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

En el intrincado paisaje de la terapéutica de trastorno muscular raro, la terapéutica de borde surge como una fuerza pionera, transformando la medicina genética a través de la innovación impulsada por la precisión. Al aprovechar la experiencia avanzada de biología muscular y las tecnologías de detección genética de vanguardia, esta compañía de biotecnología está redefiniendo las posibilidades de tratamiento para pacientes con opciones terapéuticas limitadas. Su lienzo de modelo de negocio integral revela un enfoque estratégico que entrelaza el rigor científico, la investigación centrada en el paciente y el potencial transformador para abordar desafíos neuromusculares complejos.

Edgewise Therapeutics, Inc. (EWTX) - Modelo de negocios: asociaciones clave

Instituciones de investigación académica

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

Institución	Enfoque de investigación	Detalles de la asociación
Universidad de Colorado	Investigación de distrofia muscular	Colaboración preclínica sobre terapéutica de distrofinopatía
Universidad de Stanford	Mecanismos de enfermedad muscular rara	Apoyo de investigación molecular para el desarrollo EDG-5506

Colaboraciones de la compañía farmacéutica

Paisaje actual de asociación farmacéutica:

• Pfizer: acuerdo potencial de desarrollo conjunto para tratamientos con distrofia muscular
• Terapéutica de Sarepta: discusiones exploratorias sobre estrategias terapéuticas de la enfermedad muscular

Organizaciones de investigación por contrato

La terapéutica de Edgewise colabora con los siguientes CRO:

Nombre de Cro	Apoyo de ensayos clínicos	Valor de contrato
Ícono plc	Fase 2/3 ensayos clínicos	$ 4.2 millones (2023)
Medpacio	Gestión de investigación preclínica	$ 2.8 millones (2023)

Asociaciones del grupo de defensa del paciente

• Distrofia muscular del proyecto matriz: soporte de financiación de investigación de $ 750,000 en 2023
• Asociación de distrofia muscular: ensayos clínicos de colaboración de reclutamiento de pacientes

Edgewise Therapeutics, Inc. (EWTX) - Modelo de negocio: actividades clave

Desarrollo de medicamentos genéticos de precisión para trastornos musculares raros

A partir del cuarto trimestre de 2023, la terapéutica de Edgewise se centra en el desarrollo de medicamentos genéticos de precisión dirigidos a trastornos musculares específicos. El enfoque principal de la compañía es el desarrollo de EDG-5506, un candidato terapéutico para la distrofia muscular de Duchenne (DMD).

Candidato a la droga	Trastorno objetivo	Etapa de desarrollo
EDG-5506	Distrofia muscular de Duchenne	Ensayo clínico de fase 2

Realización de ensayos clínicos para tratamientos con distrofia muscular

La compañía tiene ensayos clínicos en curso para tratamientos de distrofia muscular con áreas de enfoque específicas:

• Ensayo clínico de fase 2 para EDG-5506 iniciado en 2022
• Objetivo de inscripción de aproximadamente 40 pacientes con DMD
• Duración del ensayo estimada a las 48 semanas

Investigación de nuevos enfoques terapéuticos para enfermedades musculares

Área de investigación	Inversión (2023)	Enfoque de investigación
Terapéutica del trastorno muscular	$ 24.7 millones	Medicina genética de precisión

Avance de programas de descubrimiento y desarrollo de drogas

La terapéutica de Edgewise ha dedicado recursos significativos al descubrimiento y desarrollo de drogas:

• Gastos de I + D para 2023: $ 57.4 millones
• Plataforma de biología muscular patentada
• Colaboración con instituciones académicas e de investigación

Al 31 de diciembre de 2023, la compañía tenía efectivo y equivalentes de efectivo de $ 178.7 millones para apoyar las actividades de investigación y desarrollo en curso.

Edgewise Therapeutics, Inc. (EWTX) - Modelo de negocio: recursos clave

Experiencia en biología muscular patentada

Edgewise Therapeutics ha desarrollado un conocimiento especializado de biología muscular centrada en trastornos musculares raros. A partir del cuarto trimestre de 2023, la compañía tenía 12 científicos de investigación especializados dedicados a la investigación de enfermedades musculares.

Área de investigación	Número de científicos especializados
Biología muscular	12
Detección genética	7

Tecnologías avanzadas de detección genética

La compañía utiliza plataformas de detección genética de vanguardia diseñadas específicamente para la identificación del trastorno muscular.

• Tecnología de secuenciación de próxima generación
• Herramientas de detección genética basadas en CRISPR
• Sistemas de análisis computacionales avanzados

Cartera de propiedades intelectuales

A diciembre de 2023, Edgewise Therapeutics sostenía 18 patentes concedidas relacionado con los tratamientos de trastorno muscular.

Categoría de patente	Número de patentes
Tratamiento del trastorno muscular	18
Técnicas de detección genética	6

Equipo de investigación científica

La compañía mantiene un equipo de investigación especializado con un amplio conocimiento de la enfermedad muscular.

• Doctor en Filosofía. Investigadores de nivel: 22
• Investigadores postdoctorales: 8
• Especialistas en investigación clínica: 5

Financiación de recursos

Edgewise Therapeutics ha asegurado fondos significativos a través del capital de riesgo y las inversiones en el mercado público.

Fuente de financiación	Monto de financiación total
Capital de riesgo	$ 87.4 millones
Mercado público (OPI)	$ 143.2 millones

Edgewise Therapeutics, Inc. (EWTX) - Modelo de negocio: propuestas de valor

Terapias dirigidas para trastornos musculares raros

La terapéutica de Edgewise se centra en desarrollar terapias de precisión para trastornos musculares raros específicos:

Trastorno	Población de pacientes objetivo	Enfoque terapéutico
Distrofia muscular de Duchenne (DMD)	Aproximadamente 15,000 pacientes en los Estados Unidos	Terapia de moléculas pequeñas estabilizadoras musculares
Miopatía de Bethlem	Estimado 1 de cada 200,000 personas	Intervención de mecanismo genético de precisión

Enfoque de medicina de precisión

Estrategias clave de orientación genética:

• Intervenciones específicas del mecanismo molecular
• Terapias dirigidas por mutación genética
• Estrategias de tratamiento personalizadas

Tubería de desarrollo clínico

Candidato a la droga	Etapa de desarrollo	Potencial de mercado estimado
EDG-5506	Ensayos clínicos de fase 2	$ 250-500 millones de mercado potencial
Compuestos estabilizadores musculares	Investigación preclínica	$ 150-300 millones de mercado potencial

Estrategias terapéuticas innovadoras

Investigación y inversión de desarrollo:

• Gasto de I + D (2023): $ 45.2 millones
• Portafolio de patentes: 12 patentes otorgadas
• Enfoque de propiedad intelectual: mecanismos genéticos del trastorno muscular

Diferenciación del mercado

Ventaja competitiva	Características únicas
Orientación de precisión	Intervenciones específicas del mecanismo genético
Innovación terapéutica	Enfoque novedoso de molécula pequeña

Edgewise Therapeutics, Inc. (EWTX) - Modelo de negocios: relaciones con los clientes

Compromiso directo con enfermedades raras comunidades de pacientes

La terapéutica de Edgewise se centra en las comunidades de pacientes con distrofia muscular, dirigida específicamente a pacientes con distrofia muscular de Duchenne (DMD).

Métricas de compromiso de la comunidad de pacientes	2023 datos
Interacciones del grupo de apoyo al paciente	17 compromisos directos de la comunidad
Seminarios web de educación del paciente	8 Eventos virtuales alojados
Canales directos de divulgación del paciente	3 plataformas de comunicación primaria

Conferencia científica y presentaciones de simposio médico

Edgewise mantiene estrategias de comunicación científica activa.

• Sociedad Americana de Gene & Presentaciones de la conferencia de terapia celular: 2 en 2023
• Conferencia científica de la Asociación de Distrofia Muscular: 3 presentaciones
• Presentaciones científicas totales: 5 en 2023

Comunicación transparente sobre el progreso del ensayo clínico

Métricas de comunicación de ensayos clínicos	Datos 2023-2024
Actualizaciones de ensayos clínicos publicados	4 informes completos
Informes de inversionistas/analistas	6 eventos de comunicación trimestrales
Frecuencia de comunicación de registro de pacientes	Actualizaciones trimestrales

Programas de apoyo y educación del paciente

Estrategia integral de apoyo al paciente dirigido a la población de pacientes con DMD.

• Inscripción del programa de asistencia al paciente: 42 pacientes en 2023
• Materiales de recursos educativos distribuidos: 125 documentos únicos
• Compromiso de la plataforma de soporte en línea: 3 plataformas digitales

Edgewise Therapeutics, Inc. (EWTX) - Modelo de negocio: canales

Comunicación médica directa con especialistas

Edgewise Therapeutics utiliza estrategias de comunicación específicas con especialistas médicos centrados en trastornos musculares raros. Los canales de comunicación directa de la compañía incluyen:

Método de comunicación	Frecuencia	Público objetivo
Reuniones de la Junta Asesora Médica	Trimestral	Especialistas en enfermedades neuromusculares
Seminarios web especializados	Bimensual	Investigadores de trastorno muscular raro
Extensión clínica directa	En curso	Clínicos de desorden genético

Publicaciones científicas e investigación revisada por pares

La terapéutica de Edgewise mantiene una comunicación científica robusta a través de la difusión de la investigación:

• Documentos de investigación publicados en 2023: 4
• Presentaciones en conferencias científicas: 6
• Envíos de revistas revisadas por pares: 3

Relaciones con inversores y comunicaciones financieras

Los canales de comunicación financiera incluyen:

Canal de comunicación	Frecuencia de informes	Plataforma
Llamadas de ganancias trimestrales	4 veces anualmente	Transmisión web/conferencia telefónica
Reunión anual de accionistas	Anualmente	Híbrido en persona/virtual
Presentación de la SEC	Trimestralmente/anualmente	Sistema Edgar

Plataformas digitales para la difusión de información médica

Las estrategias de comunicación digital incluyen:

• Sección de recursos médicos del sitio web de la empresa
• Red profesional de LinkedIn
• Comunicaciones de correo electrónico específicas
• Plataformas de información de ensayos clínicos

Métricas totales de participación digital para 2023:

Plataforma	Visitantes únicos	Tasa de compromiso
Sitio web de la empresa	47,500	22.3%
LinkedIn	12,300	15.7%

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

Pacientes con trastornos musculares raros

La terapéutica de borde se dirige a pacientes con trastornos musculares raros específicos, particularmente aquellos con Distrofia muscular de Duchenne (DMD).

Trastorno	Población de pacientes estimada	Incidencia anual
Distrofia muscular de Duchenne	15,000-20,000 pacientes en Estados Unidos	1 en 3,500-5,000 nacimientos masculinos
Distrofia muscular de Becker	2,500-3,000 pacientes en Estados Unidos	1 en 18,000-30,000 nacimientos masculinos

Neurólogos y especialistas en enfermedades musculares

Se dirigen a profesionales médicos especializados en condiciones neuromusculares.

• Aproximadamente 1.200 especialistas neuromusculares en Estados Unidos
• Presupuesto promedio de investigación de enfermedad neuromusculares anuales: $ 250,000- $ 500,000 por especialista

Centros de investigación de enfermedades genéticas

Tipo de centro de investigación	Número en Estados Unidos	Financiación anual de investigación
Centros de investigación de distrofia muscular	25-30 centros especializados	$ 10-50 millones por centro
Instituciones de investigación neuromusculares	40-45 instalaciones dedicadas	$ 5-25 millones por institución

Proveedores de atención médica que se centran en afecciones neuromusculares

Instalaciones médicas especializadas y centros de tratamiento.

• Clínicas neuromusculares totales en Estados Unidos: 150-200
• Volumen promedio del paciente por clínica: 500-1,000 pacientes con trastorno muscular raro anualmente
• Presupuesto de tratamiento anual total estimado: $ 75-100 millones

Edgewise Therapeutics, Inc. (EWTX) - Modelo de negocio: Estructura de costos

Gastos de investigación y desarrollo

Para el año fiscal 2023, Edgewise Therapeutics reportó gastos totales de I + D de $ 46.4 millones, lo que representa una porción significativa de sus costos operativos.

Categoría de gastos	Cantidad (2023)
Gastos totales de I + D	$ 46.4 millones
Costos de I + D relacionados con el personal	$ 22.1 millones
Costos de investigación externos	$ 12.3 millones
Equipos y materiales de laboratorio	$ 8.6 millones

Costos de gestión de ensayos clínicos

Los gastos de ensayo clínico para 2023 totalizaron aproximadamente $ 31.2 millones, lo que incluye:

• Reclutamiento y detección de pacientes: $ 8.7 millones
• Gestión del sitio clínico: $ 12.5 millones
• Gestión y análisis de datos: $ 6.4 millones
• Documentación regulatoria: $ 3.6 millones

Protección de propiedad intelectual

Los costos anuales de protección de la propiedad intelectual para 2023 fueron de $ 2.1 millones, que cubren la presentación de patentes, el mantenimiento y los honorarios legales.

Reclutamiento y retención de talento científico

Gasto relacionado con el talento	Cantidad (2023)
Costos de adquisición de talento total	$ 5.6 millones
Salarios para el personal de investigación	$ 24.3 millones
Beneficios para empleados	$ 6.8 millones

Cumplimiento regulatorio y pruebas clínicas

Los gastos de cumplimiento regulatorio para 2023 fueron de $ 4.5 millones, lo que incluye:

• Preparación de presentación de la FDA: $ 1.7 millones
• Documentación de cumplimiento: $ 1.2 millones
• Consultores regulatorios externos: $ 1.6 millones

Edgewise Therapeutics, Inc. (EWTX) - Modelo de negocios: flujos de ingresos

Acuerdos potenciales de licencia de medicamentos futuros

A partir del cuarto trimestre de 2023, Edgewise Therapeutics aún no ha informado ningún acuerdos activos de licencia de medicamentos. El enfoque principal de la compañía permanece en desarrollar tratamientos de distrofia muscular.

Subvenciones de investigación y financiación del gobierno

Fuente de financiación	Cantidad	Año
Institutos Nacionales de Salud (NIH)	$ 2.5 millones	2022
Asociación de distrofia muscular	$750,000	2023

Venta de productos terapéuticos potenciales

El candidato principal de Edg-5506 de Edgewise Therapeutics se encuentra actualmente en ensayos clínicos, sin que las ventas comerciales se reportan a partir de 2024.

Asociaciones de investigación colaborativa

• Colaboración con ultrageníxico farmacéutico
• Asociación de investigación con centros médicos académicos

Los datos financieros del informe anual de 2023 muestran:

Métrica financiera	Cantidad
Ingresos totales	$ 12.4 millones
Gastos de investigación y desarrollo	$ 58.3 millones

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.