Conferencia de resultados del T2 de 2026 de Capricor Therapeutics (CAPR): Vía para la BLA de deramocel y actualización de la FDA

Puntos clave

• El comité asesor de la FDA votó 3 a favor y 9 en contra sobre si las pruebas respaldaban la eficacia de deramocel para tratar la miocardiopatía en pacientes con distrofia muscular de Duchenne (DMD).
• Capricor planea enmendar la BLA de deramocel con datos del estudio de extensión de etiqueta abierta HOPE-3 a 24 meses y análisis adicionales, con el objetivo de buscar una indicación afinada centrada en la función del músculo esquelético de las extremidades superiores. La FDA indicó que revisará la enmienda y extenderá la fecha de acción PDUFA actual del 22 de agosto tras recibirla.
• El criterio de valoración principal de HOPE-3 siguió siendo estadísticamente significativo. Deramocel ralentizó la progresión de la enfermedad en las extremidades superiores según la escala PUL 2.0, con una diferencia media del 4,55% y un valor p de 0,029.
• El efectivo, los equivalentes de efectivo y los valores negociables sumaban 237,9 millones de dólares al 30 de junio de 2026. La pérdida neta del segundo trimestre se amplió a 40,7 millones de dólares, o 0,70 dólares por acción.
• Los gastos operativos del segundo trimestre aumentaron a 42,9 millones de dólares desde los 27,7 millones de dólares, impulsados por inversiones clínicas, regulatorias, de fabricación y comerciales para respaldar el programa de DMD.
• Capricor está ralentizando ciertos gastos de preparación comercial y ha pausado el trabajo en proyectos de desarrollo no relacionados con deramocel a la espera de una mayor claridad regulatoria.

Datos financieros clave

Desempeño operativo y del negocio

Vía regulatoria de deramocel

El voto en contra del comité asesor de la FDA abordó una cuestión específica relativa a la miocardiopatía en la DMD. La directiva enfatizó que la miocardiopatía era un criterio de valoración secundario clave en HOPE-3, mientras que el ensayo se diseñó y dimensionó en torno a un criterio de valoración principal de la función esquelética de las extremidades superiores.

Tras las conversaciones con la FDA, Capricor planea presentar una enmienda a la BLA que contiene datos de extensión de etiqueta abierta a 24 meses y análisis adicionales del conjunto de datos existente. La indicación propuesta se centraría en el criterio de valoración del músculo esquelético de las extremidades superiores medido en HOPE-3.

El criterio de valoración principal mostró una diferencia media del 4,55% a favor de deramocel en PUL 2.0, con un valor p de 0,029. Capricor señaló que esto se correspondía con una diferencia absoluta de aproximadamente 1,2 puntos.

La empresa ha administrado alrededor de 1.300 infusiones intravenosas a más de 200 pacientes con DMD a lo largo de tres ensayos clínicos. Más de 80 pacientes participan en estudios de extensión de etiqueta abierta, y algunos reciben infusiones continuas desde hace más de cinco años.

Actualización estadística de HOPE-3

Durante la revisión por pares y las conversaciones con la FDA y The Lancet, Capricor identificó un problema con el modelo estadístico utilizado para el criterio de valoración de la fracción de eyección del ventrículo izquierdo. Bajo el modelo preespecificado, la diferencia de tratamiento para todos los pacientes cambió de los 2,4 puntos porcentuales informados previamente (con un valor p de 0,04) a 1,8 puntos porcentuales con un valor p de 0,09.

La directiva afirmó que el criterio de valoración principal de HOPE-3 no se vio afectado. En el subgrupo de miocardiopatía preespecificado, el resultado también se mantuvo sin cambios, con una diferencia de tratamiento de 2,8 puntos porcentuales y un valor p de 0,02. Los criterios de valoración por debajo de la fracción de eyección del ventrículo izquierdo en la jerarquía de pruebas ahora se caracterizan como nominalmente significativos, sin cambios en sus efectos del tratamiento.

Fabricación y comercialización

La planta de fabricación GMP propia de Capricor en San Diego está operativa y en condiciones de respaldar un lanzamiento comercial inicial si se aprueba deramocel. La ampliación de la segunda planta de las instalaciones continúa, y la directiva prevé la validación completa y la aprobación de la FDA del espacio ampliado en 2027.

Las actividades de preparación comercial continúan a un ritmo más lento a la espera de claridad regulatoria. Michael Moore se incorporó a Capricor como director comercial y está estructurando la organización para el lanzamiento junto con el equipo de liderazgo de acceso al mercado de la empresa.

Litigio con NS Pharma y prioridades de desarrollo

Capricor retiró su moción de medida cautelar sin perjuicio de sus derechos y planea resolver la disputa contractual con NS Pharma mediante arbitraje. La directiva calcula que el arbitraje comenzará en el otoño de 2026 y continúa buscando la rescisión del acuerdo en Estados Unidos.

El trabajo en los programas en desarrollo no relacionados directamente con deramocel está en pausa. Capricor ha iniciado gestiones regulatorias en Europa y Japón, mientras que los posibles estudios en pacientes con DMD más jóvenes y en distrofia muscular de Becker dependerán del avance del proceso regulatorio en Estados Unidos.

Perspectivas de la dirección

La directiva prevé que la FDA extienda la fecha de acción PDUFA actual del 22 de agosto tras recibir la enmienda a la BLA prevista. La empresa está finalizando el calendario de presentación.

Capricor sigue modulando los gastos comerciales y afirmó que mantiene flexibilidad sobre el despliegue de capital para el resto de 2026. La ampliación de la capacidad de fabricación de la empresa mantiene como objetivo la validación completa y la aprobación de la FDA en 2027, sujeto al proceso regulatorio.

Riesgos y aspectos a vigilar

• La BLA de deramocel sigue bajo revisión de la FDA, y el comité asesor votó en contra de las pruebas que respaldaban la indicación de miocardiopatía propuesta.
• La enmienda prevista a la BLA extenderá el calendario regulatorio, y la fecha PDUFA revisada dependerá de la presentación y de la revisión por parte de la FDA.
• El análisis revisado del criterio de valoración de la fracción de eyección del ventrículo izquierdo arrojó un valor p de 0,09 en la población total del estudio.
• Una inspección de supervisión de investigación biológica por parte de la FDA dio lugar a un Formulario 483 con una observación. Capricor presentó su respuesta y está a la espera de comentarios.
• La disputa contractual con NS Pharma continúa sin resolverse y se prevé que pase a arbitraje.
• El gasto comercial, los plazos de desarrollo y los planes de expansión siguen condicionados a una mayor claridad regulatoria respecto a deramocel.

Transcripción completa de la conferencia de resultados

Transcripción completa de la conferencia de resultados

Comentarios de la dirección

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.