카프리코 테라퓨틱스(CAPR) 2026년 2분기 실적 발표 콘퍼런스 콜: 데라모셀 BLA 경로 및 FDA 업데이트

핵심 요약

• 미국 식품의약국(FDA) 자문위원회는 뒤셴 근디스트로피(DMD) 환자의 심근병증 치료에 대한 데라모셀(deramocel)의 유효성을 입증하는 데이터와 관련해 찬성 3표, 반대 9표로 부결 표결했습니다.
• 카프리코(Capricor)는 24개월간의 HOPE-3 오픈라벨 연장 임상 데이터와 추가 분석 결과를 반영해 데라모셀의 생물학적 제제 품목허가 신청(BLA)을 수정하고, 상지 골격근 기능에 중점을 둔 정제된 적응증을 추진할 계획입니다. FDA는 해당 수정안을 접수하면 검토에 착수하고 기존 8월 22일로 예정되었던 PDUFA 심사 기한을 연장할 것이라고 밝혔습니다.
• HOPE-3의 1차 평가변수는 통계적 유의성을 유지했습니다. 데라모셀은 PUL 2.0 검사에서 평균 4.55%의 차이와 p-값 0.029를 기록하며 상지 질환의 진행을 완화했습니다.
• 2026년 6월 30일 기준 현금, 현금성 자산 및 매도가능증권은 총 2억 3,790만 달러를 기록했습니다. 2분기 순손실은 4,070만 달러(주당 0.70달러)로 확대되었습니다.
• 2분기 영업비용은 DMD 프로그램 지원을 위한 임상, 규제, 제조 및 상업화 투자가 늘어나면서 전년 동기 2,770만 달러에서 4,290만 달러로 증가했습니다.
• 카프리코는 규제 관련 불확실성이 해소될 때까지 일부 상업화 준비 지출을 줄이고 데라모셀과 무관한 파이프라인 개발 작업을 일시 중단했습니다.

주요 재무 데이터

사업 및 영업 성과

데라모셀 규제 승인 경로

FDA 자문위원회의 반대 표결은 DMD 환자의 심근병증에 관한 지극히 한정된 안건에 대한 것이었습니다. 경영진은 심근병증이 HOPE-3 임상의 주요 2차 평가변수였던 반면, 해당 임상은 상지 골격근 기능을 1차 평가변수로 삼아 설계되고 검증력을 확보했다고 강조했습니다.

FDA와의 논의에 따라 카프리코는 24개월 오픈라벨 연장 데이터와 기존 데이터셋에 대한 추가 분석을 포함한 BLA 수정안을 제출할 계획입니다. 제안된 적응증은 HOPE-3에서 측정된 상지 골격근 평가변수에 중점을 둘 예정입니다.

1차 평가변수는 PUL 2.0에서 데라모셀 우위의 평균 4.55% 차이와 p-값 0.029를 나타냈습니다. 카프리코는 이것이 절대적 수치로 약 1.2점 차이에 해당한다고 밝혔습니다.

회사 측은 3건의 임상시험을 통해 200명 이상의 DMD 환자에게 약 1,300회의 정맥 투여를 실시했습니다. 80명 이상의 환자가 오픈라벨 연장 연구에 참여 중이며, 일부 환자는 5년 이상 지속적으로 투여받고 있습니다.

HOPE-3 통계 업데이트

동료 평가(Peer review) 및 FDA, 랜싯(The Lancet)과의 논의 과정에서 카프리코는 좌심실 구출률 평가변수에 사용된 통계 모델의 문제를 확인했습니다. 사전 정의된 모델에 따르면 전체 환자에 대한 치료 효과 차이는 기존에 보고된 2.4%포인트(p-값 0.04)에서 1.8%포인트(p-값 0.09)로 변경되었습니다.

경영진은 HOPE-3의 1차 평가변수에는 영향을 미치지 않았다고 밝혔습니다. 사전 정의된 심근병증 하위 그룹의 경우에도 치료 효과 차이 2.8%포인트, p-값 0.02로 결과가 유효하게 유지되었습니다. 검증 계층구조상 좌심실 구출률 하위에 위치한 평가변수들은 치료 효과 자체는 동일하게 유지되었으나 현재 명목상 유의한(nominally significant) 것으로 규정되었습니다.

제조 및 상업화

샌디에이고에 위치한 카프리코의 자체 GMP 제조 시설은 정상 가동 중이며 데라모셀이 승인될 경우 초기 상업적 출시를 지원할 수 있는 체계를 갖추고 있습니다. 시설 2층 증설 작업이 계속되고 있으며, 경영진은 2027년까지 증설 공간에 대한 최종 검증 및 FDA 승인을 완료하는 것을 목표로 하고 있습니다.

상업화 준비 활동은 규제 불확실성이 해소될 때까지 완만한 속도로 진행되고 있습니다. 마이클 무어(Michael Moore)가 최고상업책임자(CCO)로 카프리코에 합류했으며, 시장 접근성(market-access) 리더십팀과 함께 제품 출시 조직을 구축하고 있습니다.

NS파마 분쟁 및 파이프라인 우선순위

카프리코는 가처분 신청을 소의 이익 보류(without prejudice) 상태로 취하했으며, NS파마(NS Pharma)와의 계약 분쟁을 중재 절차를 통해 해결할 계획입니다. 경영진은 중재가 2026년 가을에 시작될 것으로 예상하며, 미국 계약의 해제를 지속적으로 추진하고 있습니다.

데라모셀과 직접 관련되지 않은 파이프라인 프로그램 작업은 중단된 상태입니다. 카프리코는 유럽과 일본에서 규제 당국과의 협의를 시작했으나, 더 어린 DMD 환자 및 베커 근디스트로피 환자를 대상으로 한 후속 연구는 미국 규제 절차의 진행 상황에 따라 결정될 예정입니다.

경영진 전망

경영진은 예정된 BLA 수정안을 제출하면 FDA가 기존 8월 22일 PDUFA 심사 기한을 연장할 것으로 예상하고 있습니다. 회사는 현재 제출 시기를 최종 조율 중입니다.

카프리코는 상업화 관련 지출 속도를 조절하고 있으며, 2026년 잔여 기간 동안 자본 집행의 유연성을 유지할 것이라고 밝혔습니다. 제조 시설 증설은 규제 절차에 따라 2027년 최종 검증 및 FDA 승인을 받는 것을 목표로 유지하고 있습니다.

리스크 및 주시 사항

• 데라모셀 BLA는 여전히 FDA 심사 중에 있으나, 자문위원회는 제안된 심근병증 적응증을 뒷받침하는 데이터에 대해 반대 표결을 던졌습니다.
• 계획된 BLA 수정안 제출로 인해 규제 일정이 연장될 것이며, 수정된 PDUFA 기한은 제출 시점 및 FDA의 심사 진행에 따라 결정됩니다.
• 좌심실 구출률 평가변수에 대한 수정 분석 결과 전체 연구 대상군에서 p-값 0.09가 산출되었습니다.
• FDA의 바이오리서치 모니터링 실사 결과 1건의 지적 사항이 담긴 양식 483(Form 483)이 발급되었습니다. 카프리코는 이에 대한 답변서를 제출하고 피드백을 기다리고 있습니다.
• NS파마와의 계약 분쟁은 미결 상태로 남아 있으며 중재 절차로 이행될 예정입니다.
• 상업화 지출, 파이프라인 일정 및 시설 확장 계획은 데라모셀에 대한 규제 명확성이 더욱 확보되는지 여부에 달려 있습니다.

실적 발표 컨퍼런스 콜 전문

전체 실적 발표 컨퍼런스 콜 녹취록

경영진 발표

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.