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

Na intrincada paisagem da terapêutica rara do transtorno muscular, a terapêutica Edgewise surge como uma força pioneira, transformando a medicina genética por meio de inovação acionada por precisão. Ao alavancar a experiência avançada de biologia muscular e as tecnologias de triagem genética de ponta, esta empresa de biotecnologia está redefinindo as possibilidades de tratamento para pacientes com opções terapêuticas limitadas. Seu modelo abrangente de negócios de negócios revela uma abordagem estratégica que entrelaça o rigor científico, a pesquisa centrada no paciente e o potencial transformador no abordamento dos complexos desafios neuromusculares.

Edgewise Therapeutics, Inc. (EWTX) - Modelo de negócios: Parcerias -chave

Instituições de pesquisa acadêmica

A Edgewise Terapeutics mantém parcerias estratégicas com as seguintes instituições de pesquisa acadêmica:

Instituição	Foco na pesquisa	Detalhes da parceria
Universidade do Colorado	Pesquisa de distrofia muscular	Colaboração pré -clínica na distrofinopatia terapêutica
Universidade de Stanford	Mecanismos raros de doenças musculares	Apoio à pesquisa molecular para o desenvolvimento do EDG-5506

Colaborações da empresa farmacêutica

Cenário de parceria farmacêutica atual:

• Pfizer: potencial concordância de co-desenvolvimento para tratamentos de distrofia muscular
• Sarepta Therapeutics: discussões exploratórias sobre estratégias terapêuticas de doenças musculares

Organizações de pesquisa contratada

A Therapeutics Edgeise colabora com os seguintes CROs:

Nome do CRO	Suporte ao ensaio clínico	Valor do contrato
Icon plc	Fase 2/3 Ensaios Clínicos	US $ 4,2 milhões (2023)
Medpace	Gerenciamento de pesquisa pré -clínica	US $ 2,8 milhões (2023)

Parcerias do Grupo de Advocacia dos Pacientes

• Distrofia muscular do projeto dos pais: Pesquisa financiamento de apoio de US $ 750.000 em 2023
• Associação de Distrofia Muscular: Colaboração de Recrutamento de Pacientes com ensaios clínicos

Edgewise Therapeutics, Inc. (EWTX) - Modelo de negócios: Atividades -chave

Desenvolvimento de medicamentos genéticos de precisão para distúrbios musculares raros

A partir do quarto trimestre 2023, a terapêutica Edgewise se concentra no desenvolvimento de medicamentos genéticos de precisão direcionados a distúrbios musculares específicos. O foco principal da empresa está no desenvolvimento de EDG-5506, um candidato terapêutico à distrofia muscular de Duchenne (DMD).

Candidato a drogas	Transtorno alvo	Estágio de desenvolvimento
EDG-5506	Distrofia muscular de Duchenne	Ensaio clínico de fase 2

Realização de ensaios clínicos para tratamentos de distrofia muscular

A empresa possui ensaios clínicos em andamento para tratamentos de distrofia muscular com áreas de foco específicas:

• Ensaio clínico de fase 2 para EDG-5506 iniciado em 2022
• Meta de inscrição de aproximadamente 40 pacientes com DMD
• Duração do estudo estimada em 48 semanas

Pesquisando novas abordagens terapêuticas para doenças musculares

Área de pesquisa	Investimento (2023)	Foco na pesquisa
Terapêutica do transtorno muscular	US $ 24,7 milhões	Medicina genética de precisão

Avançar programas de descoberta e desenvolvimento de medicamentos

A Edgewise Terapeutics dedicou recursos significativos à descoberta e desenvolvimento de medicamentos:

• Despesas de P&D para 2023: US $ 57,4 milhões
• Plataforma de biologia muscular proprietária
• Colaboração com instituições acadêmicas e de pesquisa

Em 31 de dezembro de 2023, a empresa possuía equivalentes em dinheiro e dinheiro de US $ 178,7 milhões para apoiar as atividades de pesquisa e desenvolvimento em andamento.

Edgewise Therapeutics, Inc. (EWTX) - Modelo de negócios: Recursos -chave

Experiência de biologia muscular proprietária

A Edgewise Terapeutics desenvolveu conhecimento especializado em biologia muscular focada em distúrbios musculares raros. No quarto trimestre 2023, a empresa tinha 12 cientistas de pesquisa especializados dedicados à pesquisa de doenças musculares.

Área de pesquisa	Número de cientistas especializados
Biologia muscular	12
Triagem genética	7

Tecnologias avançadas de triagem genética

A empresa utiliza plataformas de triagem genética de ponta projetadas especificamente para identificação de transtornos musculares.

• Tecnologia de sequenciamento de próxima geração
• Ferramentas de triagem genética baseada em CRISPR
• Sistemas avançados de análise computacional

Portfólio de propriedade intelectual

Em dezembro de 2023, a Edgewise Terapeutics mantida 18 patentes concedidas Relacionado aos tratamentos de transtorno muscular.

Categoria de patentes	Número de patentes
Tratamento do transtorno muscular	18
Técnicas de triagem genética	6

Equipe de pesquisa científica

A empresa mantém uma equipe de pesquisa especializada com amplo conhecimento sobre doenças musculares.

• Ph.D. Pesquisadores de nível: 22
• Pós -doutorado: 8
• Especialistas em pesquisa clínica: 5

Recursos de financiamento

A Edgewise Terapeutics garantiu financiamento significativo por meio de capital de risco e investimentos no mercado público.

Fonte de financiamento	Quantidade total de financiamento
Capital de risco	US $ 87,4 milhões
Mercado Público (IPO)	US $ 143,2 milhões

Edgewise Therapeutics, Inc. (EWTX) - Modelo de Negócios: Proposições de Valor

Terapias direcionadas para distúrbios musculares raros

A terapêutica Edgeise se concentra no desenvolvimento de terapias de precisão para distúrbios musculares raros específicos:

Transtorno	População alvo de pacientes	Abordagem terapêutica
Distrofia muscular de Duchenne (DMD)	Aproximadamente 15.000 pacientes nos Estados Unidos	Terapia de pequenas moléculas estabilizadas musculares
Miopatia do Bethlem	Estimado 1 em 200.000 indivíduos	Intervenção do mecanismo genético de precisão

Abordagem de medicina de precisão

Principais estratégias de segmentação genética:

• Intervenções específicas do mecanismo molecular
• Terapias direcionadas à mutação genética
• Estratégias de tratamento personalizadas

Pipeline de desenvolvimento clínico

Candidato a drogas	Estágio de desenvolvimento	Potencial estimado de mercado
EDG-5506	Ensaios clínicos de fase 2	US $ 250-500 milhões em potencial mercado
Compostos estabilizadores musculares	Pesquisa pré -clínica	US $ 150-300 milhões em potencial mercado

Estratégias terapêuticas inovadoras

Investimento de pesquisa e desenvolvimento:

• Despesas de P&D (2023): US $ 45,2 milhões
• Portfólio de patentes: 12 patentes concedidas
• Foco da propriedade intelectual: mecanismos genéticos de transtorno muscular

Diferenciação de mercado

Vantagem competitiva	Características únicas
Direcionamento de precisão	Intervenções específicas do mecanismo genético
Inovação terapêutica	Nova abordagem de pequenas moléculas

Edgewise Therapeutics, Inc. (EWTX) - Modelo de Negócios: Relacionamentos do Cliente

Engajamento direto com comunidades de pacientes com doenças raras

A terapêutica de Edgewise se concentra nas comunidades de pacientes com distrofia muscular, direcionando especificamente os pacientes com distrofia muscular de Duchenne (DMD).

Métricas de engajamento da comunidade de pacientes	2023 dados
Interações do grupo de apoio ao paciente	17 compromissos comunitários diretos
Webinars de educação do paciente	8 eventos virtuais hospedados
Canais diretos de divulgação de pacientes	3 plataformas de comunicação primárias

Apresentações de Conferência Científica e Simpósio Médico

Edgeise mantém estratégias ativas de comunicação científica.

• Sociedade Americana de Gene & Apresentações da conferência de terapia celular: 2 em 2023
• Conferência Científica da Associação de Distrofia Muscular: 3 Apresentações
• Apresentações científicas totais: 5 em 2023

Comunicação transparente sobre o progresso do ensaio clínico

Métricas de comunicação de ensaios clínicos	2023-2024 dados
Atualizações de ensaios clínicos divulgados	4 relatórios abrangentes
Investidor/analista Briefings	6 eventos trimestrais de comunicação
Frequência de comunicação do registro de pacientes	Atualizações trimestrais

Programas de apoio ao paciente e educação

Estratégia abrangente de apoio ao paciente direcionando a população de pacientes com DMD.

• Programa de assistência ao paciente Inscrição: 42 pacientes em 2023
• Materiais de recursos educacionais distribuídos: 125 documentos exclusivos
• Engajamento de plataforma de suporte on -line: 3 plataformas digitais

Edgewise Therapeutics, Inc. (EWTX) - Modelo de Negócios: Canais

Comunicação médica direta com especialistas

A Edgewise Terapeutics utiliza estratégias de comunicação direcionadas com especialistas médicos focados em distúrbios musculares raros. Os canais de comunicação direta da empresa incluem:

Método de comunicação	Freqüência	Público -alvo
Reuniões do Conselho Consultivo Médico	Trimestral	Especialistas em doenças neuromusculares
Webinars especializados	Bimensal	Pesquisadores raros de transtorno muscular
Extensão clínica direta	Em andamento	Clínicos de Transtorno Genético

Publicações científicas e pesquisa revisada por pares

A Edgewise Terapeutics mantém uma comunicação científica robusta através da disseminação da pesquisa:

• Documentos de pesquisa publicados em 2023: 4
• Apresentações em conferências científicas: 6
• Revistas de revistas revisadas por pares: 3

Relações com investidores e comunicações financeiras

Os canais de comunicação financeira incluem:

Canal de comunicação	Frequência de relatório	Plataforma
Chamadas de ganhos trimestrais	4 vezes anualmente	Chamada de webcast/conferência
Reunião Anual dos Acionistas	Anualmente	Híbrido pessoalmente/virtual
Registros da SEC	Trimestralmente/anualmente	Sistema Edgar

Plataformas digitais para disseminação de informações médicas

As estratégias de comunicação digital incluem:

• Seção de recursos médicos do site da empresa
• Rede Profissional do LinkedIn
• Comunicações de email direcionadas
• Plataformas de informação de ensaios clínicos

Métricas totais de engajamento digital para 2023:

Plataforma	Visitantes únicos	Taxa de engajamento
Site da empresa	47,500	22.3%
LinkedIn	12,300	15.7%

Edgewise Therapeutics, Inc. (EWTX) - Modelo de negócios: segmentos de clientes

Pacientes com distúrbios musculares raros

A terapêutica inteira tem como alvo pacientes com distúrbios musculares raros específicos, particularmente aqueles com Distrofia muscular de Duchenne (DMD).

Transtorno	População estimada de pacientes	Incidência anual
Distrofia muscular de Duchenne	15.000-20.000 pacientes nos Estados Unidos	1 em 3.500-5.000 nascimentos masculinos
Distrofia muscular de Becker	2.500-3.000 pacientes nos Estados Unidos	1 em 18.000 a 30.000 nascimentos masculinos

Neurologistas e especialistas em doenças musculares

Profissionais médicos -alvo especializados em condições neuromusculares.

• Aproximadamente 1.200 especialistas neuromusculares nos Estados Unidos
• Orçamento médio de pesquisa de doenças neuromusculares anuais: US $ 250.000 a US $ 500.000 por especialista

Centros de pesquisa de doenças genéticas

Tipo de centro de pesquisa	Número nos Estados Unidos	Financiamento anual de pesquisa
Centros de pesquisa de distrofia muscular	25-30 centros especializados	US $ 10-50 milhões por centro
Instituições de pesquisa neuromuscular	40-45 instalações dedicadas	US $ 5-25 milhões por instituição

Provedores de saúde com foco em condições neuromusculares

Instalações médicas especializadas e centros de tratamento.

• Total de clínicas neuromusculares nos Estados Unidos: 150-200
• Volume médio do paciente por clínica: 500-1.000 pacientes raros de transtorno muscular anualmente
• Orçamento de tratamento anual total estimado: US $ 75-100 milhões

Edgewise Therapeutics, Inc. (EWTX) - Modelo de negócios: estrutura de custos

Despesas de pesquisa e desenvolvimento

Para o ano fiscal de 2023, a Edgewise Therapeutics registrou despesas totais de P&D de US $ 46,4 milhões, representando uma parcela significativa de seus custos operacionais.

Categoria de despesa	Valor (2023)
Despesas totais de P&D	US $ 46,4 milhões
Custos de P&D relacionados ao pessoal	US $ 22,1 milhões
Custos de pesquisa externos	US $ 12,3 milhões
Equipamento e materiais de laboratório	US $ 8,6 milhões

Custos de gerenciamento de ensaios clínicos

As despesas de ensaios clínicos para 2023 totalizaram aproximadamente US $ 31,2 milhões, que incluem:

• Recrutamento e triagem de pacientes: US $ 8,7 milhões
• Gerenciamento do local clínico: US $ 12,5 milhões
• Gerenciamento e análise de dados: US $ 6,4 milhões
• Documentação regulatória: US $ 3,6 milhões

Proteção à propriedade intelectual

Os custos anuais de proteção de propriedade intelectual para 2023 foram de US $ 2,1 milhões, cobrindo taxas legais de arquivamento, manutenção e legais de patentes.

Recrutamento e retenção de talentos científicos

Despesa relacionada a talentos	Valor (2023)
Custos totais de aquisição de talentos	US $ 5,6 milhões
Salários para pessoal de pesquisa	US $ 24,3 milhões
Benefícios dos funcionários	US $ 6,8 milhões

Conformidade regulatória e teste clínico

As despesas de conformidade regulatória para 2023 foram de US $ 4,5 milhões, que incluem:

• Preparação de envio da FDA: US $ 1,7 milhão
• Documentação de conformidade: US $ 1,2 milhão
• Consultores regulatórios externos: US $ 1,6 milhão

Edgewise Therapeutics, Inc. (EWTX) - Modelo de negócios: fluxos de receita

Potenciais acordos futuros de licenciamento de medicamentos

A partir do quarto trimestre 2023, a Edgewise Terapeutics ainda não relatou nenhum acordos ativos de licenciamento de medicamentos. O foco principal da empresa permanece no desenvolvimento de tratamentos de distrofia muscular.

Bolsas de pesquisa e financiamento do governo

Fonte de financiamento	Quantia	Ano
Institutos Nacionais de Saúde (NIH)	US $ 2,5 milhões	2022
Associação de Distrofia Muscular	$750,000	2023

Vendas potenciais de produtos terapêuticos

O candidato principal da Edgewise Therapeutics EDG-5506 está atualmente em ensaios clínicos, sem vendas comerciais relatadas a partir de 2024.

Parcerias de pesquisa colaborativa

• Colaboração com o Ultragenyx Pharmaceutical
• Parceria de pesquisa com centros médicos acadêmicos

Dados financeiros do relatório anual de 2023 mostram:

Métrica financeira	Quantia
Receita total	US $ 12,4 milhões
Despesas de pesquisa e desenvolvimento	US $ 58,3 milhões

Edgewise Therapeutics, Inc. (EWTX) - Canvas Business Model: Value Propositions

You're looking at the core value Edgewise Therapeutics, Inc. is promising to deliver to patients and the healthcare system as of late 2025. This is all about their pipeline and the significant gaps they aim to fill.

Orally administered, mutation-agnostic therapy (Sevasemten) for muscular dystrophies.

Sevasemten is positioned as an orally administered first-in-class fast skeletal myosin inhibitor. This oral delivery is a major convenience factor for patients dealing with progressive muscle diseases. The company is actively building commercial infrastructure to support a potential U.S. launch of sevasemten in Becker muscular dystrophy.

The clinical progress supporting this value proposition includes:

• Data from the MESA open-label extension trial showed sustained disease stabilization for Becker patients.
• Participants who switched from placebo to sevasemten in the CANYON trial showed a trend toward improvement, with an increase in North Star Ambulatory Assessment (NSAA) scores of 0.2 point since starting the drug over 18 months.
• ARCH participants on sevasemten maintained stable NSAA scores after three years of treatment.
• For Duchenne, data from the LYNX study supported a dose of 10 mg for evaluation in Phase 3, based on consistent observations across functional measures like Stride Velocity 95th Centile (SV95C) and 4 stair-climb.

First-in-class mechanism designed to protect against contraction-induced muscle damage.

The fundamental value here is a mechanism of action that targets the root cause of functional decline in these disorders-damage from muscle contraction. Edgewise Therapeutics, Inc. reports a net loss of $40.7 million for Q3 2025, with Research & Development expenses at $37.5 million, reflecting the investment in advancing this novel science.

Potential to treat Becker MD, a rare disease with no currently approved therapies.

This represents a massive unmet need. There are currently no approved therapies for individuals with Becker muscular dystrophy. The market opportunity reflects this gap; the Becker Muscular Dystrophy Treatment Market in the top 7 markets (US, EU4, UK, and Japan) was valued at USD 880.6 Million in 2024. Edgewise Therapeutics, Inc. is planning for success in the pivotal GRAND CANYON trial, with topline data expected in Q4 2026. The company plans to discuss Phase 3 design with the FDA in Q4 2025 for initiation in 2026.

Novel cardiac sarcomere modulators for Hypertrophic Cardiomyopathy (HCM).

Edgewise Therapeutics, Inc. is developing EDG-7500, a novel oral, selective, cardiac sarcomere modulator for HCM. This targets a shift in treatment from symptomatic relief to sarcomere-directed disease modification, similar to the trend seen with other cardiac myosin inhibitors.

The market context for this program is significant:

Metric	Value/Data Point	Source/Context
HCM Therapeutics Market Value (2025)	USD 572.81 million	Mordor Intelligence estimate for 2025
EDG-7500 Trial Phase	Phase 2 (CIRRUS-HCM)	Advancing in development
Upcoming Catalyst	Program update in Q4 2025	CIRRUS-HCM trial progress
Obstructive HCM Revenue Share (2024)	60.32%	Dominant phenotype in the market
Cardiac Myosin Inhibitor CAGR (to 2030)	4.23%	Fastest-growing drug class segment

The company is well-capitalized to pursue these value drivers, reporting $563.3 million in cash, cash equivalents, and marketable securities as of the end of Q3 2025.

Edgewise Therapeutics, Inc. (EWTX) - Canvas Business Model: Customer Relationships

You're looking at how Edgewise Therapeutics, Inc. maintains its vital connections with the patient and medical communities as it moves toward potential commercialization. For a rare disease company like Edgewise Therapeutics, Inc., these relationships aren't just nice-to-haves; they are the engine driving trial success and future market adoption. Honestly, the commitment shown by participants is staggering.

High-touch engagement with patient advocacy and support groups is central, especially given the focus on Becker muscular dystrophy, where there are currently no approved therapies. The enthusiasm from the community translates directly into trial participation.

• High enrollment in the MESA open-label extension trial shows deep trust.
• The company actively sponsors and participates in numerous patient-focused events.

The commitment from the Becker community is reflected in the MESA trial, which provides continued access to sevasemten. As of September 2025, 99% of eligible participants were enrolled in MESA. This level of engagement is critical for gathering the long-term safety and efficacy data needed.

Direct relationships with Key Opinion Leaders (KOLs) and clinical investigators are managed through the execution of complex, multi-site global trials. The GRAND CANYON pivotal cohort, for instance, was active in 12 countries and enrolled 175 adults before completing enrollment in February 2025. For a company in a rare disease space, the investment in these relationships often requires a deeper commitment than in broader indications. Industry benchmarks suggest that for rare or high-complexity settings, technology and enablement investments supporting KOL engagement can justify 30-35% of the expected retained value.

The strategy for providing continued access to drug via open-label extension trials (e.g., MESA) directly supports investigator and patient retention. This is where you see the long-term commitment in action. The MESA trial builds on earlier studies like CANYON, which involved 40 adults and 29 adolescents in its Phase 2 placebo-controlled cohort.

Trial Program	Patient Group Focus	Key Status/Metric (as of late 2025)
MESA (OLE)	Becker Muscular Dystrophy Participants	Enrollment at 99% of eligible participants as of September 2025
GRAND CANYON	Becker Muscular Dystrophy Adults	Enrollment completed February 2025 with 175 adults across 12 countries
CIRRUS-HCM	Hypertrophic Cardiomyopathy (HCM)	On track for program update in Q4 2025

Finally, scientific and medical education outreach to the physician community ensures that the data Edgewise Therapeutics, Inc. generates is understood by the right prescribers. This outreach is systematic and targeted to major scientific gatherings. You can see this in their Q3 2025 activity:

• Annual International Congress of the World Muscle Society.
• Becker Education and Engagement Day.
• European Society of Cardiology Congress.
• Heart Failure Society of America Annual Scientific Meeting.
• International Workshop on Cardiomyopathies.

The company is building commercial infrastructure to support a potential launch, which requires this foundational medical education. Edgewise Therapeutics, Inc. closed Q3 2025 with approximately $563.3 million in cash, which supports this extensive R&D and outreach activity, which saw R&D expenses of $37.5 million in that quarter.

Edgewise Therapeutics, Inc. (EWTX) - Canvas Business Model: Channels

The channels Edgewise Therapeutics, Inc. uses to reach its customers-patients, investigators, and the medical community-are centered around clinical development and future commercial readiness as of late 2025.

• - Global network of clinical trial sites for patient enrollment.
• - Direct sales force and specialty distribution network (future commercial).
• - Scientific and medical conferences for data dissemination.
• - ClinicalTrials.gov for trial transparency and recruitment.

For patient enrollment in its ongoing trials, Edgewise Therapeutics, Inc. utilizes a network of sites across the globe. The GRAND CANYON pivotal placebo-controlled cohort for sevasemten in Becker muscular dystrophy completed enrollment in February 2025, involving 175 adults across 12 countries. Furthermore, the Phase 2 CIRRUS-HCM trial for EDG-7500 in Hypertrophic Cardiomyopathy is active at over 20 clinical sites in the U.S. The MESA open-label extension trial for sevasemten continues to see high participation, with 99% of eligible participants enrolled as of September 2025.

While currently focused on clinical development, Edgewise Therapeutics, Inc. is actively preparing for the future commercialization of sevasemten. The company is building the commercial infrastructure to support a potential launch in Becker muscular dystrophy. This strategic channel development is supported by recent governance changes, including the appointment of Christopher Martin to the Board of Directors, who brings expertise in guiding companies through product launches.

Dissemination of scientific and medical data is a key channel for engaging with the medical community and establishing credibility. Edgewise Therapeutics, Inc. has been active in presenting data throughout 2025 and into December 2025 at major industry events.

Conference/Update Event	Date in 2025	Program Focus/Context
J.P. Morgan Healthcare Conference Presentation	January 13, 2025	Corporate Overview/Pipeline Priorities
2025 MDA Clinical and Scientific Conference	Date not specified	Sevasemten Data for Becker Muscular Dystrophy
Leerink Partners Global Healthcare Conference	March 11, 2025	Investor Update
EDG-7500: Phase 2 CIRRUS-HCM Update	April 2, 2025	Data from CIRRUS-HCM Trial
Sevasemten Program Update	June 26, 2025	Program Update
Program Update for EDG-7500 (CIRRUS-HCM)	Q4 2025 (Scheduled)	Update on Hypertrophic Cardiomyopathy Trial
Guggenheim Healthcare Innovation Conference	November 11, 2025	Fireside Chat
Piper Sandler 37th Annual Healthcare Conference	December 2, 2025	Fireside Chat
Evercore Healthcare Conference	December 3, 2025	Fireside Chat

Transparency regarding clinical trials is maintained through public registries. For instance, the Phase 2 CIRRUS-HCM trial for EDG-7500 is registered on ClinicalTrials.gov under identifier NCT06347159. The GRAND CANYON trial is listed as NCT05291091.

Edgewise Therapeutics, Inc. (EWTX) - Canvas Business Model: Customer Segments

You're a seasoned analyst looking at Edgewise Therapeutics, Inc. (EWTX) and trying to map out exactly who they are selling to-or, more accurately, who their drugs are intended to treat and who influences that decision. For a pre-revenue biotech like Edgewise Therapeutics, Inc., the customer segment is the patient population suffering from rare, debilitating muscle and heart conditions, plus the specialists who manage their care.

The focus is clearly on rare diseases where there is a significant unmet medical need, which often translates to premium pricing potential once a therapy is approved. Edgewise Therapeutics, Inc. is actively building the commercial infrastructure to support a potential U.S. launch of sevasemten in Becker muscular dystrophy. This preparation signals a clear focus on the patient groups currently in their late-stage trials.

Here is a breakdown of the primary customer segments Edgewise Therapeutics, Inc. is targeting with its pipeline assets as of late 2025:

• Patients with Becker Muscular Dystrophy (BMD).
• Patients with Duchenne Muscular Dystrophy (DMD).
• Patients with Hypertrophic Cardiomyopathy (HCM) and Heart Failure.
• Neuromuscular specialists and cardiologists treating rare muscle disease.

Let's look at the hard numbers defining the size of these patient pools. These figures help ground our valuation models, showing the potential market size for their lead candidates, sevasemten and EDG-7500.

Indication	Target Population Metric	Estimated Number/Value	Lead Candidate
Becker Muscular Dystrophy (BMD)	Estimated U.S. Population	20,000-30,000 people	Sevasemten
Hypertrophic Cardiomyopathy (HCM)	Estimated U.S. Population	Roughly 600,000 people	EDG-7500
Hypertrophic Cardiomyopathy (HCM)	Global Market Projection (by 2031)	$1.35 billion	EDG-7500
Heart Failure (HF)	Development Stage (Targeting HFpEF)	Phase 1 initiated in Q3 2025	EDG-15400

For the BMD segment, you should note that there are currently no approved therapies targeting the underlying cause of muscle degeneration. That is a massive, unaddressed need. Sevasemten is in the pivotal GRAND CANYON trial for these patients, with topline data expected by Q4 2026. The MESA open-label extension trial for sevasemten in BMD continues to enroll, with 99% of eligible participants enrolled as of September 2025.

The DMD segment is also critical. Edgewise Therapeutics, Inc. is advancing sevasemten here, and they announced encouraging top-line data from the Phase 2 LYNX and FOX trials in June 2025, identifying a 10 mg dose to evaluate in Phase 3. This trial data was a key near-term catalyst for the segment, reported in the first half of 2025.

For the cardiac segment, EDG-7500 is being tested in both obstructive (oHCM) and non-obstructive HCM. The company is on track to share a program update for the Phase 2 CIRRUS-HCM trial in the fourth quarter of 2025, with more comprehensive data expected in the first half of 2026. Furthermore, the company is looking beyond HCM to Heart Failure, initiating a Phase 1 trial for EDG-15400 in Q3 2025.

The final segment isn't patients, but the gatekeepers: the physicians. These are the neuromuscular specialists and cardiologists who treat these rare populations. Edgewise Therapeutics, Inc. is actively engaging with the scientific and patient communities through various outreach programs. Their success hinges on convincing these experts of the clinical benefit of sevasemten and EDG-7500. To support this, the company reported a net loss of $40.7 million in Q3 2025, with R&D expenses rising to $37.5 million, driven in part by clinical development activities across these programs. You need to track their spending here; it's the cost of reaching these key prescribers.

Finance: draft 13-week cash view by Friday.

Edgewise Therapeutics, Inc. (EWTX) - Canvas Business Model: Cost Structure

You're looking at the core spending that fuels Edgewise Therapeutics, Inc.'s pipeline progression right now. For a clinical-stage biopharma like Edgewise Therapeutics, Inc., the cost structure is heavily weighted toward getting their drug candidates through trials.

The dominant cost is definitely Research and Development (R&D). For the third quarter of 2025, R&D expenses hit $37.5 million. This figure reflects the scaling of their late-stage and early-stage programs, which is exactly where the money needs to go to generate future value.

We can break down where that R&D spend is going, which speaks directly to your second point about clinical trial operations and patient monitoring. The increase in R&D from the prior quarter was largely due to specific trial costs:

• - $1.4 million increase tied to initiating the Phase 1 trial for EDG-15400 in heart failure.
• - $0.9 million increase for continued patient activity in the EDG-7500 CIRRUS-HCM trial and other pharmacokinetic studies.
• - $0.5 million increase for sevasemten clinical development, covering continued patient activity in the GRAND CANYON trial and the roll over to the MESA open-label extension.

General and Administrative (G&A) expenses, which cover the overhead, executive function, and non-R&D related personnel, were $9.4 million for the third quarter of 2025. That was a slight tick up from $9.1 million in the quarter before, mostly due to additional personnel-related costs.

Manufacturing costs are also a necessary component to support the clinical supply chain and prepare for what comes next. In Q3 2025, manufacturing expenses specifically increased by $0.2 million to support clinical development across their cardiac programs. Plus, as they plan for the pivotal GRAND CANYON cohort readout in Q4 2026, Edgewise Therapeutics is actively building the commercial infrastructure to support a potential launch of sevasemten in Becker muscular dystrophy. That pre-commercial build is a forward-looking cost that will likely grow.

Here's a quick look at the key operating expenses for Q3 2025:

Cost Category	Q3 2025 Amount (USD)
Research and Development (R&D) Expenses	$37.5 million
General and Administrative (G&A) Expenses	$9.4 million
Net Loss (Total Operating Cost Indicator)	$40.7 million

You can see the R&D spend dwarfs G&A, which is typical for a company deep in late-stage trials. The total burn, reflected in the net loss, was $40.7 million for the quarter. That cash position, which stood at approximately $563.3 million as of September 30, 2025, is what funds these costs.

Edgewise Therapeutics, Inc. (EWTX) - Canvas Business Model: Revenue Streams

You're looking at the revenue side of Edgewise Therapeutics, Inc. (EWTX) right now, and the picture is entirely focused on future potential, as is common for clinical-stage biopharma. The current reality is that Edgewise Therapeutics, Inc. is pre-revenue.

For the third quarter of 2025, the company reported a net loss of $40.7 million, or $0.39 per share. That loss reflects the significant investment required to advance their pipeline candidates through late-stage trials. Honestly, the revenue streams are all about what happens after regulatory approval, so you have to look at the cash position to see how long they can fund this development.

Here's a quick look at the key financial metrics underpinning this pre-revenue phase as of the third quarter of 2025:

Financial Metric	Amount (Q3 2025)
Reported Revenue	$0
Net Loss	$40.7 million
Research & Development Expenses	$37.5 million
General & Administrative Expenses	$9.4 million
Cash and Marketable Securities	$563.3 million

Future revenue hinges on the successful commercial sales of their lead candidate, Sevasemten. This orally administered fast skeletal myosin inhibitor is designed to protect muscle against contraction-induced damage in muscular dystrophies. Edgewise Therapeutics, Inc. is actively building commercial infrastructure to support a potential launch for Becker muscular dystrophy (BMD) patients. The pivotal GRAND CANYON cohort data for BMD is expected in the fourth quarter of 2026.

For Duchenne muscular dystrophy (DMD), the path is slightly different. Edgewise Therapeutics, Inc. plans to meet with the FDA in the fourth quarter of 2025 to discuss the Phase 3 design for its DMD studies, with initiation of that pivotal study planned for 2026.

Potential revenue also exists from EDG-7500, their novel oral, selective, cardiac sarcomere modulator being developed for hypertrophic cardiomyopathy (HCM). The company is on track to provide a program update for this candidate in the fourth quarter of 2025, with more comprehensive data expected in the first half of 2026. If that data supports it, Edgewise Therapeutics, Inc. intends to initiate a Phase 3 study in the first half of 2026.

Finally, like many biotechs, Edgewise Therapeutics, Inc. has potential revenue streams from non-product activities. These would be:

• - Potential licensing payments tied to achieving specific development or regulatory milestones for Sevasemten or EDG-7500 in partnered territories or indications.
• - Potential collaboration payments from future agreements on pipeline assets like EDG-15400, which is being developed for heart failure.

The company's strong cash position of $563.3 million as of the end of the third quarter of 2025 provides a substantial runway to fund these clinical advancements before any product revenue is realized. Finance: draft 13-week cash view by Friday.