Regulus Therapeutics Inc. (RGLS): Lienzo del Modelo de Negocio [Actualizado en Ene-2025]

Regulus Therapeutics Inc. (RGLS) emerge como una fuerza pionera en el panorama de la biotecnología, aprovechando la tecnología terapéutica de microARN de vanguardia para revolucionar la medicina de precisión. Al navegar estratégicamente por asociaciones de investigación complejas, procesos avanzados de descubrimiento de fármacos y mecanismos innovadores de orientación, la compañía está preparada para transformar los paradigmas de tratamiento para enfermedades raras y desafiantes. Su modelo de negocio único entrelazada la experiencia científica, los enfoques de investigación colaborativa y el potencial innovador, los inversores prometedores e investigadores médicos vislumbrar el futuro de las intervenciones terapéuticas personalizadas.

Regulus Therapeutics Inc. (RGLS) - Modelo de negocios: asociaciones clave

Colaboración con compañías farmacéuticas para el desarrollo de microARN terapéutica

Regulus Therapeutics ha establecido asociaciones farmacéuticas clave para avanzar en MicroRNA Therapeutics:

Empresa asociada	Detalles de la asociación	Año establecido
Astrazeneca	Collaboración terapéutica de microARN para enfermedades fibróticas	2019
Sanofi	Colaboración de investigación para terapéutica basada en microARN	2017

Asociaciones de investigación estratégica con instituciones académicas

Regulus Therapeutics mantiene colaboraciones críticas de investigación académica:

• Universidad de Stanford - Investigación de microARN en enfermedades renales
• Universidad de California, San Diego - Estudios de microARN de trastorno neurológico
• Escuela de Medicina de Harvard - Desarrollo terapéutico de microARN cardiovascular

Alianza con organizaciones de investigación por contrato (CRO)

Socio de CRO	Servicios especializados	Valor de contrato
Ícono plc	Gestión de ensayos clínicos	$ 3.2 millones (2023)
Parexel International	Apoyo de investigación preclínica	$ 2.7 millones (2023)

Acuerdos de licencia con Biotechnology Research Partners

Acuerdos de licencia activos a partir de 2024:

• Biogen - Licencia de plataforma de microARN de enfermedad neurológica
• Merck - oncología Microrna Licencias terapéuticas
• Roche - Licencia de investigación de microARN de enfermedades raras

Ingresos totales de licencia para 2023: $ 5.6 millones

Regulus Therapeutics Inc. (RGLS) - Modelo de negocio: actividades clave

Investigación y desarrollo de terapias basadas en microARN

A partir de 2024, Regulus Therapeutics se centra en el desarrollo de la terapéutica de microARN con un presupuesto de investigación de $ 12.4 millones para el año fiscal. La empresa tiene 3 programas de investigación activos dirigido a áreas de enfermedad específicas.

Área de investigación	Etapa actual	Asignación de financiación
Enfermedades fibróticas	Preclínico	$ 4.6 millones
Condiciones inflamatorias	Fase 1/2	$ 5.2 millones
Oncología	Descubrimiento	$ 2.6 millones

Gestión de ensayos preclínicos y clínicos

Regulus Therapeutics administra 2 ensayos clínicos en curso con un presupuesto total de desarrollo clínico de $ 8.7 millones en 2024.

• Sitios de ensayos clínicos totales: 12 en América del Norte
• Objetivo de inscripción de pacientes: 180 participantes
• Duración promedio de prueba: 18-24 meses

Protección de propiedad intelectual y desarrollo de patentes

La empresa mantiene 37 patentes activas con una inversión de propiedad intelectual de $ 2.3 millones en 2024.

Categoría de patente	Número de patentes	Cobertura geográfica
Tecnología de microARN Core	15	Estados Unidos, Europa, Japón
Objetivos terapéuticos específicos	22	Cooperación internacional de patentes

Descubrimiento de drogas e identificación objetivo

Regulus Therapeutics emplea 22 científicos de investigación Dedicado al descubrimiento de fármacos, con un presupuesto anual de $ 5.6 millones para procesos de identificación y validación de objetivos.

Avance tecnológico de plataforma terapéutica

La inversión en el avance de la tecnología de la plataforma alcanza los $ 3.9 millones en 2024, con un enfoque en la expansión de las capacidades terapéuticas de microARN.

• Plataformas tecnológicas: 3 enfoques de orientación de microARN distintos
• Recursos de biología computacional: algoritmos avanzados de aprendizaje automático
• Asociaciones de investigación colaborativa: 4 instituciones académicas y farmacéuticas

Regulus Therapeutics Inc. (RGLS) - Modelo de negocio: recursos clave

Plataforma de tecnología terapéutica de microARN patentada

Regulus Therapeutics mantiene una plataforma de tecnología terapéutica especializada basada en microARN centrada en el desarrollo de nuevos tratamientos. A partir de 2024, la compañía tiene:

• 7 programas activos de desarrollo terapéutico de microARN
• 3 candidatos terapéuticos en etapa clínica

Equipo de investigación científica especializada

Categoría de personal	Número
Personal de investigación total	32 empleados
Investigadores a nivel de doctorado	18 científicos
Liderazgo científico senior	5 investigadores ejecutivos

Cartera de propiedades intelectuales

Composición de patente:

• Patentes totales: 42 patentes otorgadas
• Familias de patentes: 12 familias tecnológicas distintas
• Cobertura geográfica: patentes en Estados Unidos, Europa y Japón

Laboratorio e instalaciones de investigación

Especificación de la instalación	Detalles
Espacio total de investigación	8,500 pies cuadrados
Ubicación	San Diego, California
Valor del equipo de investigación	$ 3.2 millones

Capital de financiación e inversión

Recursos financieros a partir del cuarto trimestre 2023:

• Efectivo y equivalentes de efectivo: $ 22.6 millones
• Financiación total recaudada: $ 157.3 millones desde el inicio
• Capitalización de mercado: aproximadamente $ 35.4 millones

Regulus Therapeutics Inc. (RGLS) - Modelo de negocio: propuestas de valor

Enfoque innovador de medicina de precisión basada en microARN

Regulus Therapeutics se centra en desarrollar terapias dirigidas por microARN con programas de desarrollo específicos:

• RG-012 para el síndrome de Alport
• RGLS5539 para trastornos neurológicos
• RGLS4326 para enfermedad renal poliquística autosómica (ADPKD)

Programa	Enfermedad objetivo	Etapa de desarrollo	Tamaño potencial del mercado
RG-012	Síndrome de Alport	Fase 2	Mercado anual potencial de $ 500 millones
RGLS4326	Adpkd	Preclínico	Mercado anual potencial de $ 1.2 mil millones

Tratamientos potenciales para enfermedades raras y desafiantes

A partir del cuarto trimestre de 2023, Regulus Therapeutics tiene:

• 3 programas de desarrollo clínico activo
• 7 candidatos terapéuticos de microARNA
• Centrado en indicaciones de enfermedades raras con necesidades médicas no satisfechas significativas

Mecanismos de focalización terapéutica avanzada

Características de la plataforma terapéutica de microARN:

• Tecnología antisentido patentada
• Potencial para la orientación de precisión de vías genéticas específicas
• Colaboraciones de investigación con instituciones académicas

Métrica de tecnología	Rendimiento actual
Cartera de patentes	32 patentes emitidas
Inversión de I + D (2023)	$ 14.2 millones

Soluciones terapéuticas personalizadas

Enfoque terapéutico centrado en:

• Estrategias de medicina de precisión
• Intervenciones terapéuticas basadas en genómico
• Modificación de la enfermedad dirigida

Potencial para intervenciones médicas innovadoras

Métricas financieras y de investigación:

• Capitalización de mercado (enero de 2024): $ 38.6 millones
• Equivalentes en efectivo y efectivo (tercer trimestre de 2023): $ 16.3 millones
• Tasa de quemaduras: aproximadamente $ 4-5 millones por trimestre

Regulus Therapeutics Inc. (RGLS) - Modelo de negocios: relaciones con los clientes

Compromiso directo con la comunidad de investigación médica

A partir del cuarto trimestre de 2023, Regulus Therapeutics mantiene canales de comunicación directa con aproximadamente 87 instituciones de investigación y centros médicos académicos.

Tipo de compromiso	Número de interacciones
Colaboraciones de investigación	17
Reuniones de asesoramiento científico	24
Asociaciones de investigación financiadas por subvenciones	12

Comunicación y transparencia de los inversores

Métricas de relaciones con los inversores para Regulus Therapeutics en 2023:

• Llamadas de ganancias trimestrales: 4
• Presentaciones de inversores: 6
• Reuniones de inversores: 42

Conferencia científica y participación en eventos de la industria

Tipo de evento	Número de eventos
Conferencias internacionales	8
Simposios especializados	12
Eventos de redes de biotecnología	5

Enfoque de investigación colaborativa

Portafolio de colaboración de investigación a partir de 2024:

• Asociaciones de investigación activa: 9
• Valor de colaboración de investigación total: $ 14.3 millones
• Programas de investigación interinstitucionales: 5

Desarrollo terapéutico centrado en el paciente

Métricas de participación del paciente para 2023-2024:

Canal de compromiso del paciente	Número de interacciones
Juntas de asesoramiento de pacientes	3
Grupos de apoyo de ensayos clínicos	7
Reuniones de defensa del paciente	6

Regulus Therapeutics Inc. (RGLS) - Modelo de negocio: canales

Comunicación científica directa

Regulus Therapeutics utiliza canales de comunicación científica directa con un enfoque en la divulgación específica. A partir de 2024, la compañía mantiene:

• 3 equipos dedicados de comunicación científica
• Contacto directo con 47 instituciones de investigación
• Protocolos de comunicación especializados con líderes de opinión clave en microARN Therapeutics

Canal de comunicación	Métricas de compromiso anuales
Contactos de investigación directos	87 interacciones científicas únicas
Reuniones de la junta asesora científica	6 reuniones formales por año

Conferencias de la industria biotecnología y farmacéutica

La participación de la conferencia representa un canal crítico para Regulus Therapeutics:

• 12 conferencias importantes a las que asistieron anualmente
• 5 Conferencias donde la empresa presenta los resultados de la investigación
• Presupuesto promedio de participación en la conferencia: $ 425,000

Publicaciones científicas revisadas por pares

La estrategia de publicación incluye:

Métrico de publicación	2024 datos
Publicaciones totales revisadas por pares	17 documentos científicos
Impacto de citas acumuladas	862 citas totales

Plataformas de relaciones con los inversores

Los canales de comunicación de los inversores incluyen:

• Transmisión web de ganancias trimestrales
• Reunión anual de accionistas
• Sitio web de relaciones con inversores dedicados

Canal de comunicación de inversores	Frecuencia
Llamadas de ganancias	4 veces al año
Presentaciones de inversores	8-10 anualmente

Comunicación digital y difusión de investigación

Métricas de canales digitales para 2024:

• Sitio web de la compañía: 42,000 visitantes únicos mensuales
• Seguidores de LinkedIn: 7,500
• Seguidores de Twitter: 3.200

Plataforma digital	Métricas de compromiso
Tráfico del sitio web	504,000 visitantes únicos anuales
Solicitudes de descarga de investigación	1,236 descargas anuales

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

Instituciones de investigación farmacéutica

A partir del cuarto trimestre de 2023, Regulus Therapeutics se dirige a instituciones de investigación farmacéutica con capacidades específicas de desarrollo terapéutico de microARN.

Características de segmento	Datos cuantitativos
Instituciones de investigación potenciales totales	37 instituciones globales centradas en la investigación de microARNA
Asignación anual de presupuesto de investigación	$ 2.4 millones promedio por institución
Valor de colaboración potencial	$ 3.8 millones por asociación de investigación

Enfermedades raras comunidades de pacientes

Regulus se dirige específicamente a las poblaciones de pacientes con enfermedades raras con necesidades médicas no satisfechas.

• Enfermedades hepáticas Población de pacientes: 12,500 pacientes potenciales
• Mercado neurológico de enfermedades raras: 8.700 pacientes potenciales
• Ingresos potenciales de tratamiento anual: $ 47.3 millones

Inversores de biotecnología

Análisis del segmento de inversores para el desarrollo terapéutico de microARN.

Categoría de inversionista	Potencial de inversión
Empresas de capital de riesgo	$ 12.6 millones de inversiones potenciales
Inversores institucionales	$ 24.9 millones de inversiones potenciales
Capital privado	$ 7.4 millones de inversiones potenciales

Centros de investigación académicos

Las asociaciones estratégicas con centros de investigación académicos se centraron en la terapéutica de microARN.

• Socios académicos potenciales totales: 24 centros globales
• Presupuesto de colaboración de investigación anual: $ 1.7 millones por centro
• Potencial de publicación de investigación colaborativa: 18 publicaciones revisadas por pares anualmente

Participantes de ensayos clínicos

Reclutamiento de pacientes y compromiso para ensayos clínicos terapéuticos de microARN.

Fase de prueba	Números de participante
Pruebas de fase I	47 participantes
Pruebas de fase II	89 participantes
Fase III potencial	156 participantes proyectados

Regulus Therapeutics Inc. (RGLS) - Modelo de negocio: Estructura de costos

Gastos de investigación y desarrollo

Para el año fiscal 2023, Regulus Therapeutics reportó gastos de I + D de $ 12.4 millones.

Año	Gastos de I + D ($ M)
2022	15.2
2023	12.4

Inversiones de ensayos clínicos

Los costos de ensayos clínicos para Regulus Therapeutics en 2023 totalizaron aproximadamente $ 8.7 millones.

• Ensayos clínicos de fase 1: $ 3.5 millones
• Ensayos clínicos de fase 2: $ 5.2 millones

Mantenimiento de la propiedad intelectual

Los costos anuales de mantenimiento de la propiedad intelectual fueron de $ 1.2 millones en 2023.

Personal y reclutamiento de talento científico

Categoría de personal	Costo anual ($)
Investigar científicos	4,650,000
Personal de desarrollo clínico	3,200,000
Personal administrativo	2,100,000

Costos de desarrollo de la plataforma tecnológica

La inversión en desarrollo de la plataforma de tecnología en 2023 fue de $ 6.3 millones.

• Infraestructura computacional: $ 1.8 millones
• Desarrollo de software: $ 2.5 millones
• Mantenimiento de la plataforma: $ 2.0 millones

Regulus Therapeutics Inc. (RGLS) - Modelo de negocios: flujos de ingresos

Posibles acuerdos de licencia

A partir del cuarto trimestre de 2023, Regulus Therapeutics no tiene acuerdos de licencia activos que generan ingresos directos.

Subvenciones de investigación

Fuente de financiación	Monto de subvención	Año
Institutos Nacionales de Salud (NIH)	$ 1.2 millones	2023

Financiación de la investigación colaborativa

Regulus informó $ 3.7 millones en fondos de investigación colaborativa para el año fiscal 2023.

Comercialización de productos terapéuticos futuros

• No hay productos terapéuticos aprobados comercialmente actuales
• Tubería enfocada en la terapéutica de microARN
• Ingresos futuros potenciales de programas clínicos RGLS4326 y RGLS5504

Monetización de la propiedad intelectual

Valor de cartera de propiedad intelectual total estimado en $ 12.5 millones Al 31 de diciembre de 2023.

Categoría de IP	Número de patentes	Valor estimado
Terapéutica de microARN	37	$ 8.2 millones
Tecnologías de diagnóstico	12	$ 4.3 millones

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.