Capricor Therapeutics (CAPR) 2026 年第二季法說會：Deramocel BLA 申請路徑與 FDA 最新進展

重點摘要

• FDA 諮詢委員會以 3 票贊成、9 票反對的投票結果，認為現有證據不足以支持 deramocel 用於治療杜興氏肌肉營養不良症 (DMD) 患者心肌病變的療效。
• Capricor 計劃補正 deramocel 的生物製品許可申請 (BLA)，加入 HOPE-3 為期 24 個月的開放標籤延伸試驗數據及補充分析，以尋求專注於上肢骨骼肌功能的修正適應症。FDA 表示收到補正資料後將進行審查，並延長原定於 8 月 22 日的 PDUFA 目標決議日。
• HOPE-3 的主要終點仍具統計顯著性。deramocel 減緩了 PUL 2.0 評估的上肢疾病進展，平均差異為 4.55%，p 值為 0.029。
• 截至 2026 年 6 月 30 日，現金、現金等價物與有價證券總額為 2.379 億美元。第二季淨虧損擴大至 4,070 萬美元，或每股虧損 0.70 美元。
• 受支持 DMD 計畫的臨床、法規、製造與商業化投資推動，第二季營業費用從 2,770 萬美元增至 4,290 萬美元。
• Capricor 正在放緩部分商業化準備支出，並已暫停與 deramocel 無關的研發管線工作，直到監管前景更加明確。

關鍵財務數據

業務與營運表現

Deramocel 監管審查途徑

FDA 諮詢委員會的反對票僅針對 DMD 心肌病變這一特定問題。管理層強調，心肌病變是 HOPE-3 的關鍵次要終點，而該試驗設計與統計檢定力主要是圍繞上肢骨骼肌功能這一主要終點展開。

在與 FDA 討論後，Capricor 計劃提出 BLA 修正案，包含 24 個月的開放標籤延伸數據以及現有數據集的進一步分析。擬議的適應症將聚焦於 HOPE-3 所評估的上肢骨骼肌功能終點。

主要終點顯示，在 PUL 2.0 評估中，deramocel 展現出 4.55% 的平均優勢差異，p 值為 0.029。Capricor 表示這相當於約 1.2 分的絕對差異。

公司已在三項臨床試驗中向 200 多名 DMD 患者進行了約 1,300 次靜脈注射。有超過 80 名患者參與了開放標籤延伸研究，部分患者已連續接受注射超過五年。

HOPE-3 統計數據更新

在同行審查以及與 FDA 和《柳葉刀》(The Lancet) 的討論過程中，Capricor 發現用於左心室射血分數終點的統計模型存在問題。在預先設定的模型下，所有患者的治療差異從先前報導的 2.4 個百分點（p 值 0.04）變更為 1.8 個百分點（p 值 0.09）。

管理層表示，HOPE-3 的主要終點未受影響。在預先設定的心肌病變亞組中，結果亦維持不變，治療差異為 2.8 個百分點，p 值為 0.02。在測試階層中低於左心室射血分數的終點，目前被歸類為名義上顯著，其治療效果保持不變。

製造與商業化

Capricor 位於聖地牙哥的自有 GMP 製造工廠已投入營運，若 deramocel 獲批，該廠已準備好支持初期商業化上市。該設施二樓的擴建工程仍在持續進行，管理層目標是在 2027 年完成擴建空間的全面驗證並獲得 FDA 批准。

在監管前景明確之前，商業化準備工作正以較慢的速度持續推進。Michael Moore 已加入 Capricor 擔任商業長 (CCO)，正與公司市場進入 (Market Access) 領導團隊一同建置上市團隊。

NS Pharma 爭議與研發管線優先事項

Capricor 已撤回其暫時狀態處分（假處分）聲請（不影響重新提起權利），並計劃透過仲裁解決與 NS Pharma 的合約爭議。管理層預估仲裁將於 2026 年秋季開始，並繼續尋求撤銷美國區協議。

與 deramocel 無直接關聯的研發管線項目已暫停推進。Capricor 已在歐洲和日本展開監管溝通，而針對較年輕 DMD 患者及貝克型肌肉營養不良症 (Becker muscular dystrophy) 的潛在研究，將視美國監管進程的進展而定。

管理層展望

管理層預計 FDA 在收到預定的 BLA 修正案後，將延長原定於 8 月 22 日的 PDUFA 目標決議日。公司目前正確定最終的提交時間。

Capricor 持續放緩商業化支出，並表示在 2026 年剩餘時間內仍保持資本配置的靈活性。公司的製造擴建計劃目標仍是在 2027 年完成全面驗證並獲得 FDA 批准，惟仍需視監管進程而定。

風險與關注事項

• deramocel 的 BLA 仍處於 FDA 審查階段，且諮詢委員會投票反對支持擬議心肌病變適應症的證據。
• 預計提出的 BLA 修正案將延長監管時程，修訂後的 PDUFA 日期將取決於提交時間及 FDA 的審查進度。
• 左心室射血分數終點的修訂分析顯示，在全體研究受試人群中的 p 值為 0.09。
• FDA 生物醫學研究監測 (BIMO) 檢查開出了一份包含一項缺失觀察事項的 Form 483。Capricor 已提交回覆並正在等待反饋。
• 與 NS Pharma 的合約爭議尚未解決，預計將進入仲裁程序。
• 商業化支出、研發管線時程及擴建計劃仍取決於 deramocel 是否能獲得更明確的監管前景。

法說會逐字稿全文

完整財報電話會議逐字稿

管理層陳述

Operator

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

Anthony Bergmann

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

Linda Marbán

Good afternoon everyone and thank you for joining Capricor's second quarter 2026 earnings call. Our BLA for deramocel 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 3 main topics: our regulatory status, pathway for deramocel, 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.

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 deramocel for the treatment of cardiomyopathy in patients with DMD? The vote was 3 in favor, 9 against, with 0 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 deramocel to those who need it most.

I would like to provide some color in our perspective about why we continue to believe in the potential of deramocel 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 deramocel should be approved to treat cardiomyopathy. However, the measurement of deramocel'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.

By contrast, HOPE-3 was actually designed with a skeletal functional primary endpoint and with power to assess efficacy in upper limb function. [ In pre, ] 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 deramocel was favorable. 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.

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 deramocel focused on an upper limb skeletal muscle indication reflected in the primary efficacy endpoint of HOPE-3.

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. 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.

Now there were 2 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. 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 deramocel and the supplement highlights the mechanism of action as well as the individual patient-level data. There's a lot of information available publicly, and we are confident that this highly regarded publication will help support continued progress for our deramocel 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. That model, the one underlying our top-line release, included an interaction term combining 2 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. 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. 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.

Now, let me be clear that the HOPE-3 primary endpoint was unaffected and is significant both statistically and clinically. Deramocel 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 deramocel with a p-value of 0.029, which corresponds to a 1.2 point absolute change in [ total full point of ]. We believe the efficacy and safety data supporting the potential for deramocel is strong.

We have administered approximately 1,300 intravenous infusions across our clinical program to over 200 patients with DMD in 3 separate clinical trials. More than 80 patients are in our collective open-label extension studies, with some receiving continuous infusions for more than 5 years, and the long-term safety profile is consistent and well-characterized.

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 deramocel.

Also in July, as part of the review process, the FDA conducted a bioresearch monitoring inspection, or BIMO, and issued a Form 483 citing 1 observation. We have submitted our responses and are currently awaiting feedback.

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.

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.

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.

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 deramocel. 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 U.S. 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.

Now, very quickly turning to our pipeline, I would like to state that all pipeline work that is not directly related to deramocel is on hold right now until we have further regulatory clarity. Having said that, in terms of life cycle management of deramocel, we have initiated regulatory engagement in Europe and Japan. 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 deramocel in the U.S. to treat those with Duchenne muscular dystrophy later stage.

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

Anthony Bergmann

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.

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. And for the 6 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. Our expense profile this quarter reflects investment across our 3 main areas: regulatory and clinical activities in support of our DMD program, manufacturing capacity expansion efforts, and commercial readiness activities.

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

Linda Marbán

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 [indiscernible] and pleased by the HOPE-3 data. We were encouraged by the acceptance of the HOPE-3 data for resubmission in response to the [indiscernible] and disappointed by the advisory committee's recommendation. 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, we were reassured by the strength of our data by publication in The Lancet, and we were amazed by the outpouring of support for deramocel by the DMD community. We hear their voices as well and will continue to work tirelessly to try and get deramocel to every eligible patient based on their physician's recommendation.

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

Operator

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