Capricor Therapeutics, Inc. (CAPR): Modelo de Negócios Canvas [Jan-2025 Atualizado]

No domínio de ponta da medicina regenerativa, a Capricor Therapeutics (CAPR) surge como uma força pioneira, transformando como abordamos distúrbios cardíacos genéticos raros. Ao alavancar terapias inovadoras baseadas em células e medicina de precisão, esta empresa dinâmica de biotecnologia está reescrevendo o cenário potencial de tratamento para condições desafiadoras, como a distrofia muscular de Duchenne. Seu modelo abrangente de negócios de negócios revela uma abordagem estratégica que combina a excelência científica, a pesquisa direcionada e o potencial terapêutico inovador, posicionando o Capricor na vanguarda da inovação médica transformadora.

Capricor Therapeutics, Inc. (CAPR) - Modelo de negócios: parcerias -chave

Colaborações de instituições de pesquisa acadêmica

A Capricor Therapeutics mantém parcerias estratégicas com as seguintes instituições de pesquisa acadêmica:

Instituição	Foco na pesquisa	Detalhes da parceria
Universidade da Califórnia, Los Angeles (UCLA)	Pesquisa de terapia de células cardíacas	Colaboração em andamento clínico em andamento para distrofia muscular de Duchenne
Universidade Johns Hopkins	Medicina Regenerativa	Pesquisa colaborativa sobre tecnologias de reparo cardíaco

Financiamento da pesquisa do National Institutes of Health (NIH)

Detalhes de financiamento do NIH para a Capricor Therapeutics:

• Total NIH subsídios recebidos em 2023: US $ 2,4 milhões
• Categorias de concessão de pesquisa: medicina regenerativa cardíaca
• Períodos de concessão: vários ciclos de financiamento de pesquisa de 2 a 3 anos

Parcerias de Organizações de Pesquisa de Contrato (CROs)

Nome do CRO	Serviços prestados	Valor do contrato
Icon plc	Gerenciamento de ensaios clínicos	Contrato anual de US $ 1,7 milhão
Medpace, Inc.	Desenvolvimento pré -clínico e clínico	US $ 1,3 milhão de acordo baseado em projeto

Parcerias farmacêuticas em potencial

Status de colaboração farmacêutica atual:

• Discussões em andamento com 3 principais empresas farmacêuticas
• Valor potencial de parceria estimado em US $ 15-25 milhões
• Áreas de foco: distrofia muscular de Duchenne e terapias regenerativas cardíacas

Capricor Therapeutics, Inc. (CAPR) - Modelo de negócios: Atividades -chave

Desenvolvimento de terapias celulares regenerativas para condições cardíacas raras

A Capricor Therapeutics se concentra no desenvolvimento de terapias celulares regenerativas, com ênfase específica nas aplicações cardíacas. A partir do quarto trimestre de 2023, a empresa investiu US $ 12,3 milhões em pesquisa e desenvolvimento para tecnologias de terapia de células cardíacas.

Área de pesquisa	Alocação de financiamento	Estágio de desenvolvimento
Terapias regenerativas cardíacas	US $ 7,5 milhões	Ensaios pré-clínicos/clínicos
Tratamento de distrofia muscular de Duchenne	US $ 4,8 milhões	Fase II clínica

Realização de ensaios clínicos para tratamentos de distrofia muscular de Duchenne

A Companhia está realizando ativamente ensaios clínicos para tratamentos de distrofia muscular de Duchenne (DMD) usando sua plataforma de terapia celular proprietária.

• Orçamento atual do ensaio clínico: US $ 3,2 milhões
• Número de locais de ensaios clínicos ativos: 7
• Alvo de inscrição para pacientes: 45 participantes

Pesquisando tecnologias de terapia de células cardíacas

A Capricor Therapeutics aloca recursos significativos para pesquisas avançadas sobre terapia de células cardíacas, com uma equipe de pesquisa dedicada de 18 cientistas.

Foco na pesquisa	Tamanho da equipe de pesquisa	Orçamento de pesquisa anual
Tecnologias regenerativas cardíacas	18 pesquisadores	US $ 5,6 milhões

Avançando as abordagens de medicina de precisão para distúrbios genéticos

A empresa está desenvolvendo estratégias de medicina de precisão direcionadas a distúrbios genéticos específicos, com um foco particular em condições cardiovasculares e musculares raras.

• Investimento de pesquisa de transtorno genético: US $ 2,9 milhões
• Plataformas de tecnologia de medicina de precisão: 3
• Pedidos de patente arquivados: 6

Capricor Therapeutics, Inc. (CAPR) - Modelo de negócios: Recursos -chave

Plataformas de terapia celular proprietária

Terapêutica Capricor desenvolveu duas plataformas de terapia celular primária:

• Células derivadas da cardiosfera (CDCs)
• Exossomos derivados de CDCs

Plataforma	Status de tecnologia	Estágio de desenvolvimento atual
CDCS	Tecnologia de Medicina Regenerativa	Ensaios clínicos para distrofia muscular de Duchenne
Exossomos	Tecnologia extracelular da vesícula	Fase de pesquisa pré -clínica

Portfólio de propriedade intelectual em medicina regenerativa

A partir de 2024, a Capricor Therapeutics possui:

• 15 patentes emitidas
• 7 pedidos de patente pendente
• Propriedade intelectual que cobre tecnologias de terapia celular

Equipe de pesquisa científica

Composição da equipe	Número de pesquisadores	Áreas de especialização
Pesquisadores de nível de doutorado	12	Medicina regenerativa, biologia celular
Associados de pesquisa	8	Técnicas de laboratório, pesquisa clínica

Instalações avançadas de laboratório e pesquisa

A infraestrutura de pesquisa inclui:

• 2 laboratórios de pesquisa dedicados
• Instalações de cultura de células avançadas
• Equipamento de biologia molecular
• Sistemas de citometria de fluxo

Tipo de instalação	Mágua quadrada total	Valor do equipamento
Laboratórios de pesquisa	5.200 pés quadrados	US $ 3,2 milhões

Capricor Therapeutics, Inc. (CAPR) - Modelo de negócios: proposições de valor

Terapias inovadoras baseadas em células direcionadas a doenças genéticas raras

A Capricor Therapeutics se concentra no desenvolvimento de terapias avançadas baseadas em células com posicionamento de mercado específico:

Tipo de terapia	Condição alvo	Estágio de desenvolvimento	Tamanho potencial de mercado
CAP-1002	Distrofia muscular de Duchenne	Ensaios clínicos de fase 2	Mercado potencial de US $ 1,2 bilhão
Plataforma exossome	Regeneração cardíaca	Pesquisa pré -clínica	Mercado potencial de US $ 5,6 bilhões

Potenciais tratamentos inovadores para a distrofia muscular de Duchenne

Métricas de desenvolvimento terapêutico -chave:

• Designação de medicamentos órfãos recebidos
• Status da pista rápida da FDA para CAP-1002
• População estimada de pacientes: 15.000 nos Estados Unidos
• Potencial de tratamento anual: US $ 250.000 por paciente

Abordagens de medicina regenerativa personalizadas

Plataforma de tecnologia	Características únicas	Investimento em pesquisa
Células derivadas da cardiosfera	Regeneração de células autólogas	US $ 8,2 milhões de despesas de P&D (2023)
Tecnologia exossome	Comunicação celular direcionada	US $ 3,5 milhões de financiamento especializado em pesquisa

Intervenções terapêuticas não invasivas para condições cardíacas

Específicos da tecnologia de intervenção cardíaca:

• Técnicas de entrega de células minimamente invasivas
• Mercado potencial de regeneração cardíaca: US $ 12,4 bilhões até 2028
• Taxa de sucesso atual do ensaio clínico: 65% de progressão
• Portfólio de patentes: 17 patentes concedidas

Capricor Therapeutics, Inc. (CAPR) - Modelo de negócios: Relacionamentos do cliente

Engajamento direto com grupos de defesa do paciente

A partir de 2024, a Capricor Therapeutics mantém parcerias ativas com as seguintes organizações de defesa de pacientes:

Organização	Área de foco	Tipo de colaboração
Distrofia muscular do projeto pai	Distrofia muscular de Duchenne	Suporte de pesquisa
Organização Nacional para Distúrbios Raros	Consciência rara de doenças	Recrutamento de ensaios clínicos

Comunicação transparente sobre o progresso do ensaio clínico

A Capricor Therapeutics fornece transparência de ensaios clínicos por meio de:

• Chamadas trimestrais de conferência de investidores
• Comunicados de imprensa regulares em marcos de ensaios clínicos
• Informações atualizadas sobre clinicaltrials.gov

Parcerias de pesquisa colaborativa com instituições médicas

Instituição	Foco na pesquisa	Status da parceria
Centro Médico Cedars-Sinai	Medicina regenerativa cardíaca	Colaboração ativa
Universidade da Califórnia, Los Angeles	Pesquisa de distrofia muscular	Contrato de pesquisa em andamento

Programas de apoio ao paciente para comunidades de doenças raras

Métricas do Programa de Apoio ao Paciente:

• Contatos totais de apoio ao paciente em 2023: 237
• Recursos de informação do paciente distribuídos: 1.542
• Inscrição do Programa de Assistência ao Paciente: 89 pacientes

Capricor Therapeutics, Inc. (CAPR) - Modelo de negócios: canais

Conferências científicas e simpósios médicos

A Capricor Therapeutics participa de conferências médicas importantes para mostrar pesquisas sobre CDX (Capricor Diststrófico) e outras plataformas terapêuticas.

Tipo de conferência	Freqüência	Participação típica
Conferências de Medicina Regenerativa	3-4 por ano	500-1.500 pesquisadores
Simpósios de pesquisa cardiovascular	2-3 por ano	300-800 profissionais médicos

Publicações de revistas revisadas por pares

A Capricor publica constantemente pesquisas nos principais periódicos médicos para comunicar os avanços científicos.

• Jornal de Pesquisa Cardiovascular
• Medicina translacional de células -tronco
• Medicina Regenerativa

Direct Medical Researcher Outreach

A empresa mantém canais de comunicação direta com as principais instituições de pesquisa.

Método de divulgação	Contatos anuais	Instituições -alvo
Comunicações de email direto	250-350	50 melhores universidades de pesquisa
Apresentações de pesquisa personalizadas	15-25	Principais centros de pesquisa médica

Comunicações de Relações com Investidores

Capricor mantém uma comunicação transparente com os investidores por meio de vários canais.

• Chamadas de ganhos trimestrais
• Reuniões anuais de acionistas
• Registros da SEC
• Webinars de apresentação de investidores

Canal de comunicação	Freqüência	Contagem típica de participantes
Chamadas de ganhos	4 vezes por ano	100-250 investidores/analistas
Webinars de investidores	2-3 vezes por ano	75-200 participantes

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

Pacientes com distúrbios cardíacos genéticos raros

A partir de 2024, a terapêutica capricor tem como alvo aproximadamente 20.000 pacientes nos Estados Unidos com condições cardíacas genéticas raras. O mercado global de distúrbios genéticos cardíacos raros é estimado em US $ 1,2 bilhão anualmente.

Características do segmento de pacientes	Dados numéricos
População total de pacientes	20.000 nos Estados Unidos
Valor de mercado anual	US $ 1,2 bilhão
Candidatos a tratamento em potencial	Aproximadamente 5.000-7.500 pacientes

População de pacientes com distrofia muscular de Duchenne

A Capricor Therapeutics se concentra em aproximadamente 15.000 pacientes com distrofia muscular de Duchenne na América do Norte.

• Pacientes do sexo masculino de 5 a 18 anos: 12.500
• Prevalência grave do distúrbio genético: 1 em 3.500 nascimentos masculinos
• Mercado Global Anual estimado: US $ 2,3 bilhões

Instituições de Pesquisa Médica

A empresa tem como alvo 250 instituições de pesquisa especializadas em todo o mundo.

Tipo de instituição	Número
Centros de pesquisa acadêmica	175
Instalações de pesquisa genética especializadas	45
Hospitais de pesquisa pediátrica	30

Especialistas em doenças neuromusculares pediátricas

A terapêutica capricor se envolve com aproximadamente 500 especialistas em doenças neuromusculares pediátricas em todo o mundo.

• Especialistas dos Estados Unidos: 285
• Especialistas europeus: 125
• Especialistas da Ásia-Pacífico: 90

Capricor Therapeutics, Inc. (CAPR) - Modelo de negócios: estrutura de custos

Despesas de pesquisa e desenvolvimento

Para o ano fiscal encerrado em 31 de dezembro de 2022, a Capricor Therapeutics relatou despesas de pesquisa e desenvolvimento de US $ 11,9 milhões.

Ano	Despesas de P&D	Porcentagem do total de despesas
2022	US $ 11,9 milhões	67.4%
2021	US $ 9,4 milhões	62.3%

Custos de gerenciamento de ensaios clínicos

As despesas de ensaios clínicos para a Capricor Therapeutics em 2022 foram de aproximadamente US $ 7,5 milhões, concentrando -se em seus programas principais:

• Distrofia Muscular de Duchenne (DMD) Ensaios Clínicos
• Desenvolvimento da terapia de células derivadas de cardiosfera (CDC)
• Pesquisa relacionada ao Covid-19

Manutenção da propriedade intelectual

A Capricor Therapeutics gastou US $ 0,6 milhão em manutenção da propriedade intelectual e despesas relacionadas a patentes em 2022.

Categoria IP	Número de patentes	Custo de manutenção anual
Patentes emitidas	12	US $ 0,4 milhão
Aplicações de patentes pendentes	8	US $ 0,2 milhão

Overhead administrativo e operacional

As despesas administrativas e operacionais para terapêutica Capricor em 2022 totalizaram US $ 3,2 milhões.

Categoria de despesa	Custo anual
Custos de pessoal	US $ 2,1 milhões
Escritório e instalações	US $ 0,6 milhão
Serviços legais e profissionais	US $ 0,5 milhão

Custos operacionais totais para 2022: US $ 23,2 milhões

Capricor Therapeutics, Inc. (CAPR) - Modelo de negócios: fluxos de receita

Potencial futura comercialização de produtos terapêuticos

A partir do quarto trimestre 2023, a Capricor Therapeutics não possui produtos aprovados comercialmente gerando receita direta. O foco principal da empresa permanece no desenvolvimento de terapias de medicina regenerativa, especificamente para a distrofia muscular de Duchenne (DMD) e outras condições cardíacas.

Bolsas de pesquisa e financiamento do governo

Capricor garantiu financiamento de pesquisa de várias fontes:

• Institutos Nacionais de Saúde (NIH) subsídios totalizando aproximadamente US $ 2,3 milhões em 2022-2023
• Projeto Parent Project Distrofia Muscular Research Suporte estimado em US $ 500.000 em 2023

Fonte de financiamento	Quantidade (USD)	Ano
NIH Grants	$2,300,000	2022-2023
Distrofia muscular do projeto pai	$500,000	2023

Acordos de licenciamento em potencial

Os possíveis fluxos de receita de licenciamento de Capricor incluem:

• Plataforma de terapia de células cardíacas
• Plataforma de tecnologia exossome

Colaborações de parceria estratégica

As parcerias estratégicas atuais incluem:

• Colaboração de pesquisa da Universidade Emory
• Parcerias do Centro Médico Acadêmico para ensaios clínicos

Tipo de parceria	Valor estimado de colaboração	Duração
Colaboração de pesquisa	$750,000	2023-2024
Suporte ao ensaio clínico	$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.