Capricor Therapeutics (CAPR) 2026年第二季度业绩电话会：Deramocel BLA路径及FDA最新进展

核心要点

• FDA咨询委员会以3票赞成、9票反对的投票结果，认为现有证据不支持deramocel用于治疗杜氏肌营养不良症（DMD）患者心肌病的有效性。
• Capricor计划通过补充24个月HOPE-3开放标签扩展期数据及其他分析来修订deramocel的BLA申请，以寻求专注于上肢骨骼肌功能的更精准适应症。FDA表示，在收到补充材料后将进行审评，并将延长目前8月22日的PDUFA行动日期。
• HOPE-3的主要终点仍具有统计学显著性。根据PUL 2.0评估，deramocel延缓了上肢疾病进展，平均差异为4.55%，p值为0.029。
• 截至2026年6月30日，现金、现金等价物及可交易证券总计为2.379亿美元。第二季度净亏损扩大至4070万美元，即每股亏损0.70美元。
• 受支持DMD项目的临床、监管、生产和商业化投资推动，第二季度运营费用从2770万美元升至4290万美元。
• Capricor正在放缓部分商业化准备支出，并已暂停与deramocel无关的管线工作，以等待监管的进一步明确。

关键财务数据

业务与运营表现

Deramocel监管路径

FDA咨询委员会的否定投票针对的是有关DMD心肌病的一个特定狭义问题。管理层强调，心肌病是HOPE-3试验的关键次要终点，而该试验的设计和统计效力是围绕上肢骨骼肌功能这一主要终点确定的。

在与FDA讨论后，Capricor计划提交BLA补充材料，其中包括24个月开放标签扩展期数据以及对现有数据集的进一步分析。拟定的适应症将专注于HOPE-3中评估的上肢骨骼肌终点。

主要终点显示，在PUL 2.0评估中，deramocel具有4.55%的平均差异优势，p值为0.029。Capricor表示，这相当于约1.2分的绝对差异。

该公司已在三项临床试验中向200多名DMD患者输注了约1300次静脉注射。80多名患者正在参与开放标签扩展研究，其中一些患者已持续接受输注超过五年。

HOPE-3统计数据更新

在同行评审以及与FDA和《柳叶刀》（The Lancet）沟通期间，Capricor发现用于左心室射血分数终点的统计模型存在问题。在预设模型下，所有患者的治疗效果差异由此前报告的2.4个百分点（p值为0.04）变为1.8个百分点（p值为0.09）。

管理层表示，HOPE-3的主要终点未受影响。在预设的心肌病亚组中，结果也保持不变，治疗差异为2.8个百分点，p值为0.02。在检验层级中排在左心室射血分数之后的终点目前被归类为名义上显著，其治疗效果保持不变。

生产与商业化

Capricor位于圣迭戈的自营GMP生产设施已投入运营，若deramocel获批，可支持首次商业化上市。设施二楼的扩建工作正在持续推进，管理层计划在2027年完成扩建区域的全盘验证并获得FDA批准。

在监管明确之前，商业化准备活动正在以较慢的节奏推进。Michael Moore已加入Capricor担任首席商业官，目前正与公司的市场准入领导团队一道组建上市团队。

NS Pharma争议与管线优先级

Capricor在不损害自身权益的前提下撤回了初步禁令申请，并计划通过仲裁解决与NS Pharma的合同争议。管理层预计仲裁将于2026年秋季开始，并将继续寻求解约美方协议。

与deramocel无直接关系的管线项目研发工作已暂停。Capricor已在欧洲和日本启动监管沟通，而针对更年轻DMD患者及贝克型肌营养不良症的潜在研究将取决于美国监管流程的进展。

管理层展望

管理层预计，FDA在收到计划提交的BLA补充材料后，将延长目前8月22日的PDUFA行动日期。公司正在最终敲定提交时间。

Capricor继续把控商业化支出节奏，并表示在2026年剩余时间内保持资本配置的灵活性。取决于监管流程，公司生产设施扩建项目仍以2027年完成全盘验证并获得FDA批准为目标。

风险与关注事项

• deramocel的BLA仍处于FDA审评阶段，咨询委员会对支持拟定心肌病适应症的证据投了反对票。
• 计划提交的BLA补充材料将延长监管时间表，修订后的PDUFA日期将取决于提交进度和FDA审评。
• 左心室射血分数终点的修正分析显示，在全研究人群中的p值为0.09。
• FDA生物医学研究监测检查出具了一份包含一项观察项的483表。Capricor已提交回复，目前正在等待反馈。
• 与NS Pharma的合同争议仍未解决，预计将进入仲裁程序。
• 商业化支出、管线进度和扩建计划仍取决于deramocel监管前景的进一步明确。

业绩电话会议完整文字记录

完整财报电话会议逐字稿

管理层陈述

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.