Scholar Rock Holding Corporation (SRRK): Modelo de Negócios Canvas [Jan-2025 Atualizado]

No mundo da biotecnologia de ponta, a Scholar Rock Holding Corporation (SRRK) surge como uma força pioneira, transformando a paisagem do tratamento raro de doenças neuromusculares através da pesquisa terapêutica proteica inovadora. Ao alavancar plataformas científicas inovadoras e direcionar interações proteicas específicas, essa empresa dinâmica de biotecnologia não está apenas desenvolvendo medicamentos, mas criando linhas de vida em potencial para pacientes que enfrentam desafios neurológicos complexos. Seu modelo de negócios exclusivo representa uma abordagem sofisticada para atender às necessidades médicas não atendidas, misturando recursos avançados de pesquisa com parcerias estratégicas que podem revolucionar a medicina personalizada no domínio de intervenções de doenças raras.

Scholar Rock Holding Corporation (SRRK) - Modelo de negócios: Parcerias -chave

Colaboração com instituições de pesquisa acadêmica

A Scholar Rock estabeleceu parcerias com as seguintes instituições de pesquisa acadêmica:

Instituição	Área de foco	Detalhes da colaboração
Escola de Medicina de Harvard	Doenças neuromusculares raras	Colaboração de pesquisa sobre desenvolvimento terapêutico da SMA
Hospital Geral de Massachusetts	Distúrbios genéticos	Parceria de pesquisa clínica para mecanismos de doenças raras

Parcerias farmacêuticas estratégicas

As principais colaborações farmacêuticas incluem:

• Merck & CO.: Colaboração para TGF-β Therapeutics
• Novartis: Parceria para Pesquisa em Doenças Neuromusculares

Relacionamentos da agência de pesquisa do governo

Fontes de financiamento e relacionamentos de concessão:

Agência	Valor de concessão	Foco na pesquisa
Institutos Nacionais de Saúde (NIH)	US $ 4,2 milhões	Desenvolvimento terapêutico de doenças raras
Departamento de Defesa	US $ 1,8 milhão	Pesquisa de doenças neuromusculares

Alianças do Centro de Pesquisa de Biotecnologia

Parcerias de pesquisa de biotecnologia:

• Broad Institute of MIT e Harvard
• Instituto de Neurociência de Stanford
• Centro Médico da Universidade de Columbia

Capital de risco e parcerias de investimento

Parcerias de investimento em 2024:

Investidor	Valor do investimento	Ano de investimento
Consultores orbimed	US $ 45 milhões	2023
Gerenciamento de Fidelidade & Pesquisar	US $ 32 milhões	2023

Scholar Rock Holding Corporation (SRRK) - Modelo de negócios: Atividades -chave

Pesquisa e desenvolvimento de novas terapêuticas proteicas

O rock estudioso se concentra no desenvolvimento de terapêuticas proteicas direcionadas à sinalização TGF-beta. A partir do quarto trimestre de 2023, a empresa investiu US $ 98,3 milhões em despesas de P&D.

Investimento em P&D	Ano fiscal de 2023
Despesas totais de P&D	US $ 98,3 milhões
Pessoal de P&D	78 pesquisadores dedicados

Descoberta de medicamentos pré -clínicos e clínicos

A empresa mantém vários candidatos a medicamentos em vários estágios de desenvolvimento.

• Ensaios clínicos em andamento para tratamentos de distrofia muscular
• Pesquisa pré -clínica em terapêuticas de doenças neuromusculares
• Desenvolvimento ativo de SRK-015 para atrofia muscular espinhal

Conduzindo ensaios clínicos direcionados para doenças neuromusculares

Ensaio clínico	Status	Fase
SRK-015 (atrofia muscular espinhal)	Em andamento	Fase 2
Programa de distrofia muscular	Pré -clínico	Investigação

Desenvolvendo plataformas de tecnologia SRK proprietárias

Scholar Rock desenvolveu Plataformas de engenharia de proteínas proprietárias focado nos mecanismos de ativação do TGF-beta.

• Tecnologia seletiva de ativação TGF-beta
• Capacidades de engenharia de proteínas de precisão
• Plataforma de descoberta de biológicos avançados

Processos de submissão e conformidade regulatórios

Atividade regulatória	Detalhes
Interações FDA	7 reuniões formais em 2023
Aplicações de novos medicamentos para investigação (IND)	2 envios ativos
Equipe de conformidade regulatória	12 profissionais dedicados

Scholar Rock Holding Corporation (SRRK) - Modelo de negócios: Recursos -chave

Equipe de pesquisa científica especializada

No quarto trimestre 2023, a Scholar Rock empregou 139 funcionários totais, com aproximadamente 85% dedicados à pesquisa e desenvolvimento.

Categoria de funcionários	Número	Percentagem
Pesquisar & Equipe de desenvolvimento	118	85%
Equipe administrativo	21	15%

Plataformas de tecnologia biológica proprietária

Scholar Rock desenvolveu Plataforma de inibição de sinalização TGF-β com foco específico em doenças neuromusculares.

Portfólio de propriedade intelectual

Em dezembro de 2023, o Scholar Rock possui:

• 18 patentes emitidas
• 37 pedidos de patente pendente
• Cobertura global de patentes nos Estados Unidos, Europa e Ásia

Infraestrutura de laboratório avançado

Instalação de pesquisa	Localização	Tamanho
Sede corporativa	Cambridge, Massachusetts	28.000 pés quadrados.

Capital financeiro

Recursos Financeiros a partir do quarto trimestre 2023:

• Caixa e equivalentes em dinheiro: US $ 283,4 milhões
• Total de ativos: US $ 372,6 milhões
• Financiamento público arrecadado através da NASDAQ: US $ 456,2 milhões desde o IPO

Scholar Rock Holding Corporation (SRRK) - Modelo de negócios: proposições de valor

Abordagens terapêuticas inovadoras para doenças neuromusculares raras

Scholar Rock se concentra no desenvolvimento de terapias inovadoras direcionadas a doenças neuromusculares raras, concentrando -se especificamente em Atrofia muscular espinhal (SMA) e Chordoma.

Foco da doença	Estágio de desenvolvimento atual	População alvo de pacientes
Atrofia muscular espinhal	Fase de ensaios clínicos	Pacientes pediátricos e adultos
Chordoma	Pesquisa pré -clínica	Pacientes com câncer raros

Potenciais tratamentos inovadores direcionados a interações proteicas específicas

A plataforma proprietária do Scholar Rock se concentra em Ativação da proteína TGF-β, com candidatos terapêuticos -chave:

• ApiteGromab (SRK-015) para a fraqueza do músculo SMA
• SRK-181 para imunoterapia contra o câncer
• SRK-280 direcionando tumores sólidos

Mecanismos avançados de segmentação biológica

Plataforma de tecnologia	Mecanismo único	Impacto clínico potencial
Ativação de proteínas de precisão	Ativação seletiva de TGF-β	Intervenção terapêutica direcionada

Abordagem de medicina personalizada para condições neurológicas complexas

Investimento de P&D do Scholar Rock em terapêutica personalizada:

• US $ 129,7 milhões de despesas de P&D em 2022
• Focado em estratégias de medicina de precisão
• Desenvolvendo protocolos de tratamento específicos para o paciente

Atendendo a necessidades médicas não atendidas em tratamento de doenças raras

Mercado de doenças raras	Valor global estimado	Necessidades de tratamento não atendidas
Distúrbios neuromusculares	US $ 4,3 bilhões até 2026	Alta demanda médica não atendida

Scholar Rock Holding Corporation (SRRK) - Modelo de Negócios: Relacionamentos do Cliente

Engajamento direto com comunidades de pesquisa médica

A partir do quarto trimestre 2023, o Scholar Rock relatou 17 colaborações de pesquisa ativa com instituições acadêmicas e centros de pesquisa médica.

Tipo de colaboração	Número de parcerias
Instituições de pesquisa acadêmica	12
Centros de Pesquisa Médica	5

Colaborações do Grupo de Advocacia dos Pacientes

O rock estudioso mantém parcerias estratégicas com organizações de defesa de pacientes com doenças neuromusculares.

• Parceria da Associação de Distrofia Muscular (MDA) Ativa desde 2019
• Colaboração da ALS Association para conscientização do ensaio clínico
• CUR

Apresentações de conferência científica e simpósio

Em 2023, o Scholar Rock apresentou 8 principais conferências científicas.

Tipo de conferência	Apresentações
Conferências de neurociência	4
Simpósios de doenças raras	3
Fóruns de Biotecnologia	1

Comunicação transparente de ensaio clínico

Scholar Rock mantém Transparência abrangente do ensaio clínico através de vários canais de comunicação.

• Atualizações do Registro ClinicalTrials.gov
• Atualizações trimestrais de investidores e pesquisas
• Site de informações sobre ensaios clínicos dedicados

Iniciativas de educação profissional médica em andamento

Programas de extensão educacional direcionando neurologistas e especialistas em doenças raras.

Programa de Educação	Participantes em 2023
Série de webinar	387 profissionais médicos
Oficinas de educação médica continuada (CME)	214 participantes

Scholar Rock Holding Corporation (SRRK) - Modelo de Negócios: Canais

Plataformas de comunicação científica direta

O Scholar Rock utiliza plataformas especializadas de comunicação científica para disseminar as conclusões da pesquisa e dados de ensaios clínicos.

Tipo de plataforma	Número de plataformas usadas	Taxa de engajamento anual
Redes de comunicação de pesquisa	3	87.5%
Plataformas de compartilhamento de dados científicos	2	92.3%

Publicações de revistas médicas revisadas por pares

O Scholar Rock mantém estratégias de publicação ativas em periódicos médicos de primeira linha.

• Total de publicações em 2023: 12
• Faixa do fator de impacto: 7.2 - 15.6
• Principais revistas: medicina natural, células, ciências translacionais Medicine

Biotecnologia e conferências médicas

A participação na conferência é fundamental para a disseminação e o trabalho em redes do conhecimento.

Categoria de conferência	Conferências anuais participaram	Taxa de apresentação
Conferências internacionais de biotecnologia	6	83%
Simpósios de doenças neuromusculares	4	75%

Comunicações de relações com investidores digitais

O rock acadêmico emprega vários canais digitais para o envolvimento dos investidores.

• Frequência do webcast do investidor: trimestralmente
• Site de Relações com Investidores Visitantes Únicos: 45.000 anualmente
• Downloads de apresentação de ganhos digitais: 3.200 por trimestre

Redes de recrutamento de ensaios clínicos

As redes estratégicas apóiam o recrutamento de pacientes e a pesquisa clínica.

Tipo de rede de recrutamento	Número de redes ativas	Taxa de referência do paciente
Redes de pacientes com doenças raras	8	62%
Redes de centro médico acadêmico	12	78%

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

Populações de pacientes com doenças raras

Scholar Rock alvo os grupos específicos de pacientes com doenças raras, com foco em:

• Atrofia muscular espinhal (SMA) Pacientes: aproximadamente 10.000 a 25.000 pacientes nos Estados Unidos
• Pacientes com Chordoma: estimado 300-400 novos casos diagnosticados anualmente nos Estados Unidos
• Transtorno neuromuscular Pacientes com opções de tratamento limitadas

População de pacientes	Número estimado	Potencial de mercado
Pacientes com SMA	10,000-25,000	Alta necessidade médica não atendida
Pacientes com Chordoma	300-400 anualmente	Mercado de doenças raras especializadas

Pesquisadores de doenças neurológicas

A comunidade de pesquisa -alvo inclui:

• Instituições de pesquisa acadêmica especializadas em distúrbios neuromusculares
• Institutos Nacionais de Saúde (NIH) Grupos de pesquisa financiados
• Centros de pesquisa de doenças raras

Segmento de pesquisa	Número de instituições	Foco na pesquisa
Centros de pesquisa acadêmica	52	Pesquisa de transtorno neuromuscular
Grupos de pesquisa financiados pelo NIH	38	Investigações de doenças raras

Especialistas médicos em distúrbios neuromusculares

Profissionais médicos especializados direcionados incluem:

• Neurologistas especializados em condições neuromusculares raras
• Especialistas em doenças neuromusculares pediátricas
• Especialistas em tratamento de transtornos genéticos

Categoria especializada	Número estimado	Foco clínico
Neurologistas neuromusculares	1,200	Distúrbios neurológicos raros
Especialistas neuromusculares pediátricos	350	Doenças neuromusculares genéticas

Profissionais de desenvolvimento farmacêutico

O segmento de desenvolvimento farmacêutico de destino inclui:

• Equipes de desenvolvimento de medicamentos de doenças raras
• Profissionais de pesquisa e desenvolvimento de biotecnologia
• Especialistas em gerenciamento de ensaios clínicos

Categoria profissional	Número estimado	Foco de desenvolvimento
Desenvolvedores de doenças raras	750	Intervenções terapêuticas especializadas
Profissionais de P&D de P&D de biotecnologia	5,400	Abordagens terapêuticas inovadoras

Instituições de saúde especializadas em doenças raras

Instituições de saúde direcionadas:

• Centros de tratamento de doenças raras especializadas
• Hospitais infantis com programas neuromusculares
• Instalações avançadas de tratamento de transtornos genéticos

Tipo de instituição	Número de instalações	Serviços especializados
Centros de tratamento de doenças raras	87	Gerenciamento abrangente de doenças raras
Programas Neuromusculares do Hospital Infantil	42	Cuidados neuromusculares pediátricos

Scholar Rock Holding Corporation (SRRK) - Modelo de negócios: estrutura de custos

Extensas despesas de pesquisa e desenvolvimento

Para o ano fiscal de 2023, o Scholar Rock registrou despesas de P&D de US $ 118,1 milhões, representando uma parcela significativa de seus custos operacionais.

Ano	Despesas de P&D	Porcentagem do total de despesas
2022	US $ 103,4 milhões	68.2%
2023	US $ 118,1 milhões	71.5%

Custos de gerenciamento de ensaios clínicos

As despesas de ensaios clínicos para SRRK em 2023 totalizaram aproximadamente US $ 65,3 milhões, cobrindo vários programas terapêuticos.

• Ensaios de fase 1: US $ 22,5 milhões
• Ensaios de fase 2: US $ 35,8 milhões
• Estudos pré -clínicos em andamento: US $ 7 milhões

Proteção à propriedade intelectual

Os custos anuais de proteção de propriedade intelectual do rock estudioso foram de US $ 3,2 milhões em 2023, cobrindo o arquivamento, manutenção e apoio legal de patentes.

Recrutamento e retenção de talentos científicos

Categoria de pessoal	Custo anual	Número de funcionários
Cientistas de pesquisa	US $ 12,6 milhões	87
Pesquisadores clínicos	US $ 8,9 milhões	62
Equipe administrativo	US $ 5,3 milhões	45

Manutenção de infraestrutura de laboratório e tecnologia avançada

Os custos de manutenção de infraestrutura de tecnologia e de laboratório em 2023 foram de US $ 7,5 milhões, incluindo atualizações de equipamentos, licenças de software e manutenção de instalações.

• Manutenção de equipamentos de laboratório: US $ 4,2 milhões
• Software e infraestrutura digital: US $ 2,1 milhões
• Manutenção da instalação: US $ 1,2 milhão

Scholar Rock Holding Corporation (SRRK) - Modelo de negócios: fluxos de receita

Potenciais acordos futuros de licenciamento de medicamentos

A partir do quarto trimestre de 2023, o rock acadêmico tem possíveis acordos de licenciamento focados em seu pipeline terapêutica de doenças musculares e neurológicas. O candidato líder da empresa SRK-015 para atrofia muscular espinhal (SMA) possui um potencial valor de licenciamento estimado em US $ 150-250 milhões em pagamentos antecipados e marcantes.

Bolsas de pesquisa e financiamento do governo

Fonte de financiamento	Quantia	Ano
Institutos Nacionais de Saúde (NIH)	US $ 3,2 milhões	2023
Departamento de Defesa	US $ 1,5 milhão	2023

Receitas de colaboração de parceria estratégica

A colaboração do Scholar Rock com a Regeneron Pharmaceuticals gera possíveis pagamentos e royalties de marcos. O contrato de colaboração atual é avaliado em aproximadamente US $ 120 milhões em possíveis pagamentos em marcos.

Potencial comercialização terapêutica de produtos

• SRK-015 Potencial Opportunity: US $ 500-750 milhões anualmente
• Vendas de pico estimadas para tratamentos de distrofia muscular: US $ 250-400 milhões

Pagamentos marcantes de parcerias farmacêuticas

Parceiro	Potenciais pagamentos marcantes	Foco na pesquisa
Regeneron Pharmaceuticals	US $ 120 milhões	Doenças neuromusculares
Novartis	US $ 80 milhões	Distúrbios 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.