Capricor Therapeutics, Inc. (CAPR): Lienzo del Modelo de Negocio [Actualizado en Ene-2025]

En el reino de vanguardia de la medicina regenerativa, Capricor Therapeutics (CAPR) emerge como una fuerza pionera, transformando cómo nos acercamos a los trastornos cardíacos genéticos raros. Al aprovechar las innovadoras terapias basadas en células y la medicina de precisión, esta compañía dinámica de biotecnología está reescribiendo el panorama potencial de tratamiento para afecciones desafiantes como la distrofia muscular de Duchenne. Su lienzo de modelo de negocio integral revela un enfoque estratégico que combina excelencia científica, investigación específica y potencial terapéutico innovador, posicionando el caprictor a la vanguardia de la innovación médica transformadora.

Capricor Therapeutics, Inc. (CAPR) - Modelo de negocio: asociaciones clave

Instituciones de investigación académica colaboraciones

Capricor Therapeutics mantiene asociaciones estratégicas con las siguientes instituciones de investigación académica:

Institución	Enfoque de investigación	Detalles de la asociación
Universidad de California, Los Ángeles (UCLA)	Investigación de terapia de células cardíacas	Colaboración de ensayos clínicos en curso para la distrofia muscular de Duchenne
Universidad de Johns Hopkins	Medicina regenerativa	Investigación colaborativa sobre tecnologías de reparación cardíaca

Financiación de la investigación de los Institutos Nacionales de Salud (NIH)

Detalles de financiación de NIH para Capricor Therapeutics:

• Subvenciones totales de NIH recibidas en 2023: $ 2.4 millones
• Categorías de subvenciones de investigación: medicina regenerativa cardíaca
• Períodos de subvención: múltiples ciclos de financiación de investigación de 2-3 años

Asociaciones de organizaciones de investigación de contratos (CRO)

Nombre de Cro	Servicios proporcionados	Valor de contrato
Ícono plc	Gestión de ensayos clínicos	Contrato anual de $ 1.7 millones
Medpace, Inc.	Desarrollo de fármacos preclínicos y clínicos	Acuerdo basado en proyectos de $ 1.3 millones

Posibles asociaciones farmacéuticas

Estado actual de colaboración farmacéutica:

• Discusiones continuas con 3 principales compañías farmacéuticas
• Valor de asociación potencial estimado en $ 15-25 millones
• Áreas de enfoque: distrofia muscular de Duchenne y terapias regenerativas cardíacas

Capricor Therapeutics, Inc. (CAPR) - Modelo de negocio: actividades clave

Desarrollo de terapias celulares regenerativas para condiciones cardíacas raras

Capricor Therapeutics se centra en desarrollar terapias celulares regenerativas con énfasis específico en aplicaciones cardíacas. A partir del cuarto trimestre de 2023, la compañía ha invertido $ 12.3 millones en investigación y desarrollo para tecnologías de terapia de células cardíacas.

Área de investigación	Asignación de financiación	Etapa de desarrollo
Terapias regenerativas cardíacas	$ 7.5 millones	Ensayos preclínicos/clínicos
Tratamiento de distrofia muscular de Duchenne	$ 4.8 millones	Fase clínica II

Realización de ensayos clínicos para tratamientos con distrofia muscular de Duchenne

La compañía está realizando activamente ensayos clínicos para los tratamientos de distrofia muscular de Duchenne (DMD) utilizando su plataforma de terapia celular patentada.

• Presupuesto actual de ensayo clínico: $ 3.2 millones
• Número de sitios de ensayos clínicos activos: 7
• Objetivo de inscripción de pacientes: 45 participantes

Investigación de tecnologías de terapia de células cardíacas

Capricor Therapeutics asigna recursos significativos a la investigación avanzada de terapia de células cardíacas, con un equipo de investigación dedicado de 18 científicos.

Enfoque de investigación	Tamaño del equipo de investigación	Presupuesto de investigación anual
Tecnologías regenerativas cardíacas	18 investigadores	$ 5.6 millones

Avance de los enfoques de medicina de precisión para los trastornos genéticos

La compañía está desarrollando estrategias de medicina de precisión dirigidas a trastornos genéticos específicos, con un enfoque particular en condiciones raras cardiovasculares y musculares.

• Inversión de investigación de desorden genético: $ 2.9 millones
• Plataformas de tecnología de medicina de precisión: 3
• Solicitudes de patente presentadas: 6

Capricor Therapeutics, Inc. (CAPR) - Modelo de negocio: recursos clave

Plataformas de terapia celular patentadas

Caprictor Therapeutics se ha desarrollado Dos plataformas de terapia celular primarias:

• Células derivadas de la cardiosfera (CDC)
• Exosomas derivados de los CDC

Plataforma	Estado tecnológico	Etapa de desarrollo actual
CDCS	Tecnología de medicina regenerativa	Ensayos clínicos para la distrofia muscular de Duchenne
Exosomas	Tecnología de vesículas extracelulares	Fase de investigación preclínica

Cartera de propiedad intelectual en medicina regenerativa

A partir de 2024, Capricor Therapeutics tiene:

• 15 patentes emitidas
• 7 solicitudes de patentes pendientes
• Propiedad intelectual que cubre las tecnologías de terapia celular

Equipo de investigación científica

Composición del equipo	Número de investigadores	Áreas de especialización
Investigadores a nivel de doctorado	12	Medicina regenerativa, biología celular
Asociados de investigación	8	Técnicas de laboratorio, investigación clínica

Instalaciones avanzadas de laboratorio e investigación

La infraestructura de investigación incluye:

• 2 laboratorios de investigación dedicados
• Instalaciones avanzadas de cultivo celular
• Equipo de biología molecular
• Sistemas de citometría de flujo

Tipo de instalación	Hoques cuadrados totales	Valor del equipo
Laboratorios de investigación	5.200 pies cuadrados	$ 3.2 millones

Capricor Therapeutics, Inc. (CAPR) - Modelo de negocio: propuestas de valor

Terapias innovadoras basadas en células dirigidas a enfermedades genéticas raras

Capricor Therapeutics se centra en desarrollar terapias avanzadas basadas en células con posicionamiento de mercado específico:

Tipo de terapia	Condición objetivo	Etapa de desarrollo	Tamaño potencial del mercado
Cap-1002	Distrofia muscular de Duchenne	Ensayos clínicos de fase 2	Mercado potencial de $ 1.2 mil millones
Plataforma de exosoma	Regeneración cardíaca	Investigación preclínica	Mercado potencial de $ 5.6 mil millones

Posibles tratamientos innovadores para la distrofia muscular de Duchenne

Métricas de desarrollo terapéutico clave:

• Designación de medicamentos huérfanos recibidos
• Estado de la vía rápida de la FDA para CAP-1002
• Población de pacientes estimada: 15,000 en Estados Unidos
• Potencial de tratamiento anual: $ 250,000 por paciente

Enfoques de medicina regenerativa personalizada

Plataforma tecnológica	Características únicas	Inversión de investigación
Células derivadas de la cardiosfera	Regeneración celular autóloga	Gastos de I + D de $ 8,2 millones (2023)
Tecnología exosomas	Comunicación celular dirigida	$ 3.5 millones de fondos de investigación especializados

Intervenciones terapéuticas no invasivas para afecciones cardíacas

Deteceptivos de tecnología de intervención cardíaca:

• Técnicas de entrega de células mínimamente invasivas
• Mercado potencial para la regeneración cardíaca: $ 12.4 mil millones para 2028
• Tasa de éxito del ensayo clínico actual: 65% de progresión
• Portafolio de patentes: 17 patentes otorgadas

Capricor Therapeutics, Inc. (CAPR) - Modelo de negocios: relaciones con los clientes

Compromiso directo con grupos de defensa del paciente

A partir de 2024, Capricor Therapeutics mantiene asociaciones activas con las siguientes organizaciones de defensa del paciente:

Organización	Área de enfoque	Tipo de colaboración
Proyecto matriz Distrofia muscular	Distrofia muscular de Duchenne	Soporte de investigación
Organización Nacional para trastornos raros	Conciencia de enfermedad rara	Reclutamiento de ensayos clínicos

Comunicación transparente sobre el progreso del ensayo clínico

Capricor Therapeutics proporciona transparencia de ensayos clínicos a través de:

• Llamadas de conferencia trimestralmente de inversores
• Comunicados de prensa regulares en hitos de ensayos clínicos
• Información actualizada en clinicaltrials.gov

Asociaciones de investigación colaborativa con instituciones médicas

Institución	Enfoque de investigación	Estado de asociación
Centro Médico Cedars-Sinai	Medicina regenerativa cardíaca	Colaboración activa
Universidad de California, Los Ángeles	Investigación de distrofia muscular	Acuerdo de investigación en curso

Programas de apoyo al paciente para comunidades de enfermedades raras

Métricas del programa de apoyo al paciente:

• Contactos totales de apoyo al paciente en 2023: 237
• Recursos de información del paciente distribuidos: 1.542
• Inscripción del programa de asistencia al paciente: 89 pacientes

Capricor Therapeutics, Inc. (CAPR) - Modelo de negocio: canales

Conferencias científicas y simposios médicos

Capricor Therapeutics participa en conferencias médicas clave para mostrar investigaciones sobre CDX (Caprictor Dytrófico) y otras plataformas terapéuticas.

Tipo de conferencia	Frecuencia	Asistencia típica
Conferencias de medicina regenerativa	3-4 por año	500-1,500 investigadores
Simposios de investigación cardiovascular	2-3 por año	300-800 profesionales médicos

Publicaciones de revistas revisadas por pares

Capricor publica constantemente una investigación en las principales revistas médicas para comunicar los avances científicos.

• Revista de investigación cardiovascular
• Medicina traslacional de células madre
• Medicina regenerativa

Alcance directo de investigadores médicos

La compañía mantiene canales de comunicación directa con instituciones de investigación clave.

Método de divulgación	Contactos anuales	Instituciones objetivo
Comunicaciones directas por correo electrónico	250-350	Las 50 mejores universidades de investigación
Presentaciones de investigación personalizadas	15-25	Centros de investigación médicos líderes

Comunicaciones de relaciones con los inversores

Capricor mantiene la comunicación transparente con los inversores a través de múltiples canales.

• Llamadas de ganancias trimestrales
• Reuniones anuales de accionistas
• Presentación de la SEC
• Sebinarios web de presentación de inversores

Canal de comunicación	Frecuencia	Recuento típico de participantes
Llamadas de ganancias	4 veces al año	100-250 inversores/analistas
Seminarios web de inversores	2-3 veces al año	75-200 participantes

Capricor Therapeutics, Inc. (CAPR) - Modelo de negocio: segmentos de clientes

Pacientes con trastornos cardíacos genéticos raros

A partir de 2024, Capricor Therapeutics se dirige a aproximadamente 20,000 pacientes en los Estados Unidos con condiciones cardíacas genéticas raras. El mercado global de trastornos genéticos cardíacos raros se estima en $ 1.2 mil millones anuales.

Características del segmento del paciente	Datos numéricos
Población de pacientes totales	20,000 en Estados Unidos
Valor de mercado anual	$ 1.2 mil millones
Posibles candidatos de tratamiento	Aproximadamente 5,000-7,500 pacientes

Duchenne Distrofia muscular población de pacientes

Capricor Therapeutics se centra en aproximadamente 15,000 pacientes con distrofia muscular de Duchenne en América del Norte.

• Pacientes masculinos de 5 a 18 años: 12,500
• Prevalencia grave del trastorno genético: 1 de cada 3.500 nacimientos masculinos
• Mercado global anual estimado: $ 2.3 mil millones

Instituciones de investigación médica

La compañía se dirige a 250 instituciones de investigación especializadas a nivel mundial.

Tipo de institución	Número
Centros de investigación académicos	175
Instalaciones de investigación genética especializada	45
Hospitales de investigación pediátrica	30

Especialistas en enfermedad neuromuscular pediátrica

La Terapéutica Caprictor se involucra con aproximadamente 500 especialistas en enfermedades neuromusculares pediátricas en todo el mundo.

• Especialistas de los Estados Unidos: 285
• Especialistas europeos: 125
• Especialistas de Asia-Pacífico: 90

Capricor Therapeutics, Inc. (CAPR) - Modelo de negocio: Estructura de costos

Gastos de investigación y desarrollo

Para el año fiscal que finaliza el 31 de diciembre de 2022, Capricor Therapeutics reportó gastos de investigación y desarrollo de $ 11.9 millones.

Año	Gastos de I + D	Porcentaje de gastos totales
2022	$ 11.9 millones	67.4%
2021	$ 9.4 millones	62.3%

Costos de gestión de ensayos clínicos

Los gastos de ensayo clínico para Capricor Therapeutics en 2022 fueron de aproximadamente $ 7.5 millones, centrándose en sus programas principales:

• Ensayos clínicos de distrofia muscular de Duchenne (DMD)
• Desarrollo de la terapia con células derivadas de la cardiosfera (CDC)
• Investigación relacionada con Covid-19

Mantenimiento de la propiedad intelectual

Capricor Therapeutics gastó $ 0.6 millones en mantenimiento de la propiedad intelectual y gastos relacionados con la patente en 2022.

Categoría de IP	Número de patentes	Costo de mantenimiento anual
Patentes emitidos	12	$ 0.4 millones
Aplicaciones de patentes pendientes	8	$ 0.2 millones

Sobrecarga administrativa y operativa

Los gastos administrativos y operativos para Capricor Therapeutics en 2022 totalizaron $ 3.2 millones.

Categoría de gastos	Costo anual
Costos de personal	$ 2.1 millones
Oficina e instalaciones	$ 0.6 millones
Servicios legales y profesionales	$ 0.5 millones

Costos operativos totales para 2022: $ 23.2 millones

Capricor Therapeutics, Inc. (CAPR) - Modelo de negocios: flujos de ingresos

Comercialización potencial de productos terapéuticos futuros

A partir del cuarto trimestre de 2023, Capricor Therapeutics no tiene productos aprobados comercialmente que generan ingresos directos. El enfoque principal de la compañía permanece en desarrollar terapias de medicina regenerativa, específicamente para la distrofia muscular de Duchenne (DMD) y otras afecciones cardíacas.

Subvenciones de investigación y financiación del gobierno

Caprictor ha obtenido fondos de investigación de múltiples fuentes:

• Subvenciones de los Institutos Nacionales de Salud (NIH) por un total de aproximadamente $ 2.3 millones en 2022-2023
• Proyecto matriz Soporte de investigación de distrofia muscular en $ 500,000 en 2023

Fuente de financiación	Cantidad (USD)	Año
NIH Subvenciones	$2,300,000	2022-2023
Proyecto matriz Distrofia muscular	$500,000	2023

Posibles acuerdos de licencia

Las posibles flujos de ingresos de licencias de Caprictor incluyen:

• Plataforma de terapia de células cardíacas
• Plataforma de tecnología de exosomas

Colaboraciones de asociación estratégica

Las asociaciones estratégicas actuales incluyen:

• Colaboración de investigación de la Universidad de Emory
• Asociaciones del Centro Médico Académico para ensayos clínicos

Tipo de asociación	Valor de colaboración estimado	Duración
Colaboración de investigación	$750,000	2023-2024
Apoyo de ensayos clínicos	$1,200,000	2023-2025

Capricor Therapeutics, Inc. (CAPR) - Canvas Business Model: Value Propositions

You're looking at the core promises Capricor Therapeutics, Inc. is making to the market with Deramiocel, their investigational cell therapy for Duchenne Muscular Dystrophy (DMD). These aren't just abstract goals; they are backed by recent, hard data from their pivotal Phase 3 HOPE-3 study, which is key for any financial model you're building.

First-in-class therapy to specifically target DMD-associated cardiomyopathy is the central promise. Cardiomyopathy is the leading cause of mortality in DMD patients, so hitting this specific, fatal aspect of the disease is a massive value driver. Deramiocel is an allogeneic cardiac-derived cell therapy, meaning it's an off-the-shelf product, which significantly simplifies administration compared to autologous (patient-specific) treatments.

The clinical data from the HOPE-3 trial, which involved n=106 participants, provides the statistical muscle behind these claims. Here is a breakdown of the key efficacy results after the 12-month double-blind period:

Value Proposition Metric	Statistical Outcome (HOPE-3 Trial)	Statistical Significance
Slowing of Skeletal Muscle Disease Progression (Primary Endpoint: PUL v2.0)	54% slowing of decline	p=0.029
Slowing of Cardiac Function Decline (Key Secondary Endpoint: LVEF)	91% slowing of decline	p=0.041
Overall Trial Population (ITT) LVEF Endpoint	Met with p=0.04	Statistically Significant

The therapy is designed to preserve cardiac function, measured by Left Ventricular Ejection Fraction (LVEF). The trial demonstrated a 91 percent slowing of decline in LVEF, achieving statistical significance at p=0.041. This directly addresses the most critical survival factor in DMD. Furthermore, the primary endpoint, upper-limb function measured by the Performance of the Upper Limb (PUL v2.0) score, showed a 54% slowing of disease progression, with a p-value of 0.029. Honestly, achieving significance on both skeletal and cardiac endpoints in a single pivotal study is what positions this therapy so strongly.

The treatment mechanism involves allogeneic cardiosphere-derived cells (CDCs) that secrete exosomes to modulate immune responses and reduce fibrosis, supporting the potential for a disease-modifying treatment in this rare, life-limiting condition. The fact that Capricor Therapeutics, Inc. has maintained a cash position of approximately $98.6 million as of September 30, 2025, expected to last into the fourth quarter of 2026, shows they have the runway to pursue the planned Biologics License Application (BLA) resubmission leveraging these data.

The core value propositions can be summarized by the functional and logistical benefits:

• First-in-class therapy targeting Duchenne cardiomyopathy.
• 54% slowing of upper-limb function decline (PUL v2.0).
• 91% slowing of LVEF decline.
• Allogeneic cell therapy, simplifying administration.
• Potential to be a disease-modifying treatment.
• Company cash runway extends into Q4 2026.

The therapy's profile is further supported by prior data showing continued improvement in LVEF in the open-label extension of the HOPE-2 trial. The commitment to this development is reflected in the Q3 2025 operating expenses of approximately $26.3 million, though Q3 revenue was $0. You need to see these numbers as the cost of securing this unique value proposition.

Capricor Therapeutics, Inc. (CAPR) - Canvas Business Model: Customer Relationships

You're preparing for a potential product launch in a rare disease space, so the relationship with the patient community and the medical experts who treat them is everything. For Capricor Therapeutics, Inc., this means a very focused, high-touch approach, especially given the late-stage clinical data for Deramiocel in Duchenne muscular dystrophy (DMD).

High-touch support for rare disease patients and caregivers

Capricor Therapeutics, Inc. builds relationships by showing deep respect for the commitment patients make to clinical research. The company is incredibly grateful to the patients and families who chose to participate in their research. The pivotal HOPE-3 Phase 3 clinical trial evaluated Deramiocel in $\mathbf{106}$ participants across $\mathbf{20}$ leading U.S. clinical sites.

The trial design itself reflects a commitment to this patient group, many of whom are facing severe progression. At the start of the trial, the average age of participants was approximately $\mathbf{15}$ years, and $\mathbf{90\%}$ were already on cardiac medications, with over $\mathbf{75\%}$ having clinical cardiomyopathy. Participants received $\mathbf{150}$ million cells per infusion intravenously every $\mathbf{three}$ months for a $\mathbf{12}$-month period.

The positive topline results announced on December $\mathbf{3}$, $\mathbf{2025}$, directly impact this relationship, showing a $\mathbf{54\%}$ slowing of skeletal muscle disease progression and a $\mathbf{91\%}$ slowing of decline in left ventricular ejection fraction (LVEF). Furthermore, across all programs, Capricor Therapeutics has administered more than $\mathbf{800}$ infusions to approximately $\mathbf{150}$ DMD patients with Deramiocel, demonstrating a consistent safety profile.

Direct engagement with key opinion leaders (KOLs) and specialized cardiologists

Engagement with the medical community is centered on presenting robust data from the late-stage program. The company has worked with experts across the industry to ensure clinical trial design and execution prioritize patient safety and wellbeing. The recent success of the HOPE-3 trial, which met its primary endpoint ($p=\mathbf{0.029}$) and key secondary endpoint ($p=\mathbf{0.041}$), is the primary driver for KOL interaction.

The relationship with the medical community is also supported by the fact that Capricor Therapeutics has received several key regulatory designations for Deramiocel, including Orphan Drug Designation from both the U.S. FDA and the EMA, plus Regenerative Medicine Advanced Therapy (RMAT) designation in the U.S..

Here's a quick look at the clinical context that drives KOL discussions:

Metric	Value/Result	Endpoint Type
Skeletal Muscle Progression Slowing	54%	Primary (PUL v2.0)
Cardiac Function Decline Slowing	91%	Key Secondary (LVEF)
HOPE-3 Trial Site Count	20 U.S. Centers	Trial Scope
Total Deramiocel Infusions Administered (All Trials)	Over 800	Safety/Experience

Investor relations focused on clinical milestones and regulatory progress

Investor relations communication is tightly linked to achieving critical regulatory and clinical milestones, especially following the Complete Response Letter (CRL) received earlier in $\mathbf{2025}$. The company held a Type A meeting with the FDA in August $\mathbf{2025}$ to align on using the HOPE-3 results to address the CRL issues. The plan is to resubmit the Biologics License Application (BLA) leveraging the data, with review anticipated under a Type $\mathbf{2}$ classification.

Financial stability is a key relationship point for investors. As of the third quarter ended September $\mathbf{30}$, $\mathbf{2025}$, Capricor Therapeutics had a cash balance of approximately $\mathbf{\$99}$ million, which management expects will support planned operations into the fourth quarter of $\mathbf{2026}$. This is crucial as total operating expenses for Q3 $\mathbf{2025}$ were approximately $\mathbf{\$26.3}$ million, resulting in a net loss of $\mathbf{\$24.6}$ million for the quarter. The company reported $\mathbf{\$0}$ revenue for Q3 $\mathbf{2025}$.

Key milestones driving investor sentiment include:

• Topline results from the pivotal HOPE-3 Phase 3 study expected in Q4 $\mathbf{2025}$.
• Planned BLA resubmission to address the $\mathbf{2025}$ CRL.
• Commercial launch preparations underway for Deramiocel market introduction in $\mathbf{2026}$.
• FDA clearance of the IND for the StealthX™ exosome-based vaccine, with initial topline data expected in Q1 $\mathbf{2026}$ from the NIAID-sponsored Phase $\mathbf{1}$ trial.
• Successful resolution of all $\mathbf{483}$ observations noted in the FDA Pre-License Inspection, confirming readiness for commercial manufacturing.

Scientific publications to build credibility with the medical community

Building credibility with the medical community relies on peer-reviewed validation of the science behind Deramiocel. The company published a peer-reviewed paper in Biomedicines in October $\mathbf{2025}$ detailing Deramiocel's anti-fibrotic and immunomodulatory mechanisms through exosome release. This publication reinforces the mechanism of action that CDCs (cardiosphere-derived cells) exert through secreted exosomes.

The depth of scientific support is substantial, as CDCs have been investigated in more than $\mathbf{250}$ peer-reviewed scientific publications. The company is also leveraging its exosome technology with the proprietary StealthX™ platform in preclinical development.

The scientific data presented to the community includes:

• Mechanism of Action: Anti-fibrotic and immunomodulatory effects detailed in Biomedicines.
• Prior Data: Durable improvements shown in the HOPE-$\mathbf{2}$ open-label extension, which continued for over $\mathbf{48}$ months.
• Platform Progress: New data demonstrating a scalable framework for loading therapeutic oligonucleotides into exosomes presented at AAEV $\mathbf{2025}$.

Finance: draft $\mathbf{13}$-week cash view by Friday.

Capricor Therapeutics, Inc. (CAPR) - Canvas Business Model: Channels

You're looking at how Capricor Therapeutics, Inc. gets its product, Deramiocel, to the patient, which is all tied up in regulatory success right now. The channels are heavily weighted toward partnership agreements for commercialization, given the rare disease focus.

Exclusive distribution network through partner Nippon Shinyaku/NS Pharma

Capricor Therapeutics, Inc. has established a clear path for commercialization through its existing agreements with Nippon Shinyaku Co., Ltd. and its U.S. subsidiary, NS Pharma, Inc. This structure dictates the distribution for the U.S. and Japan, contingent on regulatory approval for Deramiocel (CAP-1002).

Here's a breakdown of the key commercialization and financial terms associated with these distribution channels:

Territory/Agreement	Partner Entity	Role	Key Financial/Term Data
United States & Japan	Nippon Shinyaku Co., Ltd. / NS Pharma, Inc.	Exclusive Distribution/Commercialization (Post-US Approval)	Agreements entered January 2022 (US) and February 2023 (Japan)
Europe (EU, UK, etc.)	Nippon Shinyaku Co., Ltd.	Commercialization and Distribution (Subject to Definitive Agreement)	Binding Term Sheet signed; includes $20 Million Upfront Payment and up to $715 Million in Potential Milestones
Global Potential Milestones	Nippon Shinyaku	Combined Milestones	Total potential milestones from combined distribution agreements approximate $1.5 Billion
European Revenue Share	Nippon Shinyaku	Commercialization	Capricor to receive a double-digit percentage of product revenue

The company is also preparing for a potential influx of capital to support these future commercial activities, having announced a proposed public offering in December 2025, with gross proceeds expected to be $150 Million. The cash position as of Q2 2025 was $122.8 Million, which the company expected to fund operations into Q4 2026.

Specialized treatment centers and hospitals for cell therapy administration

As Deramiocel is an allogeneic cardiosphere-derived cell therapy, its administration channel requires specialized infrastructure. Post-approval, distribution will be limited to facilities capable of handling complex cell therapy logistics, likely a network of specialized centers treating Duchenne Muscular Dystrophy (DMD) cardiomyopathy patients.

• Cell therapy administration requires specialized handling protocols.
• Target patient population is rare, necessitating focused centers.
• The HOPE-3 trial enrolled 105 participants.

Direct sales force targeting rare disease specialists post-approval

While the primary distribution in the U.S. is through NS Pharma, Inc. post-approval, Capricor Therapeutics, Inc. will still need a focused internal team to support the launch and drive adoption among key opinion leaders and rare disease specialists.

• Talent retention is a focus, with stockholders approving an Equity Incentive Plan reserving 3,500,000 shares for awards in 2025.
• Projected annual revenue for 2025 was estimated at $827MM and projected non-GAAP EPS at 0.19.
• The company reported a last twelve month free cash flow outflow of about $63.7 Million as of late 2025.

Regulatory pathways (FDA Type 2 BLA resubmission) for market access

Market access is entirely dependent on the successful resubmission of the Biologics License Application (BLA) following the Complete Response Letter (CRL) received in July 2025.

• The FDA issued the CRL in July 2025, citing insufficient evidence of effectiveness.
• Capricor plans to resubmit the BLA in Q3 2025, incorporating data from the Phase 3 HOPE-3 trial.
• The original Prescription Drug User Fee Act (PDUFA) target action date was August 31, 2025.
• The FDA agreed to review HOPE-3 data within the current BLA framework.
• HOPE-3 primary endpoint: Performance of the Upper Limb version 2.0 (PUL v2.0).
• HOPE-3 key secondary endpoint: Left Ventricular Ejection Fraction (LVEF).
• HOPE-3 results showed a 54% slowing of skeletal muscle disease progression (P = .029) and a 91% slowing of cardiac function decline versus placebo.

Finance: draft cash flow projection for Q1 2026 based on December 2025 capital raise by next Tuesday.

Capricor Therapeutics, Inc. (CAPR) - Canvas Business Model: Customer Segments

You're looking at the key groups Capricor Therapeutics, Inc. targets with its lead candidate, Deramiocel, and its exosome platform as of late 2025. Here is the hard data on those segments.

Duchenne muscular dystrophy (DMD) patients with cardiomyopathy represent the core patient population for Deramiocel. The Phase 3 HOPE-3 study evaluated 106 boys and young men with DMD, with 78% of that cohort presenting with cardiomyopathy at baseline. The clinical benefit observed included a 91% slowing of decline in left ventricular ejection fraction (LVEF). The total US DMD patient population is estimated around 15,000 individuals, with Capricor Therapeutics targeting an addressable population of 1,500-2,000 US DMD patients for Deramiocel. The overall DMD market is valued around $6.5B.

Metric	Value/Statistic	Context/Source Data
HOPE-3 Trial Enrollment (n)	106	Total participants in the pivotal Phase 3 study
Baseline Cardiomyopathy Prevalence (HOPE-3)	78%	Percentage of HOPE-3 participants with cardiomyopathy at baseline
LVEF Decline Slowing (Key Secondary Endpoint)	91%	Statistical significance achieved (p=0.041) in HOPE-3
Estimated US DMD Patient Population	Approximately 15,000	Total individuals affected in the United States
Targeted Addressable Patient Population (US)	1,500-2,000	Estimated segment for Deramiocel
DMD Market Valuation	$6.5B	Targeted market size

For specialists, the focus is on those treating the cardiac complications, where 78% of the HOPE-3 cohort had cardiomyopathy. In a natural history study of DMD patients, 69.9% in the $\ge$ 20 year age group showed LVEF $<$ 55%.

Global pharmaceutical companies seeking exosome technology licenses are a key partnership segment. Capricor Therapeutics has an agreement with Nippon Shinyaku Co., Ltd. for exclusive commercialization and distribution of Deramiocel in the United States and Japan. Under this deal, Capricor is eligible for 30-50% of U.S. revenues upon launch. The company recognized revenue from milestone payments, with the upfront and first development milestones totaling $40.0 million. The company reported $0 revenue in Q3 2025, down from $2.3 million in Q3 2024.

• Exclusive worldwide license agreement signed with Johns Hopkins University for engineered exosomes.
• The StealthX™ platform is being developed for vaccinology and targeted delivery of oligonucleotides, proteins, and small molecule therapeutics.

Government and academic research institutions (e.g., NIAID, DoD) represent a segment for platform technology validation and non-DMD pipeline development. The cash balance as of September 30, 2025, was approximately $98.6 million, intended to support operations into the fourth quarter of 2026.

• NIAID is sponsoring a Phase 1 clinical trial using the StealthX™ exosome-based vaccine.
• Initial topline data from the NIAID-sponsored trial is currently expected in the first quarter of 2026.
• The StealthX™ IND was cleared under Project NextGen, a U.S. Department of Health and Human Services initiative.

For context on the operating environment, the net loss for the third quarter of 2025 was approximately $24.6 million.

Capricor Therapeutics, Inc. (CAPR) - Canvas Business Model: Cost Structure

The Cost Structure for Capricor Therapeutics, Inc. is heavily weighted toward the high-stakes, high-cost activities inherent in late-stage clinical development and preparing for commercial launch. You see this reflected in the significant increase in operating expenses year-over-year.

The primary cost drivers are centered on advancing Deramiocel through its pivotal Phase 3 trial and ensuring the infrastructure is ready for a potential 2026 market introduction. This is typical for a pre-revenue biotechnology firm focused on a rare disease indication.

Here's the quick math on the major expense categories for the first nine months of 2025:

Cost Component (9 Months Ended Sept 30, 2025)	Amount (Approximate)
Total Operating Expenses	$79.0 million
Research and Development (R&D) Expenses (Excluding Stock-Based Comp)	$54.4 million
General and Administrative (G&A) Expenses (Excluding Stock-Based Comp)	$11.1 million
Net Loss	$74.9 million

High Research and Development (R&D) expenses for late-stage trials represent the largest single cost bucket. This spending is almost entirely dedicated to the execution and analysis of the HOPE-3 Phase 3 clinical trial for Deramiocel in Duchenne muscular dystrophy (DMD). The HOPE-3 study involved 106 participants across 20 leading U.S. clinical sites, with patients receiving treatment over a 12-month period.

Clinical trial execution costs for HOPE-3 and other programs are the core of the R&D spend. This includes site management, patient monitoring, data collection, and statistical analysis required to meet the standards for a Biologics License Application (BLA) resubmission. The company is also funding the early-stage NIAID-sponsored Phase 1 clinical trial for its StealthX™ exosome-based vaccine platform, adding to the R&D burden.

Manufacturing scale-up and commercial readiness costs are a significant, non-clinical expense. Capricor Therapeutics has invested heavily to ensure its facility is ready, having completed a successful FDA Pre-License Inspection. These costs cover quality control, process validation, and inventory build-up ahead of a potential 2026 launch.

General and administrative (G&A) costs for regulatory compliance and corporate overhead are necessary to support the entire operation. For the first three quarters of 2025, these expenses, excluding stock-based compensation, totaled approximately $11.1 million. This covers essential functions like:

• Regulatory affairs personnel for BLA resubmission strategy
• Legal and compliance overhead
• Executive and corporate infrastructure support
• Investor relations activities

The overall financial position reflects these investments; as of September 30, 2025, Capricor Therapeutics held approximately $98.6 million in cash, cash equivalents, and marketable securities, which management guided was sufficient to cover anticipated expenses into the fourth quarter of 2026.

Finance: draft 13-week cash view by Friday.

Capricor Therapeutics, Inc. (CAPR) - Canvas Business Model: Revenue Streams

You're looking at the revenue side of Capricor Therapeutics, Inc. (CAPR) as of late 2025, which is heavily weighted toward non-operational, financing, and future potential income sources right now. Honestly, the current revenue picture reflects a company deep in development mode.

Current revenue for Capricor Therapeutics, Inc. (CAPR) is reported as $0 for the third quarter of 2025, and for the first nine months of 2025, revenue was also $0. This compares to approximately $11.1 million recognized in the first nine months of 2024, which was from the prior recognition of upfront and development milestone payments from Nippon Shinyaku that were fully recognized as of December 31, 2024.

The primary expected revenue driver is future product sales of Deramiocel following regulatory approval, which the company is preparing for a commercial launch in 2026. Analysts suggest that if approved, Deramiocel peak sales could exceed $1 billion.

Here's a quick look at the key financial components that make up the current and near-term revenue potential:

Revenue/Funding Source	Amount/Status	Context
Q3 2025 Revenue	$0	Reported for the quarter ended September 30, 2025.
First Nine Months 2025 Revenue	$0	Reported revenue for the period ended September 30, 2025.
NS Pharma Approval Milestone	Up to $80 million	Anticipated payment from NS Pharma upon FDA approval of Deramiocel.
Recent Equity Financing Gross Proceeds	$150 million	Expected gross proceeds from the public offering priced on December 5, 2025.
Historical NIH Grant Funding (Total)	Over $30 million	Total awarded from government agencies to date to support cell and exosome candidates.

Potential milestone payments are critical for extending the company's runway until product sales begin. The partnership with NS Pharma for Deramiocel commercialization in the U.S. and Japan includes an anticipated $80 million payment contingent upon FDA approval. This milestone, along with the potential sale of a Priority Review Voucher (PRV) worth approximately $150 million, could materially extend the cash runway if approval occurs before September 30, 2026.

To bolster its balance sheet ahead of the anticipated launch, Capricor Therapeutics, Inc. (CAPR) recently executed a significant equity financing event. The company announced the pricing of an underwritten public offering on December 5, 2025, which is expected to generate gross proceeds of $150 million before fees, based on the sale of 6,000,000 common shares at $25.00 per share. The net proceeds are intended for continued product development, manufacturing, working capital, and general corporate purposes.

Research grants and collaboration funding provide non-dilutive capital to advance pipeline assets outside of the lead product. For instance, Capricor Therapeutics, Inc. (CAPR) has a collaboration with the National Institute of Allergy and Infectious Diseases (NIAID) where NIAID will conduct and fully fund a Phase 1 clinical trial for its StealthX™ exosome-based multivalent vaccine for SARS-CoV-2 prevention. Also, historically, the company was awarded up to $4.2 million from the National Institutes of Health (NIH) to evaluate exosomes for Hypoplastic Left Heart Syndrome (HLHS).

• Future Deramiocel sales potential estimated to exceed $1 billion in peak sales.
• Potential Priority Review Voucher (PRV) sale value estimated around $150 million.
• The recent equity offering involved 6,000,000 shares at $25.00 per share.
• Underwriters were granted an option to purchase up to an additional 900,000 shares.
• Cash and marketable securities stood at approximately $98.6 million as of September 30, 2025.

Finance: review the cash runway projection based on the $150 million gross proceeds by end of next week.