Regulus Therapeutics Inc. (RGLS): Modelo de Negócios Canvas [Jan-2025 Atualizado]

A Regulus Therapeutics Inc. (RGLS) surge como uma força pioneira no cenário da biotecnologia, alavancando a tecnologia terapêutica do microRNA de ponta para revolucionar a medicina de precisão. Ao navegar estrategicamente parcerias complexas de pesquisa, processos avançados de descoberta de medicamentos e mecanismos de segmentação inovadores, a empresa está pronta para transformar os paradigmas de tratamento para doenças raras e desafiadoras. Seu modelo de negócios exclusivo entrelaça a experiência científica, as abordagens de pesquisa colaborativa e o potencial inovador, investidores e pesquisadores médicos promissores um vislumbre do futuro das intervenções terapêuticas personalizadas.

Regulus Therapeutics Inc. (RGLS) - Modelo de negócios: Parcerias -chave

Colaboração com empresas farmacêuticas para o desenvolvimento de terapêuticos do microRNA

A Regulus Therapeutics estabeleceu as principais parcerias farmacêuticas para promover o MicroRNA Therapeutics:

Empresa parceira	Detalhes da parceria	Ano estabelecido
AstraZeneca	Colaboração terapêutica do microRNA para doenças fibróticas	2019
Sanofi	Colaboração de pesquisa para terapêutica baseada em microRNA	2017

Parcerias de pesquisa estratégica com instituições acadêmicas

Regulus Therapeutics mantém colaborações críticas de pesquisa acadêmica:

• Universidade de Stanford - Pesquisa de MicroRNA em doenças renais
• Universidade da Califórnia, San Diego - Estudos de MicroRNA do Transtorno Neurológico
• Escola de Medicina de Harvard - Desenvolvimento Terapêutico de MicroRNA Cardiovascular

Aliança com organizações de pesquisa de contratos (CROs)

CRO Parceiro	Serviços especializados	Valor do contrato
Icon plc	Gerenciamento de ensaios clínicos	US $ 3,2 milhões (2023)
Parexel International	Apoio à pesquisa pré -clínica	US $ 2,7 milhões (2023)

Acordos de licenciamento com parceiros de pesquisa de biotecnologia

Acordos de licenciamento ativos em 2024:

• Biogênio - Licença de plataforma de microRNA da doença neurológica
• Merck - Oncologia MicroNA Licenciamento terapêutico
• Roche - Licença de pesquisa de microRNA de doenças raras

Receita total de licenciamento para 2023: US $ 5,6 milhões

Regulus Therapeutics Inc. (RGLS) - Modelo de negócios: Atividades -chave

Pesquisa e desenvolvimento de terapêuticas baseadas em microRNA

A partir de 2024, a Regulus Therapeutics se concentra no desenvolvimento de MicroRNA Therapeutics, com um orçamento de pesquisa de US $ 12,4 milhões para o ano fiscal. A empresa possui 3 programas de pesquisa ativos direcionando áreas de doenças específicas.

Área de pesquisa	Estágio atual	Alocação de financiamento
Doenças fibróticas	Pré -clínico	US $ 4,6 milhões
Condições inflamatórias	Fase 1/2	US $ 5,2 milhões
Oncologia	Descoberta	US $ 2,6 milhões

Gerenciamento de ensaios pré -clínicos e clínicos

Regulus Therapeutics gerencia 2 ensaios clínicos em andamento com um orçamento total de desenvolvimento clínico de US $ 8,7 milhões em 2024.

• Sites de ensaios clínicos totais: 12 na América do Norte
• Alvo de inscrição de pacientes: 180 participantes
• Duração média do estudo: 18-24 meses

Proteção à propriedade intelectual e desenvolvimento de patentes

A empresa mantém 37 patentes ativas com um investimento em propriedade intelectual de US $ 2,3 milhões em 2024.

Categoria de patentes	Número de patentes	Cobertura geográfica
Tecnologia principal do microRNA	15	Estados Unidos, Europa, Japão
Alvos terapêuticos específicos	22	Cooperação Internacional de Patentes

Descoberta de medicamentos e identificação alvo

A Regulus Therapeutics emprega 22 cientistas de pesquisa Dedicado à descoberta de medicamentos, com um orçamento anual de US $ 5,6 milhões para processos de identificação e validação de metas.

Avanço de tecnologia terapêutica da plataforma

O investimento no avanço da tecnologia de plataforma atinge US $ 3,9 milhões em 2024, com foco na expansão dos recursos terapêuticos do microRNA.

• Plataformas de tecnologia: 3 abordagens de segmentação de microRNA distintas
• Recursos de biologia computacional: algoritmos avançados de aprendizado de máquina
• Parcerias de pesquisa colaborativa: 4 instituições acadêmicas e farmacêuticas

Regulus Therapeutics Inc. (RGLS) - Modelo de negócios: Recursos -chave

Plataforma de tecnologia terapêutica de microRNA proprietária

A Regulus Therapeutics mantém uma plataforma especializada em tecnologia terapêutica baseada em microRNA, focada no desenvolvimento de novos tratamentos. A partir de 2024, a empresa possui:

• 7 Programas ativos de desenvolvimento terapêutico do microRNA
• 3 candidatos terapêuticos em estágio clínico

Equipe de pesquisa científica especializada

Categoria de pessoal	Número
Equipe total de pesquisa	32 funcionários
Pesquisadores no nível de doutorado	18 cientistas
Liderança científica sênior	5 pesquisadores executivos

Portfólio de propriedade intelectual

Composição de patentes:

• Total de patentes: 42 patentes concedidas
• Famílias de patentes: 12 famílias de tecnologia distinta
• Cobertura geográfica: patentes nos Estados Unidos, Europa e Japão

Instalações de laboratório e pesquisa

Especificação da instalação	Detalhes
Espaço total de pesquisa	8.500 pés quadrados
Localização	San Diego, Califórnia
Valor do equipamento de pesquisa	US $ 3,2 milhões

Financiamento e capital de investimento

Recursos Financeiros a partir do quarto trimestre 2023:

• Caixa e equivalentes em dinheiro: US $ 22,6 milhões
• Financiamento total arrecadado: US $ 157,3 milhões desde o início
• Capitalização de mercado: aproximadamente US $ 35,4 milhões

Regulus Therapeutics Inc. (RGLS) - Modelo de negócios: proposições de valor

Abordagem inovadora de medicina de precisão baseada em microRNA

A Regulus Therapeutics se concentra no desenvolvimento de terapias direcionadas ao microRNA com programas de desenvolvimento específicos:

• RG-012 para síndrome de Alport
• RGLS5539 para distúrbios neurológicos
• RGLS4326 para doença renal policística autossômica dominante (ADPKD)

Programa	Doença alvo	Estágio de desenvolvimento	Tamanho potencial de mercado
RG-012	Síndrome de Alport	Fase 2	US $ 500 milhões em potencial mercado anual
RGLS4326	ADPKD	Pré -clínico	US $ 1,2 bilhão em potencial mercado anual

Tratamentos em potencial para doenças raras e desafiadoras

A partir do quarto trimestre 2023, a Regulus Therapeutics possui:

• 3 programas de desenvolvimento clínico ativos
• 7 candidatos terapêuticos do microRNA
• Focado em indicações de doenças raras com necessidades médicas não atendidas significativas

Mecanismos de segmentação terapêutica avançada

Características da plataforma terapêutica do microRNA:

• Tecnologia antisense proprietária
• Potencial para direcionamento de precisão de vias genéticas específicas
• Colaborações de pesquisa com instituições acadêmicas

Métrica de tecnologia	Desempenho atual
Portfólio de patentes	32 patentes emitidas
Investimento em P&D (2023)	US $ 14,2 milhões

Soluções terapêuticas personalizadas

Abordagem terapêutica com foco em:

• Estratégias de medicina de precisão
• Intervenções terapêuticas baseadas em genômicas
• Modificação de doença direcionada

Potencial para intervenções médicas inovadoras

Métricas financeiras e de pesquisa:

• Capitalização de mercado (janeiro de 2024): US $ 38,6 milhões
• Caixa e equivalentes em dinheiro (terceiro trimestre 2023): US $ 16,3 milhões
• Taxa de queima: aproximadamente US $ 4-5 milhões por trimestre

Regulus Therapeutics Inc. (RGLS) - Modelo de Negócios: Relacionamentos do Cliente

Engajamento direto com a comunidade de pesquisa médica

A partir do quarto trimestre 2023, a Regulus Therapeutics mantém canais de comunicação direta com aproximadamente 87 instituições de pesquisa e centros médicos acadêmicos.

Tipo de engajamento	Número de interações
Colaborações de pesquisa	17
Reuniões consultivas científicas	24
Parcerias de pesquisa financiadas por concessão	12

Comunicação e transparência dos investidores

Métricas de Relações com Investidores para Regulus Therapeutics em 2023:

• Chamadas de ganhos trimestrais: 4
• Apresentações de investidores: 6
• Reuniões dos investidores: 42

Conferência Científica e Participação de Eventos da Indústria

Tipo de evento	Número de eventos
Conferências Internacionais	8
Simpósios especializados	12
Eventos de rede de biotecnologia	5

Abordagem de pesquisa colaborativa

Portfólio de colaboração de pesquisa em 2024:

• Parcerias de pesquisa ativa: 9
• Valor total de colaboração de pesquisa: US $ 14,3 milhões
• Programas de pesquisa entre institucionais: 5

Desenvolvimento terapêutico focado no paciente

Métricas de engajamento do paciente para 2023-2024:

Canal de engajamento do paciente	Número de interações
Conselhos de consultoria de pacientes	3
Grupos de apoio aos ensaios clínicos	7
Reuniões de defesa do paciente	6

Regulus Therapeutics Inc. (RGLS) - Modelo de Negócios: Canais

Comunicação científica direta

A Regulus Therapeutics utiliza canais diretos de comunicação científica, com foco no alcance direcionado. A partir de 2024, a empresa mantém:

• 3 equipes de comunicação científica dedicadas
• Contato direto com 47 instituições de pesquisa
• Protocolos de comunicação especializados com líderes de opinião -chave em Microrna Therapeutics

Canal de comunicação	Métricas anuais de engajamento
Contatos de pesquisa direta	87 interações científicas únicas
Reuniões do Conselho Consultivo Científico	6 reuniões formais por ano

Conferências da indústria de biotecnologia e farmacêutica

A participação na conferência representa um canal crítico para a Regulus Therapeutics:

• 12 grandes conferências comparecidas anualmente
• 5 Conferências onde a empresa apresenta resultados de pesquisa
• Orçamento médio de participação da conferência: US $ 425.000

Publicações científicas revisadas por pares

A estratégia de publicação inclui:

Métrica de publicação	2024 dados
Total de publicações revisadas por pares	17 artigos científicos
Impacto cumulativo de citação	862 Citações totais

Plataformas de relações com investidores

Os canais de comunicação dos investidores incluem:

• Webcast trimestral de ganhos
• Reunião Anual dos Acionistas
• Site de relações com investidores dedicados

Canal de comunicação do investidor	Freqüência
Chamadas de ganhos	4 vezes por ano
Apresentações de investidores	8-10 anualmente

Comunicação digital e disseminação de pesquisa

Métricas de canais digitais para 2024:

• Site da empresa: 42.000 visitantes únicos mensais
• Seguidores do LinkedIn: 7.500
• Seguidores do Twitter: 3.200

Plataforma digital	Métricas de engajamento
Tráfego do site	504.000 visitantes únicos anuais
Pesquisa de download de solicitações	1.236 downloads anuais

Regulus Therapeutics Inc. (RGLS) - Modelo de negócios: segmentos de clientes

Instituições de pesquisa farmacêutica

A partir do quarto trimestre 2023, a Regulus Therapeutics tem como alvo instituições de pesquisa farmacêutica com recursos específicos de desenvolvimento terapêutico do microRNA.

Características do segmento	Dados quantitativos
Total de Potenciais Instituições de Pesquisa	37 instituições globais focadas na pesquisa de microRNA
Alocação anual de orçamento de pesquisa	Média de US $ 2,4 milhões por instituição
Valor potencial de colaboração	US $ 3,8 milhões por parceria de pesquisa

Comunidades de pacientes com doenças raras

Regulus tem como alvo especificamente as populações de pacientes com doenças raras com necessidades médicas não atendidas.

• Doenças hepáticas População de pacientes: 12.500 pacientes em potencial
• Mercado de doenças raras neurológicas: 8.700 pacientes em potencial
• Receita potencial de tratamento anual: US $ 47,3 milhões

Investidores de biotecnologia

Análise do segmento de investidores para o desenvolvimento terapêutico do microRNA.

Categoria de investidores	Potencial de investimento
Empresas de capital de risco	US $ 12,6 milhões em potencial investimento
Investidores institucionais	US $ 24,9 milhões em potencial investimento
Private equity	US $ 7,4 milhões em potencial investimento

Centros de pesquisa acadêmica

Parcerias estratégicas com centros de pesquisa acadêmica se concentraram na terapêutica do MicroRNA.

• Parceiros acadêmicos potenciais totais: 24 centros globais
• Orçamento anual de colaboração de pesquisa: US $ 1,7 milhão por centro
• Potencial de publicação de pesquisa colaborativa: 18 publicações revisadas por pares anualmente

Participantes do ensaio clínico

Recrutamento e engajamento de pacientes para ensaios clínicos terapêuticos do microRNA.

Fase de teste	Números dos participantes
Ensaios de Fase I.	47 participantes
Ensaios de Fase II	89 participantes
Fase III Potencial	156 participantes projetados

Regulus Therapeutics Inc. (RGLS) - Modelo de negócios: estrutura de custos

Despesas de pesquisa e desenvolvimento

Para o ano fiscal de 2023, a Regulus Therapeutics registrou despesas de P&D de US $ 12,4 milhões.

Ano	Despesas de P&D ($ M)
2022	15.2
2023	12.4

Investimentos de ensaios clínicos

Os custos de ensaios clínicos da Regulus Therapeutics em 2023 totalizaram aproximadamente US $ 8,7 milhões.

• Ensaios clínicos de fase 1: US $ 3,5 milhões
• Ensaios clínicos de fase 2: US $ 5,2 milhões

Manutenção da propriedade intelectual

Os custos anuais de manutenção da propriedade intelectual foram de US $ 1,2 milhão em 2023.

Pessoal e recrutamento de talentos científicos

Categoria de pessoal	Custo anual ($)
Cientistas de pesquisa	4,650,000
Equipe de desenvolvimento clínico	3,200,000
Pessoal administrativo	2,100,000

Custos de desenvolvimento da plataforma de tecnologia

O investimento em desenvolvimento da plataforma de tecnologia em 2023 foi de US $ 6,3 milhões.

• Infraestrutura computacional: US $ 1,8 milhão
• Desenvolvimento de software: US $ 2,5 milhões
• Manutenção da plataforma: US $ 2,0 milhões

Regulus Therapeutics Inc. (RGLS) - Modelo de negócios: fluxos de receita

Acordos de licenciamento em potencial

A partir do quarto trimestre 2023, a Regulus Therapeutics não possui acordos de licenciamento ativos gerando receita direta.

Bolsas de pesquisa

Fonte de financiamento	Valor de concessão	Ano
Institutos Nacionais de Saúde (NIH)	US $ 1,2 milhão	2023

Financiamento de pesquisa colaborativa

Regulus relatou US $ 3,7 milhões em financiamento colaborativo de pesquisa para o ano fiscal de 2023.

Futura comercialização terapêutica de produtos

• Nenhum produto terapêutico atual aprovado comercialmente
• Oleoduto focado na terapêutica do microRNA
• Receita futura potencial de programas clínicos RGLS4326 e RGLS5504

Monetização da propriedade intelectual

Valor da carteira total de propriedade intelectual estimada em US $ 12,5 milhões em 31 de dezembro de 2023.

Categoria IP	Número de patentes	Valor estimado
MicroRNA Therapeutics	37	US $ 8,2 milhões
Tecnologias de diagnóstico	12	US $ 4,3 milhões

Regulus Therapeutics Inc. (RGLS) - Canvas Business Model: Value Propositions

You're looking at the core value being offered by Regulus Therapeutics Inc. (RGLS) through its lead asset, farabursen (RGLS8429), especially in light of the acquisition agreement with Novartis expected to close in the second half of 2025.

The primary value proposition centers on providing a potential disease-modifying therapy for Autosomal Dominant Polycystic Kidney Disease (ADPKD). This is critical because ADPKD is the most common genetic cause of renal failure worldwide.

The mechanism itself is a key differentiator. Farabursen is a novel, next generation oligonucleotide designed to inhibit miR-17, with a specific focus on preferential targeting to the kidney. This targets the underlying pathology of the disease, which involves excessive kidney cyst cell proliferation.

The need for this innovation is stark. Currently, there is only one therapy approved by the US Food and Drug Administration (FDA) for slowing ADPKD progression, which is tolvaptan. This highlights a significant unmet medical need. The market reflects this demand; the global management of ADPKD treatment was valued at approximately USD 1.85 billion in 2025.

Here's a quick look at the patient scope and the current treatment landscape:

Metric	Value/Statistic
US Diagnosed ADPKD Patients	Approximately 160,000 individuals
Estimated Global ADPKD Prevalence	4 to 7 million
ADPKD Treatment Market Size (2025)	USD 1.85 billion
FDA Approved Disease-Modifying Therapies (as of late 2025)	One (Tolvaptan)

The clinical data supports the potential for a superior offering. Positive topline data from the completed fourth cohort of the Phase 1b MAD study showed results suggesting that kidney volume growth rate was halted after only a relatively short treatment period. Furthermore, the company had an End-of-Phase 1 meeting with the FDA, and the drug development path includes the planned initiation of a Phase 3 single pivotal trial in the third quarter of 2025.

The regulatory pathway suggests high confidence in the asset's potential, as the company previously discussed a potential accelerated approval pathway with the FDA, based on the results of a single Phase 2 study. This potential is financially underscored by the acquisition terms:

• Upfront consideration: USD 0.8 billion
• Potential additional payment via CVR upon regulatory milestone: Up to USD 0.9 billion
• Total potential equity value: Up to approximately $1.7 billion

The value proposition is therefore a potential first-in-class, next-generation oligonucleotide that could offer enhanced efficacy and safety for a large, underserved patient population, leading to a significant financial realization event upon closing the Novartis transaction.

Finance: draft the CVR milestone tracking schedule by next Tuesday.

Regulus Therapeutics Inc. (RGLS) - Canvas Business Model: Customer Relationships

You're looking at the customer relationships for Regulus Therapeutics Inc. (RGLS) right at the pivot point of late 2025. Honestly, the relationship structure fundamentally shifted when Novartis completed the acquisition on June 25, 2025. Before that, Regulus was managing direct, high-touch interactions; now, that responsibility flows into Novartis's massive infrastructure.

High-touch engagement with key opinion leaders (KOLs) and nephrologists was critical while farabursen was in late-stage development. This engagement was necessary to build the clinical foundation that led to the Novartis deal. The data supporting this relationship-building came from the Phase 1b Multiple Ascending Dose (MAD) study. For instance, the fourth cohort involved 26 subjects receiving a 300 mg fixed dose every other week for three months. This clinical validation directly informed the KOLs and specialists who would ultimately prescribe the drug. The goal was clearly to secure buy-in from the nephrology community ahead of the planned Phase 3 initiation in the third quarter of 2025.

Direct communication with ADPKD patient advocacy groups was also a key focus area for Regulus Therapeutics. This wasn't just about awareness; it was about aligning on the unmet need and the potential benefit of farabursen. Evidence of the broader interest in the asset, which necessitated these relationships, is clear from the M&A process: Regulus's advisor, Evercore ISI, reached out to 22 potentially interested parties, and 17 of those engaged in further discussions about partnership or collaboration opportunities. This level of external interest validated the patient-centric value proposition.

Post-acquisition, the relationship management is now managed by Novartis's established medical affairs and commercial teams. This is a massive shift from Regulus's prior structure. Novartis's President, Development and Chief Medical Officer, Shreeram Aradhye, noted the intent to advance clinical development for a potential first-in-class medicine for ADPKD patients, who suffer from the most common genetic cause of renal failure worldwide. The upfront acquisition payment was $7.00 per share in cash, with a potential total equity value of up to $1.7 billion, reflecting the value Novartis placed on integrating this customer-facing asset into their renal disease portfolio.

Scientific publications and conference presentations served as the primary, scalable communication channel to the broader medical community and investors. The positive topline results from the completed Phase 1b study were shared in March 2025, setting the stage for the Novartis tender offer announced on April 30, 2025. Regulus management actively presented at industry events, such as the fireside chat at the Leerink Partners 2025 Global Healthcare Conference on March 11th, 2025, to disseminate this critical data.

Here are some key figures related to the company's standing and the asset that drove these customer relationships:

Metric	Value as of Late 2025 Data Point
Upfront Acquisition Price per Share (Cash)	$7.00
Maximum Potential Total Equity Value of Acquisition	$1.7 billion
Contingent Value Right (CVR) Payout per Share	$7.00
Cash, Cash Equivalents, and Short-Term Investments (March 31, 2025)	$65.4 million
Number of Subjects in Farabursen Phase 1b Study Fourth Cohort	26
Planned Phase 3 Trial Initiation Quarter	Q3 2025
Number of Interested Parties Engaged Post-Outreach	17

The relationship strategy was definitely about proving the science to the right specialists, and it worked. If onboarding takes 14+ days, churn risk rises, but here the transition is to a global giant. Finance: draft the post-merger integration budget for the Novartis renal team by Friday.

Regulus Therapeutics Inc. (RGLS) - Canvas Business Model: Channels

The Channels component of the Regulus Therapeutics Inc. (RGLS) business model, particularly as it transitions under the anticipated Novartis acquisition in late 2025, centers on clinical execution, regulatory navigation, and leveraging a future global commercial infrastructure.

Global clinical trial sites for Phase 3 patient enrollment

The primary channel for late-stage clinical validation is the planned Phase 3 pivotal trial for farabursen (RGLS8429) in Autosomal Dominant Polycystic Kidney Disease (ADPKD). Regulus Therapeutics Inc. confirmed alignment with the U.S. Food and Drug Administration (FDA) to proceed with a single pivotal study, which is set to initiate in the third quarter of 2025.

This pivotal trial is designed to enroll an estimated 336 adults to support both accelerated and full approval pathways. While specific global site counts for the Phase 3 trial are not yet public, the preceding Phase 1b study (NCT05521191) utilized multiple sites across the United States, including locations in:

• Arizona (Mesa)
• California (Los Angeles)
• Connecticut (New Haven)
• Florida (Jacksonville, Orlando)
• Georgia (Augusta)
• Idaho (Boise, Chubbuck, Idaho Falls)
• Illinois (Chicago)
• Iowa (Iowa City)
• Kansas (Kansas City, Wichita)
• Maryland (Baltimore)

The Phase 1b study structure involved cohorts with approximately 12 subjects each in the randomized portion and up to 30 subjects in the open-label fixed-dose cohort.

Regulatory bodies like the U.S. FDA and European Medicines Agency (EMA)

Regulatory engagement serves as a critical gatekeeping channel for market access. Regulus Therapeutics Inc. achieved significant alignment with the U.S. FDA following an End-of-Phase 1 meeting in December 2024. This alignment confirmed the acceptability of the Chemistry, Manufacturing, and Controls (CMC), non-clinical, and clinical pharmacology plans for the Phase 3 trial.

The agreed-upon Phase 3 design incorporates two primary endpoints to facilitate a streamlined review process:

Endpoint	Target Approval Pathway	Duration
Height-Adjusted Total Kidney Volume (htTKV) Change	Accelerated Approval	12-month
Estimated Glomerular Filtration Rate (eGFR) Change	Full Approval	24-month

The company is pursuing this accelerated pathway, which is a key strategic channel for faster patient access.

Novartis's global pharmaceutical distribution and sales network (future)

The most significant channel shift is the planned acquisition by Novartis AG, expected to close in the second half of 2025. This transaction immediately integrates farabursen into Novartis's established global development and commercial capabilities, which is vital for a drug targeting a condition with an estimated global prevalence of 4 to 7 million people.

The financial structure of this channel integration is substantial:

Consideration Type	Amount Per Share	Total Potential Equity Value
Upfront Cash Payment	$7.00	Upfront cash consideration is $0.8 billion
Contingent Value Right (CVR)	Up to $7.00 (upon regulatory milestone)	Total up to $1.7 billion

This acquisition transforms the distribution channel from a pre-commercial, small-cap biotech model to a large, established global pharmaceutical network.

Scientific and medical conferences for data dissemination

Dissemination of clinical data through scientific and medical conferences is a crucial channel for establishing credibility with the medical community and investors. Regulus Therapeutics Inc. actively utilized this channel throughout late 2024 and early 2025 to communicate positive Phase 1b results.

Key conference participation events included:

• Evercore 7th Annual HealthCONx Conference (December 3, 2024)
• Piper Sandler 36th Annual Healthcare Conference (December 4, 2024)
• Leerink Partners 2025 Global Healthcare Conference (March 4, 2025)
• Oppenheimer 35th Annual Healthcare Life Sciences Conference (February 4, 2025)

These presentations served to validate the mechanism of action, including the impact on urinary polycystins PC1 and PC2, and the halting of height-adjusted total kidney volume (htTKV) growth rate.

Regulus Therapeutics Inc. (RGLS) - Canvas Business Model: Customer Segments

As of late 2025, Regulus Therapeutics Inc. operates as an indirect wholly owned subsidiary of Novartis AG, following the completion of the acquisition in the second half of 2025.

Adult patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD)

• ADPKD is the most common genetic cause of renal failure worldwide.
• Worldwide prevalence is estimated to range from 1:400 to 1:1,000 individuals.
• Approximately 500,000 people in the United States suffer from Polycystic Kidney Disease (PKD).
• ADPKD accounts for approximately 90% of all PKD cases in the US.
• ADPKD is responsible for almost 10% of all end-stage renal disease (ESKD) cases.
• The US demand for ADPKD treatment was expected to start at USD 0.6 billion in 2025.
• The lead asset, farabursen, was on track for initiation of a Phase 3 single pivotal trial in the third quarter of 2025.

Nephrologists and kidney specialists who treat ADPKD

This segment includes the physicians responsible for diagnosing, managing, and prescribing therapies for ADPKD patients, a population that is distinct in its diagnostic criteria and prognostic markers.

• There were 5,187 businesses in the Nephrologists industry in the United States as of 2025.
• 35,818 people were employed in the Nephrologists in the US as of 2025.
• The market size for the Nephrologists industry in the United States was $9.7bn in 2025.
• An estimated 10,370 to 12,939 nephrologists currently practice in the United States.
• It is believed that each clinical nephrology practice has at least 1 family with ADTKD (Autosomal Dominant Tubulointerstitial Kidney Disease), suggesting high local need for expertise in monogenic kidney diseases.

The following table summarizes key market and workforce statistics relevant to the clinical customer base as of 2025:

Metric	Value/Amount	Context
US ADPKD Treatment Market Demand (Start of 2025)	USD 0.6 billion	US Demand for ADPKD Treatment in 2025.
US ADPKD Treatment Market Demand (Forecast 2035)	USD 1.0 billion	US Demand for ADPKD Treatment by 2035.
US Nephrology Businesses (2025)	5,187	Number of businesses in the Nephrologists industry in the US.
US Employed Nephrologists (2025)	35,818	Number of people employed in the Nephrologists in the US.
ADPKD Treatment Market CAGR (2025-2035)	5.16%	Compound Annual Growth Rate for the Global ADPKD Treatment Market.
ADPKD Kidney Failure Treatment Market Share (USA)	38%	Share of the US ADPKD treatment market held by kidney failure treatments.

Payers and government health systems covering specialty pharmaceuticals

This segment includes commercial insurers and government programs that determine coverage, pricing, and reimbursement for high-cost, specialty pharmaceuticals like farabursen, should it be approved.

• Nephrologists depend on reimbursements from private insurers more than any other revenue source.
• Commercial insurers pay higher prices to nephrologists for patient care than public insurers.
• Government initiatives, such as the Accelerating Medicines Partnership (AMP) initiative, fuel market growth with public-private partnership development.
• The ADPKD treatment market is moving towards innovation and accessibility between 2025 to 2035.

The parent company, Novartis, as the immediate internal customer

Since the acquisition, Regulus Therapeutics Inc. functions as an internal asset within the larger Novartis structure, making Novartis the primary entity driving the final development, regulatory strategy, and commercialization decisions for farabursen.

• Novartis completed the acquisition of Regulus Therapeutics on June 25, 2025.
• The upfront cash payment for the acquisition was $7.00 per share, totaling $0.8 billion.
• Total potential consideration, contingent upon a regulatory milestone, was approximately $1.7 billion.
• The contingent value right (CVR) offered an additional $7.00 per share in cash upon regulatory approval of farabursen.
• The transaction was unanimously approved by the Boards of Directors of both companies.

Regulus Therapeutics Inc. (RGLS) - Canvas Business Model: Cost Structure

You're looking at the expense side of the Regulus Therapeutics Inc. ledger as of late 2025, right before the Novartis acquisition was expected to close in the second half of the year. For a clinical-stage biopharma, the cost structure is dominated by one thing: getting that lead asset across the finish line.

The most significant drain on cash flow, as you'd expect, is the science itself. Research and Development (R&D) is the engine here, and it runs hot. For the first quarter ended March 31, 2025, R&D expenses hit $6.8 million. This figure covers everything from lab work to the massive undertaking of clinical studies.

Speaking of clinical studies, the costs associated with RGLS8429, which is farabursen for Autosomal Dominant Polycystic Kidney Disease (ADPKD), are front and center. The company was on track to initiate the single pivotal Phase 3 trial in the third quarter of 2025. While the specific Phase 3 expense isn't isolated in the Q1 report, this upcoming trial represents the single largest expected cost driver moving forward, dwarfing prior Phase 1b spending.

Beyond the lab and the clinic, you have the necessary corporate overhead. General and Administrative (G&A) expenses for the same three-month period in Q1 2025 totaled $3.7 million. This covers the operational costs to keep the lights on, including personnel, legal, and finance functions. Honestly, for a company of this size, that overhead is what keeps the core team functioning.

Here's a quick look at the key spending categories from the most recent reported quarter:

Cost Category	Amount (Q1 2025)	Context
Research and Development (R&D) Expenses	$6.8 million	Three months ended March 31, 2025
General and Administrative (G&A) Expenses	$3.7 million	Three months ended March 31, 2025
Net Loss	$9.63 million	Three months ended March 31, 2025
Cash, Cash Equivalents, and Short-Term Investments	$65.4 million	As of March 31, 2025

The maintenance and defense of the microRNA IP portfolio is an ongoing, though less granularly reported, cost. This is baked into the R&D and G&A figures, representing the legal and administrative expense required to secure and protect the oligonucleotide technology platform. You have to pay to play in this space, defintely.

The operational structure supporting these costs included a team of 34 employees as of a recent profile date. The burn rate implied by the Q1 2025 net loss of $9.63 million, set against the cash position of $65.4 million as of March 31, 2025, suggested a cash runway extending into early 2026, prior to the expected merger close.

The costs associated with advancing farabursen are further broken down by the clinical stage:

• Internal and external costs for advancing the pipeline.
• Costs associated with the planned pivotal Phase 3 trial initiation in Q3 2025.
• Costs from the recently completed Phase 1b Multiple-Ascending Dose (MAD) study.

Regulus Therapeutics Inc. (RGLS) - Canvas Business Model: Revenue Streams

You're looking at the revenue structure for Regulus Therapeutics Inc. after the June 2025 acquisition by Novartis AG. The primary revenue realization event has already occurred, shifting the focus from traditional product sales to transaction-based payments.

Acquisition-Related Cash Realization

The most immediate and realized revenue stream for former Regulus Therapeutics Inc. shareholders stems from the tender offer and subsequent merger with Novartis, which closed on June 25, 2025. Each existing Regulus Therapeutics Inc. Common Share was converted into the right to receive a specific cash amount plus a CVR.

• Realized upfront cash payment per share: $7.00 cash.
• Total upfront consideration value at closing: approximately $0.8 billion.
• The upfront cash represented a premium of 274 percent to the 60-day volume-weighted average stock price prior to the announcement.

Here's a quick look at the transaction economics as detailed in the merger agreement:

Component	Value Per Share	Total Potential Value	Status as of Late 2025
Upfront Cash Payment	$7.00	Approx. $0.8 billion	Realized upon closing on June 25, 2025
Contingent Value Right (CVR)	$7.00	Up to Approx. $0.9 billion additional	Potential future payment contingent on milestone
Total Potential Consideration	$14.00	Up to Approx. $1.7 billion	Contingent on CVR achievement

Contingent Value Right (CVR) Potential

The second key element of the revenue structure is the non-transferable Contingent Value Right (CVR). This represents a contingent revenue stream tied directly to the clinical and regulatory success of farabursen, Regulus Therapeutics Inc.'s lead asset for Autosomal Dominant Polycystic Kidney Disease (ADPKD).

• Potential future payment per share via CVR: $7.00 cash.
• The payment is contingent upon the achievement of a specified regulatory milestone for farabursen.
• The milestone is tied to regulatory approval, with some reports indicating a deadline of the end of 2034.

Independent Operating Revenue

As a clinical-stage subsidiary operating under Novartis following the June 2025 close, Regulus Therapeutics Inc. does not generate significant independent product sales revenue. Prior to the acquisition, the company's financial performance reflected this pre-commercial status. For the first quarter ended March 31, 2025, the company reported Research and Development (R&D) expenses of $6.8 million and a net loss of $9.6 million. Furthermore, prior financial trends indicated a consistent revenue downturn across five years at -100%.

Collaboration Milestones

While the Novartis deal dominates the current financial outlook, the historical business model included potential milestone payments from pre-existing collaborations. Any such payments remaining or realized post-acquisition would be minimal compared to the transaction consideration. The company's focus has narrowed to farabursen, which was the basis for the large Novartis deal structure.