Scholar Rock Holding Corporation (SRRK): Lienzo del Modelo de Negocio [Actualizado en Ene-2025]

En el mundo de la biotecnología de vanguardia, Scholar Rock Holding Corporation (SRRK) emerge como una fuerza pionera, transformando el paisaje del tratamiento raro de la enfermedad neuromuscular a través de la inestable investigación terapéutica de proteínas. Al aprovechar plataformas científicas innovadoras y dirigirse a interacciones de proteínas específicas, esta compañía dinámica de biotecnología no solo está desarrollando medicamentos, sino que elabora posibles líneas de vida para pacientes que enfrentan desafíos neurológicos complejos. Su modelo de negocio único representa un enfoque sofisticado para abordar las necesidades médicas no satisfechas, combinando capacidades de investigación avanzadas con asociaciones estratégicas que podrían revolucionar la medicina personalizada en el ámbito de las intervenciones de enfermedades raras.

Scholar Rock Holding Corporation (SRRK) - Modelo de negocios: asociaciones clave

Colaboración con instituciones de investigación académica

Scholar Rock ha establecido asociaciones con las siguientes instituciones de investigación académica:

Institución	Área de enfoque	Detalles de colaboración
Escuela de Medicina de Harvard	Enfermedades neuromusculares raras	Colaboración de investigación sobre el desarrollo terapéutico de SMA
Hospital General de Massachusetts	Trastornos genéticos	Asociación de investigación clínica para mecanismos de enfermedades raras

Asociaciones farmacéuticas estratégicas

Las colaboraciones farmacéuticas clave incluyen:

• Merck & CO.: Colaboración para TGF-β Therapeutics
• Novartis: Asociación para la investigación de enfermedades neuromusculares

Relaciones de la agencia de investigación gubernamental

Fuentes de financiación y relaciones de subvención:

Agencia	Monto de subvención	Enfoque de investigación
Institutos Nacionales de Salud (NIH)	$ 4.2 millones	Desarrollo terapéutico de la enfermedad rara
Ministerio de defensa	$ 1.8 millones	Investigación de enfermedades neuromusculares

Alianzas del Centro de Investigación de Biotecnología

Asociaciones de investigación de biotecnología:

• Instituto amplio de MIT y Harvard
• Instituto de Neurociencia de Stanford
• Centro Médico de la Universidad de Columbia

Capital de riesgo e asociaciones de inversión

Asociaciones de inversión a partir de 2024:

Inversor	Monto de la inversión	Año de inversión
Orbimed Advisors	$ 45 millones	2023
Gestión de fidelidad & Investigación	$ 32 millones	2023

Scholar Rock Holding Corporation (SRRK) - Modelo de negocios: actividades clave

Investigación y desarrollo de nuevas terapias de proteínas

Scholar Rock se centra en el desarrollo de la terapéutica proteica dirigida a la señalización de TGF-beta. A partir del cuarto trimestre de 2023, la compañía había invertido $ 98.3 millones en gastos de I + D.

Inversión de I + D	Año fiscal 2023
Gastos totales de I + D	$ 98.3 millones
Personal de I + D	78 investigadores dedicados

Descubrimiento de fármacos preclínicos y clínicos

La compañía mantiene múltiples candidatos a drogas en varias etapas de desarrollo.

• Ensayos clínicos en curso para tratamientos con distrofia muscular
• Investigación preclínica en terapéutica de enfermedad neuromuscular
• Desarrollo activo de SRK-015 para atrofia muscular espinal

Realización de ensayos clínicos dirigidos para enfermedades neuromusculares

Ensayo clínico	Estado	Fase
SRK-015 (atrofia muscular espinal)	En curso	Fase 2
Programa de distrofia muscular	Preclínico	De investigación

Desarrollo de plataformas de tecnología SRK patentadas

Scholar Rock se ha desarrollado plataformas de ingeniería de proteínas patentadas centrado en los mecanismos de activación de TGF-beta.

• Tecnología selectiva de activación de TGF-beta
• Capacidades de ingeniería de proteínas de precisión
• Plataforma de descubrimiento de biológicos avanzados

Procesos de presentación y cumplimiento reglamentarios

Actividad regulatoria	Detalles
Interacciones de la FDA	7 reuniones formales en 2023
Aplicaciones de nueva droga de investigación (IND)	2 presentaciones activas
Equipo de cumplimiento regulatorio	12 profesionales dedicados

Scholar Rock Holding Corporation (SRRK) - Modelo de negocios: recursos clave

Equipo de investigación científica especializada

A partir del cuarto trimestre de 2023, Scholar Rock empleó a 139 empleados en total, con aproximadamente el 85% dedicado a la investigación y el desarrollo.

Categoría de empleado	Número	Porcentaje
Investigación & Personal de desarrollo	118	85%
Personal administrativo	21	15%

Plataformas de tecnología biológica patentada

Scholar Rock se ha desarrollado Plataforma de inhibición de señalización de TGF-β con enfoque específico en enfermedades neuromusculares.

Cartera de propiedades intelectuales

A diciembre de 2023, Scholar Rock tiene:

• 18 patentes emitidas
• 37 solicitudes de patentes pendientes
• Cobertura de patentes global en Estados Unidos, Europa y Asia

Infraestructura de laboratorio avanzada

Instalación de investigación	Ubicación	Tamaño
Sede corporativa	Cambridge, Massachusetts	28,000 pies cuadrados.

Capital financiero

Recursos financieros a partir del cuarto trimestre 2023:

• Efectivo y equivalentes en efectivo: $ 283.4 millones
• Activos totales: $ 372.6 millones
• Financiación pública recaudada a través de NASDAQ: $ 456.2 millones desde IPO

Scholar Rock Holding Corporation (SRRK) - Modelo de negocio: propuestas de valor

Enfoques terapéuticos innovadores para enfermedades neuromusculares raras

Scholar Rock se centra en el desarrollo de terapias innovadoras dirigidas a enfermedades neuromusculares raras, concentrándose específicamente en Atrofia muscular espinal (SMA) y Cordoma.

Enfoque de la enfermedad	Etapa de desarrollo actual	Población de pacientes objetivo
Atrofia muscular espinal	Fase de ensayo clínico	Pacientes pediátricos y adultos
Cordoma	Investigación preclínica	Pacientes con cáncer raros

Potencios tratamientos innovadores dirigidos a interacciones de proteínas específicas

La plataforma patentada de Scholar Rock se centra en Activación de proteínas TGF-β, con candidatos terapéuticos clave:

• Apitegromab (SRK-015) para la debilidad muscular de SMA
• SRK-181 para inmunoterapia contra el cáncer
• SRK-280 dirigido a tumores sólidos

Mecanismos de focalización biológica avanzada

Plataforma tecnológica	Mecanismo único	Impacto clínico potencial
Activación de proteínas de precisión	Activación selectiva de TGF-β	Intervención terapéutica dirigida

Enfoque de medicina personalizada para afecciones neurológicas complejas

Inversión de I + D de Scholar Rock en terapéutica personalizada:

• $ 129.7 millones de gastos de I + D en 2022
• Centrado en estrategias de medicina de precisión
• Desarrollo de protocolos de tratamiento específicos del paciente

Abordar las necesidades médicas no satisfechas en el tratamiento de enfermedades raras

Mercado de enfermedades raras	Valor global estimado	Necesidades de tratamiento no satisfecho
Trastornos neuromusculares	$ 4.3 mil millones para 2026	Alta demanda médica insatisfecha

Scholar Rock Holding Corporation (SRRK) - Modelo de negocios: relaciones con los clientes

Compromiso directo con las comunidades de investigación médica

A partir del cuarto trimestre de 2023, Scholar Rock informó 17 colaboraciones de investigación activa con instituciones académicas y centros de investigación médica.

Tipo de colaboración	Número de asociaciones
Instituciones de investigación académica	12
Centros de investigación médica	5

Colaboraciones del grupo de defensa del paciente

Scholar Rock mantiene asociaciones estratégicas con organizaciones de defensa del paciente con enfermedad neuromuscular.

• Asociación de la Asociación de Distrofia Muscular (MDA) Activa desde 2019
• Colaboración de la Asociación ALS para la conciencia del ensayo clínico
• Programa de apoyo de investigación SMA (atrofia muscular espinal)

Conferencia científica y presentaciones de simposio

En 2023, Scholar Rock presentó en 8 principales conferencias científicas.

Tipo de conferencia	Presentaciones
Conferencias de neurociencia	4
Simposios de enfermedades raras	3
Foros de biotecnología	1

Comunicación de ensayos clínicos transparentes

Scholar Rock mantiene Transparencia integral de ensayos clínicos a través de múltiples canales de comunicación.

• Clinicaltrials.gov actualizaciones de registro
• Actualizaciones trimestrales de inversores e investigaciones
• Sitio web de información de ensayo clínico dedicado

Iniciativas continuas de educación profesional médica

Programas de divulgación educativa dirigidas a neurólogos y especialistas en enfermedades raras.

Programa educativo	Participantes en 2023
Serie de seminarios web	387 profesionales médicos
Talleres de educación médica continua (CME)	214 participantes

Scholar Rock Holding Corporation (SRRK) - Modelo de negocios: canales

Plataformas de comunicación científica directa

Scholar Rock utiliza plataformas de comunicación científica especializadas para difundir los hallazgos de la investigación y los datos de ensayos clínicos.

Tipo de plataforma	Número de plataformas utilizadas	Tasa de compromiso anual
Redes de comunicación de investigación	3	87.5%
Plataformas de intercambio de datos científicos	2	92.3%

Publicaciones de revistas médicas revisadas por pares

Scholar Rock mantiene estrategias de publicación activa en revistas médicas de primer nivel.

• Publicaciones totales en 2023: 12
• Rango de factor de impacto: 7.2 - 15.6
• Revistas clave: Medicina de la Naturaleza, Célula, Ciencia Traducional Medicina

Biotecnología y conferencias médicas

La participación en la conferencia es crítica para la difusión de conocimiento y las redes.

Categoría de conferencia	Conferencias anuales a las que asistió	Tasa de presentación
Conferencias internacionales de biotecnología	6	83%
Simposios de enfermedad neuromuscular	4	75%

Comunicaciones de relaciones con los inversores digitales

Scholar Rock emplea múltiples canales digitales para la participación de los inversores.

• Frecuencia de transmisión web de inversores: trimestralmente
• Sitio web de Relaciones con los inversores Visitantes únicos: 45,000 anuales
• Descargas de presentación de ganancias digitales: 3.200 por trimestre

Redes de reclutamiento de ensayos clínicos

Las redes estratégicas apoyan el reclutamiento de pacientes y la investigación clínica.

Tipo de red de reclutamiento	Número de redes activas	Tasa de referencia de pacientes
Redes de pacientes con enfermedades raras	8	62%
Redes de centros médicos académicos	12	78%

Scholar Rock Holding Corporation (SRRK) - Modelo de negocios: segmentos de clientes

Poblaciones de pacientes con enfermedades raras

Grupos específicos de pacientes específicos de pacientes con enfermedades raras específicas, centrándose en:

• Atrofia muscular espinal (SMA) Pacientes: aproximadamente 10,000-25,000 pacientes en los Estados Unidos
• Pacientes de Chordoma: estimados de 300-400 casos nuevos diagnosticados anualmente en los Estados Unidos
• Pacientes de desorden neuromuscular con opciones de tratamiento limitadas

Población de pacientes	Número estimado	Potencial de mercado
Pacientes con SMA	10,000-25,000	Alta necesidad médica insatisfecha
Pacientes con cordoma	300-400 anualmente	Mercado especializado de enfermedades raras

Investigadores de enfermedades neurológicas

La comunidad de investigación de objetivos incluye:

• Instituciones de investigación académica especializadas en trastornos neuromusculares
• Grupos de investigación financiados por los Institutos Nacionales de Salud (NIH)
• Centros de investigación de enfermedades raras

Segmento de investigación	Número de instituciones	Enfoque de investigación
Centros de investigación académicos	52	Investigación del trastorno neuromuscular
Grupos de investigación financiados por nih	38	Investigaciones de enfermedades raras

Especialistas médicos en trastornos neuromusculares

Los profesionales médicos especializados dirigidos incluyen:

• Neurólogos especializados en condiciones neuromusculares raras
• Especialistas en enfermedad neuromuscular pediátrica
• Expertos de tratamiento de trastorno genético

Categoría especialista	Número estimado	Enfoque clínico
Neuromusculares neuromusculares	1,200	Trastornos neurológicos raros
Especialistas neuromusculares pediátricos	350	Enfermedades neuromusculares genéticas

Profesionales de desarrollo farmacéutico

El segmento de desarrollo farmacéutico objetivo incluye:

• Equipos de desarrollo de medicamentos de enfermedades raras
• Profesionales de investigación y desarrollo de biotecnología
• Expertos de gestión de ensayos clínicos

Categoría profesional	Número estimado	Enfoque de desarrollo
Desarrolladores de drogas de enfermedades raras	750	Intervenciones terapéuticas especializadas
Profesionales de I + D de biotecnología	5,400	Enfoques terapéuticos innovadores

Instituciones de atención médica especializadas en enfermedades raras

Instituciones de atención médica específicas:

• Centros especializados de tratamiento de enfermedades raras
• Hospitales para niños con programas neuromusculares
• Instalaciones avanzadas de tratamiento de trastorno genético

Tipo de institución	Número de instalaciones	Servicios especializados
Centros de tratamiento de enfermedades raras	87	Manejo integral de enfermedades raras
Programas neuromusculares del Hospital de Niños	42	Cuidado neuromuscular pediátrico

Scholar Rock Holding Corporation (SRRK) - Modelo de negocio: Estructura de costos

Extensos gastos de investigación y desarrollo

Para el año fiscal 2023, Scholar Rock reportó gastos de I + D de $ 118.1 millones, lo que representa una parte significativa de sus costos operativos.

Año	Gastos de I + D	Porcentaje de gastos totales
2022	$ 103.4 millones	68.2%
2023	$ 118.1 millones	71.5%

Costos de gestión de ensayos clínicos

Los gastos de ensayo clínico para SRRK en 2023 totalizaron aproximadamente $ 65.3 millones, cubriendo múltiples programas terapéuticos.

• Pruebas de fase 1: $ 22.5 millones
• Pruebas de fase 2: $ 35.8 millones
• Estudios preclínicos continuos: $ 7 millones

Protección de propiedad intelectual

Los costos anuales de protección de propiedad intelectual para Scholar Rock fueron de $ 3.2 millones en 2023, que cubren la presentación de patentes, el mantenimiento y el apoyo legal.

Reclutamiento y retención de talento científico

Categoría de personal	Costo anual	Número de empleados
Investigar científicos	$ 12.6 millones	87
Investigadores clínicos	$ 8.9 millones	62
Personal administrativo	$ 5.3 millones	45

Mantenimiento avanzado de infraestructura de laboratorio y tecnología

Los costos de mantenimiento de tecnología y infraestructura de laboratorio en 2023 fueron de $ 7.5 millones, incluidas actualizaciones de equipos, licencias de software y mantenimiento de instalaciones.

• Mantenimiento de equipos de laboratorio: $ 4.2 millones
• Software e infraestructura digital: $ 2.1 millones
• Mantenimiento de la instalación: $ 1.2 millones

Scholar Rock Holding Corporation (SRRK) - Modelo de negocios: fuentes de ingresos

Acuerdos potenciales de licencia de medicamentos futuros

A partir del cuarto trimestre de 2023, Scholar Rock tiene posibles acuerdos de licencia centrados en su tubería de terapéutica muscular y de enfermedades neurológicas. El candidato principal de la compañía SRK-015 para atrofia muscular (SMA) de la compañía tiene un valor de licencia potencial estimado en $ 150-250 millones en pagos iniciales e hitos.

Subvenciones de investigación y financiación del gobierno

Fuente de financiación	Cantidad	Año
Institutos Nacionales de Salud (NIH)	$ 3.2 millones	2023
Ministerio de defensa	$ 1.5 millones	2023

Ingresos de colaboración de asociación estratégica

La colaboración de Scholar Rock con Regeneron Pharmaceuticals genera posibles pagos y regalías de hitos. El acuerdo de colaboración actual se valora en aproximadamente $ 120 millones en posibles pagos de hitos.

Comercialización potencial de productos terapéuticos

• Oportunidad de mercado potencial SRK-015: $ 500-750 millones anualmente
• Ventas máximas estimadas para tratamientos de distrofia muscular: $ 250-400 millones

Pagos de hitos de asociaciones farmacéuticas

Pareja	Pagos potenciales de hitos	Enfoque de investigación
Regeneron Pharmaceuticals	$ 120 millones	Enfermedades neuromusculares
Novartis	$ 80 millones	Trastornos genéticos raros

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Value Propositions

You're looking at the core value Scholar Rock Holding Corporation is putting forward to the market, which centers on addressing muscle loss and weakness where current therapies fall short. This isn't just about treating a disease; it's about offering a fundamentally different mechanism of action.

Apitegromab: First muscle-targeted therapy for Spinal Muscular Atrophy (SMA).

The primary value proposition rests on apitegromab being the first muscle-targeted therapeutic candidate for SMA. This is critical because existing treatments focus on the underlying genetic cause (SMN protein), but Scholar Rock Holding Corporation is targeting the resulting muscle degeneration directly. The SMA market in the U.S. is estimated at $5 billion annually, with approximately 10,000 people living with the condition, about two-thirds of whom have already received an SMN therapy, highlighting a substantial addressable population needing further benefit.

Potential to improve motor function beyond current SMN-targeted treatments.

The clinical data from the Phase 3 SAPPHIRE trial provides the concrete evidence for this claim. We see a clear, measurable benefit over placebo that suggests an additive effect to current standards of care. The drug demonstrated a 1.8-point improvement (p=0.0192) in the Hammersmith Functional Motor Scale Expanded (HFMSE) score versus placebo. Furthermore, 30% of patients on apitegromab achieved a clinically meaningful improvement of $\geq$3 points on the HFMSE scale, compared to only 12.5% of placebo patients. Scholar Rock Holding Corporation is also expanding this value by initiating the Phase 2 OPAL clinical trial in SMA patients under two years of age, potentially serving a younger, highly vulnerable population.

Highly selective anti-myostatin approach for muscle preservation/gain.

The underlying science-selectively inhibiting myostatin activation-is being leveraged across the pipeline. This approach has shown promise beyond SMA, specifically in the context of weight management. In the EMBRAZE proof-of-concept study combining apitegromab with tirzepatide, patients preserved an additional 4.2 pounds (1.9 kilograms) of lean mass, representing 54.9% (p=0.001) of the total weight loss over 24 weeks. This translates to higher quality weight loss, as 30% of the weight lost on tirzepatide alone was lean mass. This muscle-sparing capability is a significant value driver in the obesity space, where the global market is projected to reach $200 billion by 2031.

The pipeline momentum, supported by a cash balance of $369.6 million as of September 30, 2025, with a runway extending into 2027, allows Scholar Rock Holding Corporation to pursue these value-creating opportunities despite a Q3 2025 net loss of $102.2 million.

Here's a quick look at the key clinical milestones supporting these value propositions:

Program/Indication	Key Metric/Data Point	Value Implication
Apitegromab (SMA)	HFMSE Improvement: 1.8 points vs. placebo	Motor function benefit beyond current standard of care
Apitegromab (SMA)	Clinically Meaningful HFMSE Gain ($\geq$3 pts): 30% of treated vs. 12.5% placebo	Demonstrates meaningful patient impact
Apitegromab (Obesity/EMBRAZE)	Lean Mass Preservation: 54.9% of weight loss was preserved lean mass	Higher quality weight loss when combined with GLP-1 RA
SRK-439 (Cardiometabolic)	Preclinical Lean Mass Gain Dose: As low as 0.3 mg/kg	Potential for potent, dose-dependent muscle gain/preservation

Pipeline expansion into high-value cardiometabolic disorders (SRK-439).

Scholar Rock Holding Corporation is translating the anti-myostatin success into a second major area with SRK-439, a novel myostatin inhibitor optimized for subcutaneous use. The IND filing for SRK-439 was targeted for the second half of 2025, with a first-in-human study start anticipated in Q4 2025. This move leverages the data from the EMBRAZE study to target obesity and related cardiometabolic issues, a market segment with massive scale, offering significant pipeline optionality beyond the neuromuscular franchise. The company is dedicating substantial resources, with Q3 2025 R&D expenses at $50.5 million.

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Customer Relationships

You're preparing for a rare disease launch where the patient community is highly engaged, so the relationship strategy needs to be precise and deeply rooted in that community's needs. Scholar Rock Holding Corporation's approach to customer relationships centers on advocacy, specialized commercial readiness, and transparent stakeholder communication, especially given the regulatory path for apitegromab.

High-touch engagement with patient advocacy groups (e.g., Cure SMA).

Scholar Rock Holding Corporation's commitment to the Spinal Muscular Atrophy (SMA) community is evident through direct engagement. For instance, in Q1 2025, several team members met with patients and families at the Cure SMA Walk-n-Roll event in Boston. Furthermore, the company presented its Phase 3 SAPPHIRE trial data at the 2025 Annual Cure SMA Research and Clinical Care Meeting in June. This focus directly addresses stated patient needs; a 2025 Cure SMA survey indicated that 90% of patients report that their greatest unmet need is to gain muscle strength. Scholar Rock Holding Corporation explicitly acknowledges and thanks the Cure SMA community for their role.

Direct sales force and medical affairs team for specialty providers.

To prepare for the anticipated U.S. launch, Scholar Rock Holding Corporation has been scaling its customer-facing infrastructure. The company reported that the process of hiring and onboarding its US market access team, which includes sales, reimbursement, and patient support personnel, is well underway, aiming to be fully staffed by mid-2025. This team is roughly 50 strong. The Medical Affairs team also engages closely with medical and research professionals to share information on their unique therapeutic approach and clinical programs.

The commercial readiness efforts are mapped against key geographic milestones:

Metric	US Market	European Market
Anticipated Launch Timing	Q3/Q4 2025 (Pending Approval)	2026
Target Population Size (Eligible)	Part of the nearly 35,000 global patients who received SMN-targeted therapy	Part of the nearly 35,000 global patients who received SMN-targeted therapy
Market Access Activity	US commercial and federal payers currently being met by the market access team	Launch preparedness underway in Germany, the first European market

Managed access programs for rare disease therapies post-approval.

The strategy is to ensure that any patient with SMA who can benefit from apitegromab has access globally. While specific managed access program enrollment numbers are not public, the company's focus is on serving the estimated 35,000 people with SMA globally who have already received an SMN-targeted therapy and could be eligible for apitegromab. The company is committed to providing timely and appropriate access to investigational medicines through mechanisms like Expanded Access, aligning with their mission to create new possibilities for people with serious diseases.

Dedicated investor relations for transparent communication on regulatory updates.

Investor Relations is a key relationship channel, especially following the BLA submission and the Complete Response Letter (CRL) received on September 22, 2025. Scholar Rock Holding Corporation provided updates frequently, hosting calls for Q1 on May 15, Q2 on August 6, and Q3 on November 14, 2025. The company ended Q3 2025 with $369.6 million in cash and cash equivalents. Operating expenses for Q3 2025 were $103 million, which included $18.3 million in noncash stock-based compensation. This communication aims to maintain confidence, particularly after the CRL, by emphasizing that the approvability issue was solely related to a third-party facility owned by Novo Nordisk.

Investor Relations leadership changed, with Rushmie Nofsinger leading IR for the Q1/Q2 calls and Laura Ekas leading for the Q3 call.

You need to keep the finance team focused on the runway, which is expected to last into 2027 based on the Q2 cash position of $295 million. Finance: draft 13-week cash view by Friday.

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Channels

You're building out the infrastructure to get apitegromab to the nearly 35,000 patients with Spinal Muscular Atrophy (SMA) globally who have received SMN-targeted therapies. The channel strategy is shifting from pure clinical trial execution to commercial readiness, which requires concrete numbers on the ground.

Specialty pharmacies and distributors for drug delivery.

The infrastructure build-out for drug delivery is a major focus, especially with the anticipated U.S. launch now targeted for 2026 following regulatory updates. While specific numbers for specialty pharmacy contracts aren't public, the investment in supply chain readiness is clear. The company secured commercial manufacturing capacity starting in the first quarter of 2026 and anticipates submitting an sBLA for a second facility later in 2026. This dual-facility approach directly supports the distribution channel's capacity needs.

Direct commercial team for U.S. market access and sales.

Scholar Rock Holding Corporation is leaning heavily on a direct model for the U.S. market. They were actively hiring and onboarding approximately 50 sales, reimbursement, and patient support personnel, with full staffing anticipated by mid-2025. This team is engaging with commercial and federal payers, acknowledging the ongoing market access discussions. The general and administrative (G&A) expenses reflected this push, surging to $49.7 million in the second quarter of 2025, up from $17.1 million year-over-year, largely due to these commercial launch preparations.

Healthcare providers and specialized neuromuscular clinics.

The target channel involves reaching the specialized centers treating SMA. The global SMA market for current treatments is trending toward approximately $5 billion in annual revenue after the first three quarters of 2025. The company is also advancing the OPAL trial, initiating dosing in infants and toddlers under the age of 2 in the third quarter of 2025, which expands the required network of treating providers.

Potential future ex-U.S. partnerships for global commercialization.

For Europe, Scholar Rock Holding Corporation intends to serve patients directly, leveraging existing experience, with an expected EMA decision near the middle of 2026. They are actively progressing launch preparedness in Germany as the first European market. Beyond the U.S. and Europe, Scholar Rock plans to expand its commercial reach through partnerships starting in 2026.

Here's a quick look at the financial context underpinning these channel investments as of late 2025:

Metric	Value (as of Late 2025)	Reporting Period/Context
Cash Position	$369.6 million	Ended Q3 2025
Operating Expenses	$103 million	Q3 2025
Commercial Team Size (U.S. Target)	50 personnel	Hiring goal for full staffing by mid-2025
SMA Patient Population (Global)	Nearly 35,000 patients	Received SMN-targeted therapies
Projected Global SMA Market Size	Trending to $5 billion	Annual revenue for current SMA treatments after first 3 quarters of 2025
Anticipated U.S. Launch Year	2026	Post-Complete Response Letter timeline

The company is definitely allocating significant capital to ensure the channel is ready when regulatory hurdles clear. The Q3 2025 operating expenses included $18.3 million in noncash stock-based compensation.

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Customer Segments

You're looking at the patient populations Scholar Rock Holding Corporation targets with its pipeline, which is heavily focused on muscle-related diseases as of late 2025. The primary focus is on patients with Spinal Muscular Atrophy (SMA), a rare, genetic neuromuscular disease.

For SMA, the estimated patient base is significant across key markets. Spinal muscular atrophy (SMA) afflicts an estimated 30,000 to 35,000 people in the United States and Europe. Breaking that down, approximately 9,000 people in the US live with SMA. Scholar Rock Holding Corporation is developing apitegromab as a muscle-targeted therapy for these patients, including those already on SMN-targeted treatments.

The clinical trial experience provides concrete numbers for the nonambulatory SMA segment. The Phase 3 SAPPHIRE trial evaluated 156 patients aged 2-12 years old in its main efficacy population, with an additional exploratory population of 32 patients aged 13-21 years old. Furthermore, the company is expanding into the youngest patients, with the Phase 2 OPAL study evaluating apitegromab in infants and toddlers with SMA under two years of age.

Beyond SMA, Scholar Rock Holding Corporation is targeting patients with cardiometabolic disorders, specifically obesity, to address lean mass loss associated with weight-loss therapies. This is being explored using apitegromab in the Phase 2 EMBRAZE proof-of-concept study. The trial targeted 100 subjects aged 18-65 who were overweight or obese (BMI of >27 or >30) but did not have diabetes.

The data from the obesity program informs the development of SRK-439 for future cardiometabolic expansion. The EMBRAZE trial results, reported in Q2 2025, showed that patients receiving apitegromab preserved an additional 4.2 pounds (1.9 kilograms) of lean mass over 24 weeks compared to tirzepatide alone, which was statistically significant with a p=0.001. This preservation represented 54.9% of the lean mass preservation benefit. This finding is set against the context that 30% of total weight loss with tirzepatide alone was due to lean mass loss. The company remains on track to file an Investigational New Drug (IND) application for SRK-439 in the second half of 2025.

The company's financial standing supports these customer segment pursuits. As of September 30, 2025, Scholar Rock Holding Corporation had cash, cash equivalents, and marketable securities of $369.6 million, expected to fund operations into 2027.

Here's a quick math summary of the patient populations and trial sizes:

Customer Segment Focus	Product Candidate	Patient Population/Trial Size	Key Metric/Data Point
SMA (US/Europe)	Apitegromab	Estimated 30,000 to 35,000 people	Targeted for US launch in 2025 upon approval
SMA (US only)	Apitegromab	Approximately 9,000 people	FDA PDUFA target action date of September 22, 2025
SMA (SAPPHIRE Trial)	Apitegromab	Main Efficacy: 156 patients (aged 2-12)	Exploratory Population: 32 patients (aged 13-21)
Obesity/Cardiometabolic	Apitegromab (EMBRAZE)	Target Enrollment: 100 subjects (aged 18-65)	Primary Endpoint: Change in lean muscle mass over 24 weeks
Obesity/Cardiometabolic	SRK-439 (Future)	Preclinical: DIO mice	IND filing targeted for second half of 2025

The customer segments Scholar Rock Holding Corporation is engaging with or planning to engage with include:

• Children and adults with Spinal Muscular Atrophy (SMA) receiving SMN-targeted treatment.
• Nonambulatory SMA patients aged 2-12 in the SAPPHIRE trial main population.
• SMA patients under two years of age targeted by the Phase 2 OPAL study.
• Adults with obesity/overweight receiving GLP-1 RA therapy, with 100 subjects targeted in the EMBRAZE trial.
• Patients with other rare, severe neuromuscular disorders, including preclinical work in a Duchenne muscular dystrophy (DMD) model.

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Cost Structure

You're looking at the cost side of Scholar Rock Holding Corporation's business as they push toward a potential commercial launch. For a pre-revenue biotech, this structure is dominated by getting the science and the supply chain ready. Here's the quick math on what they spent in the third quarter of 2025.

The primary cost drivers reflect the intense, non-recurring investments needed to transition from clinical development to a commercial-ready entity, especially after the regulatory setback earlier in the year.

The total operating expenses for Scholar Rock Holding Corporation in Q3 2025 hit $103 million. Excluding the non-cash component, operating expenses were $85.3 million.

Here is a breakdown of the major expense categories for the quarter ended September 30, 2025:

Cost Component	Q3 2025 Amount (USD)	Notes
Research and Development (R&D) Expense	$50.5 million	Includes investment in commercial manufacturing and launch readiness.
General and Administrative (G&A) Expense	$53.1 million	Significantly up year-over-year due to pre-launch infrastructure, headcount, and professional services.
Total Stock-based Compensation (SBC)	$18.3 million	A non-cash expense included in the operating costs.
R&D Stock-based Compensation	$5.5 million	Portion of R&D expense.
G&A Stock-based Compensation	$12.8 million	Portion of G&A expense.
Net Loss	$102.2 million	Reflects the high operating burn rate before revenue generation.

You'll see that the R&D and G&A figures provided in the outline add up to more than the total operating expense because the SBC is double-counted in the line items but correctly accounted for in the total OpEx calculation when using the non-SBC figure. The total R&D and G&A spend for the quarter was $103.6 million ($50.5M + $53.1M), which aligns closely with the reported total operating expense of $103 million, accounting for rounding or minor classification differences in the reported figures.

Specifics on other major cost areas include:

• - Manufacturing and drug supply costs for commercial inventory build are a primary driver of the increased R&D expense year-over-year.
• - Clinical trial expenses support the ongoing OPAL Phase 2 study in infants and toddlers with Spinal Muscular Atrophy (SMA) and the planned start of Phase 1 for SRK-439 in Q4 2025.
• - Personnel costs are embedded within both R&D and G&A, with G&A specifically citing headcount and severance costs contributing to its rise.

The company ended Q3 2025 with $369.6 million in cash, cash equivalents, and marketable securities, which management expects will fund operations into 2027. Finance: draft 13-week cash view by Friday.

Scholar Rock Holding Corporation (SRRK) - Canvas Business Model: Revenue Streams

You're looking at the revenue side of Scholar Rock Holding Corporation's business model as of late 2025. Honestly, for a company at this stage, the revenue streams are almost entirely potential, tied to clinical success and regulatory approvals. The current financial reality reflects a pre-commercial entity heavily focused on R&D investment.

The most immediate data point from the third quarter of 2025 shows a clear picture of their current revenue generation status.

• - Scholar Rock Holding Corporation did not record any product revenue for the quarter ended September 30, 2025, which is expected for a company in this phase. This translates to $0.0 million in product revenue for Q3 2025.

The primary near-term revenue driver is the anticipated commercialization of apitegromab for Spinal Muscular Atrophy (SMA). You know the Biologics License Application (BLA) faced a delay due to a third-party manufacturing site issue, but management is pushing hard now. They anticipate resubmitting the BLA and launching apitegromab in the U.S. in 2026, pending FDA reinspection results. The European Medicines Agency (EMA) decision is expected around the middle of 2026. This product targets the SMA market, which is estimated to be worth approximately $5 billion annually.

The structure of the strategic collaboration with Gilead Sciences provides a clear, albeit contingent, revenue framework. This deal, focused on TGF$\beta$ inhibitors for fibrotic diseases, is a classic biotech financing mechanism, securing non-dilutive cash and future upside. Here's a breakdown of the key financial components from that agreement:

Revenue Component	Amount/Structure	Status/Notes
Upfront Payment	$80 million total ($50 million cash, $30 million stock purchase)	Received upon agreement execution (2018)
Preclinical Milestone Payment	$25 million	Earned for successful preclinical in vivo proof-of-concept studies
Potential Future Milestones (Aggregated)	Up to an additional $1,425 million	Based on research, development, regulatory, and commercialization success across three programs
Royalties on Future Sales	High single-digit to low double-digit tiered royalties	Contingent on commercial sales of licensed product candidates

Beyond apitegromab, Scholar Rock Holding Corporation is advancing other pipeline assets, which represent longer-term, more speculative revenue opportunities. The next concrete step in this area involves SRK-439, a next-generation myostatin inhibitor. The company cleared the Investigational New Drug (IND) application for this asset and plans to initiate first-in-human dosing in the fourth quarter of 2025. Also, Scholar Rock Holding Corporation expects to initiate clinical development activities in a second neuromuscular indication by the end of 2025.

To summarize the non-product revenue sources that underpin operations:

• - Milestone payments from the Gilead collaboration have already included a $25 million payment.
• - The potential for future milestone payments from Gilead remains significant, totaling up to $1,425 million across the programs.
• - Royalties are structured as high single-digit to low double-digit tiers on any future commercial products from the collaboration.
• - SRK-439 is set to enter Phase 1 clinical trials in Q4 2025, which will precede any revenue generation from that program.

Finance: draft 13-week cash view by Friday.