call over to AJ to review the financial results.

接下來，我將把電話會議交給 AJ，請他回顧財務結果。

Thank you, Linda. As of June 30, 2026, Capricor had cash, cash equivalents and marketable securities totaling approximately $237.9 million, and there was no revenue recognized for the second quarter of 2026 or 2025.

謝謝你，Linda。截至 2026 年 6 月 30 日，Capricor 的現金、約當現金及有價證券合計約為 2.379 億美元，且 2026 年或 2025 年第二季皆未認列營收。

Total operating expenses for the second quarter of 2026 were approximately $42.9 million compared to approximately $27.7 million for the second quarter of 2025. The increase was primarily driven by continued investment in clinical, regulatory, and manufacturing activities as well as commercial infrastructure supporting our Duchenne program. Net loss for the second quarter of '26 was approximately $40.7 million or $0.70 per share compared to a net loss of approximately $25.9 million or $0.57 per share for the second quarter of 2025.

2026 年第二季的總營運費用約為 4,290 萬美元，相較於 2025 年第二季約 2,770 萬美元。增加主要來自於我們持續投資於臨床、監管與製造活動，以及支援杜興氏計畫的商業基礎建設。2026 年第二季淨損約為 4,070 萬美元或每股 0.70 美元，相較於 2025 年第二季淨損約 2,590 萬美元或每股 0.57 美元。

And for the six months ended June 30, 2026, our net loss was approximately $74.7 million compared to approximately $50.3 million for the same period in 2025. As of June 30, 2026, we had an accumulated deficit of approximately $379.6 million.

截至 2026 年 6 月 30 日止六個月，我們的淨損約為 7,470 萬美元，相較於 2025 年同期約 5,030 萬美元。截至 2026 年 6 月 30 日，我們的累積虧損約為 3.796 億美元。

Our expense profile this quarter reflects investment across our three main areas: regulatory and clinical activities in support of our DMD program, manufacturing capacity expansion efforts, and commercial readiness activities.

本季的費用結構反映我們在三大領域的投資：支援 DMD 計畫的監管與臨床活動、製造產能擴充工作，以及商業化準備活動。

As Linda noted, we are pacing certain commercial expenditures as the regulatory timeline develops and becomes more clear, and we continue to have flexibility in how we deploy capital across the remainder of the year.

如 Linda 所提到，隨著監管時程的發展並逐步明朗，我們正調整部分商業支出節奏，且在今年剩餘期間，我們在資本配置上仍保有彈性。

With that, I will turn the call back over to Linda for a closing.

接下來，我將把電話會議交回給 Linda 作結語。

Thank you, AJ. As all of you know, the last year has been one of highs and lows for Capricor. We were stunned by the deramiocel and pleased by the HOPE-3 data. We were encouraged by the acceptance of the HOPE-3 data for resubmission in response to the deramiocel and disappointed by the advisory committee's recommendation.

謝謝你，AJ。各位都知道，過去一年對 Capricor 而言是高低起伏的一年。我們對 deramiocel 的情況感到震驚，同時對 HOPE-3 的數據感到欣慰。我們對 HOPE-3 數據獲接受、可作為回應 deramiocel 的重新提交而感到鼓舞，但也對諮詢委員會的建議感到失望。

Although we understood it based on the narrow voting question and the disparity between the indication we had previously asked for and the data from HOPE-3, which was powered to assess skeletal muscle as its primary goal, we have previously stated this.

儘管基於投票問題的狹窄範圍，以及我們先前所申請的適應症與 HOPE-3 數據之間的落差（HOPE-3 的主要目標是評估骨骼肌，並以此作為統計檢定力設計），我們可以理解其原因；我們先前也已表達過。

We were reassured by the strength of our data, by publication in The Lancet, and we were amazed by the outpouring of support for deramiocel by the DMD community. We hear their voices as well and will continue to work tirelessly to try and get deramiocel to every eligible patient based on their physician's recommendation.

我們因數據的強度、以及在《刺胳針》（The Lancet）發表而感到安心，同時也對 DMD 社群對 deramiocel 的大量支持感到驚訝。我們也聽見他們的聲音，並將持續不懈努力，依據醫師建議，嘗試讓 deramiocel 提供給每一位符合資格的患者。

We are grateful to FDA for their flexibility and for their collaborative approach. We will be submitting updated data to the FDA as soon as possible and look forward to their review. Lastly, due to the sensitivity of our ongoing discussions with the Food and Drug Administration, we are not holding a Q&A today, and we look forward to providing updates to you as they become available.

我們感謝 FDA 的彈性與其合作式的做法。我們將儘快向 FDA 提交更新數據，並期待其審查。最後，由於我們與美國食品藥物管理局正在進行的討論具有敏感性，我們今天不進行問答環節，並期待在有最新進展時向各位提供更新。

Thank you for your time. We look forward to positive updates in the future.

感謝各位撥冗。我們期待未來能帶來正面的更新。

This concludes today's call. Thank you all for participating. You may now disconnect.

今天的電話會議到此結束。感謝各位參與。您現在可以掛線。