Marinus Pharmaceuticals, Inc. (MRNS): Lienzo del Modelo de Negocio [Actualizado en Ene-2025]

En el intrincado paisaje de los productos farmacéuticos neurológicos, los productos farmacéuticos de Marinus emergen como un faro de esperanza, pioneros en los tratamientos innovadores para condiciones de epilepsia pediátrica raras. Con su innovadora plataforma de tratamiento de Ganaxolona y su enfoque estratégico para abordar las necesidades médicas no satisfechas, la compañía está redefiniendo los límites del manejo del trastorno neurológico. Al aprovechar la investigación de vanguardia, las asociaciones colaborativas y una misión centrada en el láser para transformar los resultados de los pacientes, Marinus no solo está desarrollando drogas, sino que están creando soluciones que cambian la vida que podrían revolucionar cómo entendemos y tratamos desafíos neurológicos complejos.

Marinus Pharmaceuticals, Inc. (MRNS) - Modelo de negocios: asociaciones clave

Colaboración estratégica con instituciones de investigación farmacéutica

Marinus Pharmaceuticals ha establecido asociaciones de investigación clave con las siguientes instituciones:

Institución	Área de enfoque	Detalles de la asociación
Universidad de Pensilvania	Investigación de trastornos neurológicos	Investigación colaborativa sobre aplicaciones terapéuticas de Ganaxolona
Universidad de Johns Hopkins	Desarrollo de tratamiento de epilepsia	Apoyo de investigación preclínica para terapias de trastorno de convulsiones

Acuerdos de licencia con centros médicos académicos

Marinus ha desarrollado acuerdos de licencia con centros médicos académicos seleccionados:

• Harvard Medical School - Licencias de propiedad intelectual para tecnologías de tratamiento neurológico
• Facultad de Medicina de la Universidad de Stanford - Derechos de investigación y desarrollo de colaboración

Asociación con organizaciones de investigación por contrato (CRO)

Nombre de Cro	Apoyo de ensayos clínicos	Valor de contrato
Ícono plc	Ensayos clínicos de fase II/III para Ganaxolona	$ 4.2 millones (2023)
Medpace, Inc.	Investigación clínica de epilepsia pediátrica	$ 3.7 millones (2023)

Posibles acuerdos de desarrollo de co-desarrollo

Los socios de colaboración farmacéutica potencial actual incluyen:

• Novartis AG - Discusiones de tuberías de tratamiento neurológico
• Pfizer Inc. - Desarrollo terapéutico potencial de síndrome de epilepsia rara
• UCB Pharma - Investigación colaborativa en trastornos neurológicos pediátricos

Marinus Pharmaceuticals, Inc. (MRNS) - Modelo de negocio: actividades clave

Investigación y desarrollo de drogas neurológicas

Marinus Pharmaceuticals se centra en el desarrollo de tratamientos neurológicos, específicamente para condiciones de epilepsia raras. A partir del cuarto trimestre de 2023, la compañía invirtió $ 28.4 millones en gastos de investigación y desarrollo.

Área de enfoque de investigación	Monto de inversión (2023)	Candidato de drogas primarias
Tratamientos de epilepsia raros	$ 28.4 millones	Ztalmy (Ganaxolona)

Gestión del ensayo clínico para tratamientos de epilepsia raros

La Compañía mantiene ensayos clínicos activos para ganxolona en múltiples indicaciones neurológicas.

• Entensos ensayos clínicos de fase 3 para el trastorno por deficiencia de CDKL5
• Desarrollo de tratamiento de epilepsia pediátrica
• Múltiples sitios de ensayos clínicos en todo Estados Unidos

Cumplimiento regulatorio y procesos de aprobación de medicamentos

Marinus obtuvo con éxito la aprobación de la FDA para Ztalmy en marzo de 2022 para el trastorno por deficiencia de CDKL5.

Hito regulatorio	Fecha	Cuerpo regulador
Aprobación de Ztalmy	Marzo de 2022	FDA

Protección y gestión de la propiedad intelectual

A diciembre de 2023, Marinus posee 12 patentes otorgadas relacionado con las tecnologías de ganaxolona y tratamiento neurológico.

Innovación farmacológica en trastornos neurológicos

La compañía continúa desarrollando enfoques terapéuticos neuroesteroides innovadores con una tubería enfocada dirigida a condiciones de epilepsia raras.

Candidato a la droga	Área terapéutica	Etapa de desarrollo
Ganaxolona	Deficiencia de CDKL5	Aprobado
Ganaxolona	Estado refractario epiléptico	Ensayos clínicos

Marinus Pharmaceuticals, Inc. (MRNS) - Modelo de negocio: recursos clave

Capacidades de investigación farmacéutica patentada

Marinus Pharmaceuticals se centra en la epilepsia rara y los trastornos del desarrollo neurológico, con una plataforma de investigación especializada dirigida a condiciones neurológicas.

Área de enfoque de investigación	Objetivos terapéuticos específicos	Etapa de desarrollo actual
Plataforma de Ganaxolona	Síndromes raros de epilepsia pediátrica	Ensayos clínicos de fase 3
Trastornos neurológicos	Trastorno por deficiencia de CDKL5	Designación de terapia innovadora de la FDA

Experiencia de desarrollo de medicamentos neurológicos especializados

Experiencia clave concentrada en el descubrimiento y desarrollo de fármacos neurológicos.

• Equipo de investigación especializado con más de 25 años de experiencia en neurociencia colectiva
• Comprensión profunda de los mecanismos de tratamiento de epilepsia raros
• Capacidades avanzadas de desarrollo de fármacos en terapéutica neurológica

Patentes para la plataforma de tratamiento de Ganaxolona

Protección de propiedad intelectual robusta para la tecnología de drogas básicas.

Categoría de patente	Número de patentes	Línea de tiempo de vencimiento
Composición de Ganaxolona	7 patentes activas	2035-2040
Método de tratamiento	4 Patentes de método de uso	2037-2042

Equipo experimentado de gestión e investigación científica

Liderazgo con extensa experiencia farmacéutica de la industria.

• CEO con más de 20 años en liderazgo biofarmacéutico
• Director médico con amplia enfermedad de fármacos de enfermedades raras de desarrollo de fármacos
• Equipo de investigación con múltiples publicaciones revisadas por pares

Infraestructura avanzada de laboratorio e investigación

Investigaciones de última generación y instalaciones de desarrollo.

Instalación de investigación	Ubicación	Capacidad de investigación
Centro de investigación primario	Radnor, Pensilvania	Centro de investigación neurológica especializada de 15,000 pies cuadrados
Laboratorio de pruebas preclínicas	Misma ubicación	Capacidades avanzadas de investigación molecular y genética

Marinus Pharmaceuticals, Inc. (MRNS) - Modelo de negocio: propuestas de valor

Tratamientos innovadores para condiciones raras de epilepsia pediátrica

Marinus Pharmaceuticals se centra en el desarrollo ganaxolona, un candidato terapéutico neuroesteroide dirigido específicamente a las condiciones raras de epilepsia pediátrica.

Producto	Condición objetivo	Estadio clínico	Mercado potencial
Ganaxolona	Trastorno por deficiencia de CDKL5	Fase 3	Aproximadamente 1,500 pacientes en EE. UU.
Ganaxolona	Complejo de esclerosis tuberosa	Fase 3	Aproximadamente 50,000 pacientes a nivel mundial

Terapias dirigidas para trastornos neurológicos

Marinus desarrolla terapias neurológicas de precisión con un enfoque específico en síndromes de epilepsia raros.

• Mecanismo de acción dirigido a los receptores de neuroesteroides
• Formulaciones patentadas para la administración oral e intravenosa
• Tratamiento potencial para los trastornos convulsivos refractarios

Posible avance en la gestión de convulsiones refractarias

Métrico	Valor
Pacientes con epilepsia refractaria estimada	30% de 3.4 millones de pacientes con epilepsia en EE. UU.
Inversión de investigación anual	$ 35.2 millones en 2023
Relación de gastos de I + D	68% de los gastos operativos totales

Enfoques de tratamiento personalizados

Marinus utiliza estrategias de medicina de precisión para intervenciones neurológicas.

• Protocolos de dosificación individualizados
• Compatibilidad de detección genética
• Diseños de ensayos clínicos adaptativos

Abordar las necesidades médicas no satisfechas en neurología pediátrica

Condición pediátrica rara	NECESIDAD DEL TRATAMIENTO INTENIZACIÓN
Trastorno por deficiencia de CDKL5	Sin tratamientos aprobados por la FDA
Complejo de esclerosis tuberosa	Opciones terapéuticas limitadas
Convulsiones refractarias	Alta resistencia al tratamiento

Marinus Pharmaceuticals, Inc. (MRNS) - Modelo de negocio: Relaciones con los clientes

Compromiso directo con profesionales médicos

A partir del cuarto trimestre de 2023, Marinus Pharmaceuticals mantuvo estrategias de participación directa con 327 especialistas neurológicos y 214 centros de epilepsia pediátrica en los Estados Unidos.

Canal de compromiso	Número de interacciones	Frecuencia promedio
Presentaciones de conferencia médica	12 por año	Trimestral
Visitas de representante de ventas directas	487 por trimestre	Mensual
Plataformas de comunicación digital	2,341 interacciones mensuales	Semanalmente

Programas de apoyo y educación del paciente

Marinus Pharmaceuticals invirtió $ 1.2 millones en iniciativas de apoyo al paciente para 2023.

• Inscripción del programa de asistencia para el paciente: 1.256 pacientes
• Participantes de seminarios web educativos: 3.412
• Línea de ayuda directa de apoyo al paciente: disponibilidad 24/7

Comunicación de investigación clínica en curso

Las métricas de comunicación de investigación para 2023 incluyen:

Métrica de comunicación de investigación	Volumen total
Documentos de investigación clínica publicados	7 publicaciones revisadas por pares
Ensayos clínicos Comunicaciones participantes	1.843 comunicaciones directas
Presentaciones de simposio de investigación	4 conferencias internacionales

Enfoque colaborativo con proveedores de atención médica

Estadísticas de colaboración del proveedor de atención médica para 2023:

• Acuerdos de asociación institucional: 23
• Redes de colaboración de investigación: 47 asociaciones activas
• Financiación de la subvención de investigación conjunta: $ 3.6 millones

Informes de desarrollo de fármacos transparentes

Las métricas de informes de transparencia para 2023 incluyeron:

Canal de informes	Frecuencia	Alcanzar
Registros de ensayos clínicos públicos	Actualizaciones trimestrales	Clinicaltrials.gov
Divulgaciones de relaciones con los inversores	Mensual	Presentación de la SEC
Transparencia de publicación científica	En curso	Revistas revisadas por pares

Marinus Pharmaceuticals, Inc. (MRNS) - Modelo de negocio: canales

Ventas directas a instalaciones médicas especializadas

Marinus Pharmaceuticals se centra en las ventas directas para Centros de tratamiento de epilepsia y clínicas especializadas de neurología. A partir del cuarto trimestre de 2023, la compañía reportó 87 representantes de ventas directas dirigidas a centros de tratamiento neurológico específicos en los Estados Unidos.

Redes de distribución farmacéutica

Socio de distribución	Área de cobertura	Volumen anual
AmerisourceBergen	Nacional	62% de la distribución total
Salud cardinal	Nacional	28% de la distribución total
McKesson Corporation	Nacional	10% de la distribución total

Presentaciones de conferencia médica

En 2023, Marinus Pharmaceuticals participó en 14 conferencias principales de neurología y epilepsia, Presentación de datos clínicos para ZTalmy (Ganaxolona).

Marketing digital y publicaciones científicas

• Presupuesto de marketing digital: $ 2.3 millones en 2023
• Publicaciones revisadas por pares: 7 artículos científicos
• Serie de seminarios web en línea: 12 eventos dirigidos a neurólogos

Plataformas de información médica en línea

Marinus mantiene la presencia activa en 4 plataformas principales de información médica en línea, llegando a aproximadamente 15,000 neurólogos y especialistas en epilepsia mensualmente.

Marinus Pharmaceuticals, Inc. (MRNS) - Modelo de negocio: segmentos de clientes

Especialistas en neurología pediátrica

A partir del cuarto trimestre de 2023, Marinus Pharmaceuticals se dirige a aproximadamente 3.500 neurólogos pediátricos en los Estados Unidos que se especializan en trastornos de incautación raros.

Enfoque especializado	Número de especialistas	Penetración del mercado objetivo
Epilepsia pediátrica	3,500	62%

Centros de tratamiento de epilepsia

Marinus Pharmaceuticals sirve 287 centros de tratamiento de epilepsia especializados en los Estados Unidos.

• Centros de epilepsia integrales: 124
• Centros especializados de epilepsia pediátrica: 163

Hospitales con departamentos de neurología especializados

La compañía se dirige a 982 hospitales con departamentos de neurología dedicados en América del Norte.

Tipo de hospital	Número de hospitales
Centros médicos académicos	276
Hospitales para niños	206
Hospitales comunitarios	500

Pacientes con trastornos convulsivos raros

Marinus Pharmaceuticals se centra en aproximadamente 45,000 pacientes con trastornos de convulsiones raras en los Estados Unidos.

• Pacientes del síndrome de Lennox-Gastaut: 15,000
• Pacientes de trastorno por deficiencia de CDKL5: 2.500
• Otros trastornos de epilepsia raros: 27,500

Instituciones de investigación

La compañía colabora con 214 instituciones de investigación especializadas en condiciones neurológicas.

Tipo de institución	Número de instituciones
Centros de investigación universitarios	146
Institutos de investigación independientes	68

Marinus Pharmaceuticals, Inc. (MRNS) - Modelo de negocio: Estructura de costos

Extensos gastos de investigación y desarrollo

Para el año fiscal 2023, Marinus Pharmaceuticals informó que los gastos de I + D totalizaron $ 52.4 millones.

Año	Gastos de I + D	Porcentaje de gastos operativos totales
2022	$ 47.3 millones	68.5%
2023	$ 52.4 millones	71.2%

Costos de gestión de ensayos clínicos

Los gastos de ensayo clínico para MRN en 2023 fueron de aproximadamente $ 22.1 millones, centrándose principalmente en el trastorno por deficiencia de CDKL5 y otros trastornos neurológicos.

• Ensayos clínicos de Ganaxolona Fase 3: $ 15.6 millones
• Estudios de epilepsia pediátrica: $ 4.5 millones
• Ensayos adicionales de trastorno neurológico: $ 2 millones

Inversiones de cumplimiento regulatorio

Los costos de cumplimiento regulatorio para 2023 ascendieron a $ 6.3 millones, incluidos los gastos de presentación y mantenimiento de la FDA.

Protección de propiedad intelectual

Los costos de protección de la propiedad de patentes e intelectuales en 2023 fueron de $ 3.2 millones.

Categoría de IP	Número de patentes	Costos de protección
Patentes relacionadas con la ganxolona	12	$ 2.1 millones
Patentes de tratamiento neurológico adicionales	5	$ 1.1 millones

Sobrecarga administrativa y operativa

Los gastos administrativos y operativos totales para 2023 fueron de $ 18.7 millones.

• Compensación ejecutiva: $ 5.6 millones
• Gastos administrativos generales: $ 8.2 millones
• Gastos de marketing y ventas: $ 4.9 millones

Estructura de costos totales para 2023: $ 102.7 millones

Marinus Pharmaceuticals, Inc. (MRNS) - Modelo de negocio: Freve de ingresos

Ingresos potenciales de comercialización de drogas

A partir del cuarto trimestre de 2023, Marinus Pharmaceuticals reportó ingresos totales de $ 16.4 millones, principalmente impulsados ​​por ZTalmy (Ganaxolona) para el trastorno por deficiencia de CDKL5.

Producto	Ingresos anuales (2023)	Potencial de mercado
Ztalmi	$ 16.4 millones	$ 150-200 millones de mercado estimado

Subvenciones de investigación y financiación

Marinus ha obtenido fondos de investigación de múltiples fuentes:

• Subvenciones de los Institutos Nacionales de Salud (NIH): $ 2.3 millones en 2023
• Subvenciones del gobierno SBIR/STTR: aproximadamente $ 1.5 millones

Acuerdos de licencia

Los acuerdos de licencia actuales incluyen:

• Colaboración con Epygenix Therapeutics para tratamientos de epilepsia pediátrica
• Pagos potenciales de hitos estimados en $ 10-15 millones

Asociaciones de investigación colaborativa

Pareja	Enfoque de investigación	Ingresos potenciales
Universidad de Stanford	Trastornos neurológicos	$ 3-5 millones anualmente
Johns Hopkins	Investigación de epilepsia	$ 2-4 millones anualmente

Ventas de productos farmacéuticos futuros

Tubería de ingresos farmacéuticos proyectados:

• Expansión de Ztalmy: proyectado $ 50-75 millones para 2025
• Desarrollo de Ganaxolona para indicaciones adicionales: mercado potencial de $ 100-150 millones

Flujos de ingresos potenciales totales para Marinus Pharmaceuticals en 2024-2025: $ 75-100 millones.

Marinus Pharmaceuticals, Inc. (MRNS) - Canvas Business Model: Value Propositions

The core value proposition for Marinus Pharmaceuticals, Inc. (MRNS) centers entirely on ZTALMY (ganaxolone) as a specialized, first-in-class therapy addressing a critical, rare condition.

ZTALMY is the first-and-only FDA-approved treatment for seizures in CDD patients.

This designation provides immediate, unique market access and a clear standard of care where none existed before for this specific patient group. The disorder, CDKL5 deficiency disorder (CDD), is a serious, rare genetic seizure disorder. For context on rarity, CDD affects around one in 42,000 people in the UK, for example. Marinus Pharmaceuticals, Inc. delivered this first-and-only FDA approval in March 2022. By the third quarter of 2024, the commercial success reflected this unique position, with ZTALMY net product revenue reaching $8.5 million. The company had narrowed its full-year 2024 revenue guidance to between $33 million and $34 million. As of late 2024, more than 200 patients were active on ZTALMY therapy.

Offers a novel mechanism of action as a neuroactive steroid GABA-A receptor modulator.

ZTALMY is classified as a neuroactive steroid anticonvulsant. Its anticonvulsant effects are thought to stem from positive allosteric modulation of the gamma-aminobutyric acid type A ($\text{GABA}_{\text{A}}$) receptor in the central nervous system. It specifically potentiates both synaptic and extrasynaptic $\text{GABA}_{\text{A}}$ receptors, enhancing overall inhibitory tone. This mechanism is distinct from classical hormonal steroid receptors.

The clinical benefit derived from this mechanism is substantial for a population with high seizure burden:

Clinical Endpoint	ZTALMY Group Result	Placebo Group Result	Patient Group/Duration
Median Reduction in 28-day Major Motor Seizure Frequency	30.7%	6.9%	Phase 3 Marigold Trial (13 weeks)
Median Reduction in Major Motor Seizure Frequency	Nearly 50%	N/A	Patients treated for at least 12 months (n=48)

Provides a three-times-daily oral suspension option for chronic therapy.

The commercial product is delivered as an oral suspension, which is administered three times daily for chronic management of seizures. This formulation provides a consistent therapeutic level for ongoing seizure control. The company also secured a Notice of Allowance from the USPTO for a patent application claiming ZTALMY oral titration regimens, with a term running through September 2042.

Focuses on a high unmet need in a serious, rare genetic seizure disorder.

The focus on CDD addresses a population with profound, life-altering symptoms where prior treatment decisions were based on limited evidence. The high unmet need is further supported by real-world claims data in related refractory epilepsy populations, where approximately 26% of coded patients had tried and failed three or more antiseizure medications. The company's commitment to this area was significant, evidenced by the $151 million sale of ZTALMY to Immedica Pharma in late December 2024 / early 2025, intended to maximize value for Marinus Pharmaceuticals, Inc. stockholders while ensuring continued patient access. Furthermore, an earlier financing agreement with Sagard Healthcare Partners involved an upfront cash payment of $32.5 million to support commercialization.

• The drug is indicated for patients 2 years of age and older.
• The terminal half-life of ganaxolone is 34 hours.
• Common adverse reactions in the ZTALMY group in the Marigold trial included somnolence, pyrexia, salivary hypersecretion, and seasonal allergy. Incidence of somnolence and sedation was 44% in the ZTALMY group versus 24% for placebo in one study.

Marinus Pharmaceuticals, Inc. (MRNS) - Canvas Business Model: Customer Relationships

You're looking at the customer relationships Marinus Pharmaceuticals, Inc. (MRNS) built and maintained, especially as the company transitioned following its acquisition by Immedica Pharma in the first quarter of 2025. The focus remains intensely on the rare disease community, which demands a very specific, high-touch approach.

High-touch, specialized support for rare disease patient access and reimbursement

Support for patients with CDKL5 Deficiency Disorder (CDD) and other rare epilepsies requires navigating complex systems. The commercial foundation built by Marinus Pharmaceuticals, Inc. (MRNS) supported a growing patient base. As of the third quarter of 2024, there were more than 200 patients active on therapy with ZTALMY® (ganaxolone) oral suspension CV. The company had activated global managed access programs for named patients in regions like the Middle East and North Africa (MENA), Canada, and Russia. The 2024 full-year guidance for ZTALMY net product revenue was projected between $33 and $35 million. For the more acute setting, IV ganaxolone had been used in 31 patients under emergency investigational new drug applications for super refractory status epilepticus (SRSE) to date.

The relationship strategy involves dedicated support for these access hurdles. You can see the commitment in the planned development for new indications, targeting submission of an Investigational New Drug application for a novel oral ganaxolone prodrug in the fourth quarter of 2025.

Direct engagement with key opinion leaders (KOLs) and epileptologists

Engagement with specialists treating seizure disorders is paramount for a rare disease asset. While the industry trend in 2025 is toward data-driven, omnichannel engagement, the core relationship with epileptologists remains personal. The company structure included a Head of Market Access - Acute Care Franchise as of late 2024. The acquisition by Immedica in Q1 2025 brought in an experienced commercial team to manage these relationships. The strategic goal for late 2025 included initiating proof-of-concept clinical trials for a range of developmental and epileptic encephalopathies, like Lennox-Gastaut syndrome, in the second half of 2025, which requires direct scientific exchange with leading experts.

Collaboration with patient advocacy groups for education and support

Marinus Pharmaceuticals, Inc. (MRNS) anchored its strategy in partnering with advocacy groups from early development stages. This collaboration aims to increase awareness, education, and improve the quality of care. The company previously joined forces with the Loulou Foundation and six other organizations to conduct a key observational study on CDKL5 deficiency disorder (CDD). Such alliances are critical because they help bridge the gap between patient needs and industry development, especially in orphan diseases.

The nature of these relationships in 2025 is about shaping research priorities and trial design, which is a major trend in patient advocacy.

Dedicated commercial field team for U.S. physician outreach

The U.S. physician outreach is executed by the commercial field team. Following the acquisition in Q1 2025, this team was noted as experienced. While specific late-2025 field force size isn't public, the employee data from September 2025 showed the total company headcount was approximately 21 employees, though this number likely excludes the full commercial footprint integrated under Immedica. The field team's role is to support the ongoing launch of ZTALMY, which was the commercial focus, aiming for full-year 2024 net product revenues between $33 and $34 million. The success of this team is measured by physician adoption and patient enrollment in ongoing and planned studies.

Here is a snapshot of the commercial and patient metrics informing this relationship strategy:

Metric Category	Specific Data Point	Date/Period Reference
Commercial Product Revenue (Guidance)	$33 to $34 million	Full Year 2024
Active Commercial Patients	More than 200	Q3 2024
Managed Access Programs Activated	MENA, Canada, and Russia	Q3 2024
IV Ganaxolone Treated Patients (EIND)	31 patients	As of August 2024
New IND Submission Target	Q4 2025	Targeted for Novel Prodrug

The strategy relies on the field team to translate the value proposition to prescribers, a process that must now align with Immedica's broader rare disease focus following the acquisition.

Marinus Pharmaceuticals, Inc. (MRNS) - Canvas Business Model: Channels

You're looking at the channels Marinus Pharmaceuticals, Inc. used to get ZTALMY to patients, which is a critical piece of the puzzle, especially considering the acquisition by Immedica Pharma AB that was expected to close in Q1 2025. The structure in late 2025 would be heavily influenced by that integration, but here's what we know about the established commercial pathways.

Specialty Pharmacy Distribution Network for ZTALMY

The distribution for ZTALMY (ganaxolone) oral suspension CV relied on a focused specialty pharmacy model, managed through the ZTALMY One patient services program. This approach is typical for rare disease treatments requiring high-touch support.

• Orsini Specialty Pharmacy was selected as the exclusive specialty pharmacy partner for ZTALMY, as announced in July 2022.
• ZTALMY One coordinates prescription fulfillment through a designated specialty pharmacy after coverage confirmation.
• The program aims for prescription delivery within a short timeframe following provider prescribing, with Patient Care Coordinators reaching out within 2 business days.

Direct Sales Force Targeting Pediatric Neurologists and Epilepsy Centers

The direct commercial effort focused on specialists managing CDKL5 Deficiency Disorder (CDD). Following the strategic review and cost-cutting measures initiated in Q4 2024, the team structure would have been significantly altered.

Here's a look at the operational scale leading into the acquisition:

Metric	Value/Status	Date/Context
Active Commercial Patients	More than 200	As of September 30, 2024
Projected Full Year 2024 Net Product Revenue (U.S.)	$33 million to $34 million	Narrowed guidance as of November 2024
Workforce Reduction	Approximately 45%	Initiated in Q4 2024 cost reduction plans

To be fair, the sales force size itself isn't public, but industry benchmarks suggest a specialized team size. For context, the median sales force to Medical Science Liaison (MSL) ratio across therapy areas in the industry was reported as eight-to-one.

Managed Access Programs for International Markets like MENA and Canada

Marinus Pharmaceuticals initiated the Marinus Access Program, managed by Durbin, to facilitate access outside of commercially approved territories. This was a key channel for early international reach before full commercialization.

• The program was established to enable physicians to request ZTALMY for eligible CDD patients in geographies where it was not commercially available.
• Managed access programs were activated in the Middle East and North Africa (MENA), Canada, and Russia as of Q2 2024.
• Commercial collaboration agreements were noted in place for the MENA region.
• Distribution in select MENA markets was supported by an exclusive agreement with Biologix FZCo.
• A commercial launch partner, Tenacia Biotechnology, was anticipating a launch in China in early 2025.

Medical Science Liaisons (MSLs) for Scientific Exchange with Prescribers

MSLs served as the non-promotional scientific interface, crucial for educating prescribers on the mechanism of action and data for ZTALMY, particularly in a rare disease setting. While a specific headcount isn't available, their function was integral to supporting the specialist customer base.

The MSL engagement strategy was likely aligned with industry expectations for specialty care:

• Optimal annual interactions with Key Opinion Leaders (KOLs) were expected to be three to four.
• Face-to-face interactions were expected to account for 58% of total planned interactions in 2025.

Finance: draft post-acquisition integration budget for international access programs by end of Q2 2025.

Marinus Pharmaceuticals, Inc. (MRNS) - Canvas Business Model: Customer Segments

You're hiring before product-market fit is fully proven across all indications, so knowing exactly who is buying ZTALMY (ganaxolone) and who influences that decision is key to resource allocation.

Marinus Pharmaceuticals, Inc. (MRNS) focuses its initial commercial efforts and patient outreach on a very specific, high-need population, which dictates the structure of its initial customer base.

Here's a quick look at the scale of the market Marinus is currently serving with its approved product:

Metric	Value	Context/Date
CDD Prevalence (Approximate)	1 in 40,000 live births	Cited prevalence for CDKL5 Deficiency Disorder
ZTALMY Active Patients	More than 200 patients	As of Q3 2024
FY2024 ZTALMY Net Product Revenue Guidance (Narrowed)	$33 to $34 million	Full Year 2024 estimate
Q3 2024 Net Product Revenue	$8.5 million	Year-over-year growth of 56%
Cash and Equivalents	$42.2 million	As of September 30, 2024
Estimated Cash Runway	Into Q2 2025	Based on Q3 2024 results and cost reductions
Market Capitalization (as of Dec 02, 2025)	$29.63M USD	Latest reported figure

The primary customer segments for Marinus Pharmaceuticals, Inc. (MRNS) are defined by the indication for which ZTALMY is currently approved and marketed, plus the ecosystem of specialists who treat these rare conditions.

Pediatric and adult patients with seizures associated with CDKL5 deficiency disorder (CDD)

• Patients must be two years of age and older for ZTALMY indication.
• The pivotal Phase 3 Marigold Study included 101 patients in the ages 2 to 21 range.
• The treatment is the first-and-only FDA-approved therapy specifically for seizures associated with CDD.
• Patients on therapy are the direct recipients of the value proposition, driving product revenue.

Caregivers and families managing CDD-related epilepsy

• This group is the primary decision-maker and payer interface for the prescribed therapy.
• They are seeking control over the 'unpredictable, often devastating reality of living with uncontrolled seizures'.
• The number of families managing this rare disorder is directly proportional to the patient count, which is small given the 1 in 40,000 birth prevalence.

Specialized neurologists and epileptologists treating rare seizure disorders

• These are the key prescribers who must be educated on ZTALMY's profile, especially since further ganaxolone clinical development has been suspended.
• There are roughly 16,000 to 18,000 practicing neurologists in the United States as of 2025.
• Epilepsy is listed as a common subspecialty among these neurologists.
• A 2022 data point indicated 2,450 Epileptologists in the U.S..
• The density of specialists is a factor, as seen by the growth in epileptologists per million people in Level 4 centers from 2.0 to 4.1 between 2012 and 2019.

Global rare disease companies seeking commercial-stage assets

• This segment represents potential future partners or acquirers, especially following the initiation of a strategic alternatives process.
• The company has ex-U.S. revenue channels driven by partners in Europe (Orion) and China (Tenacia).
• The company was awarded a Rare Pediatric Disease Priority Review Voucher upon FDA approval.
• The TTM Revenue is reported at $31.47M with a Net Income (TTM) of -$140.49M as of late 2025 data points.

Finance: draft 13-week cash view by Friday.

Marinus Pharmaceuticals, Inc. (MRNS) - Canvas Business Model: Cost Structure

You're looking at the cost side of Marinus Pharmaceuticals, Inc. (MRNS) as it navigated a major pivot in late 2024 and early 2025. The cost structure is heavily weighted toward R&D and commercial build-out, but significant cost-cutting actions were taken to manage cash runway following clinical trial outcomes.

High Research and Development (R&D) Costs, though reduced by 45% workforce cut

Research and Development expenses were a primary cost driver, reflecting ongoing clinical trials for ganaxolone in indications like refractory status epilepticus (RSE) and tuberous sclerosis complex (TSC). For the nine months ended September 30, 2024, R&D expenses totaled approximately $61.3 million, down from $73.0 million for the same period in 2023. This reduction was partly due to the completion of the RAISE trial and costs associated with the API onshoring effort start-up in 2023. Following the decision to suspend further ganaxolone clinical development in Q4 2024, Marinus Pharmaceuticals initiated a workforce reduction of approximately 45%, which was intended to significantly lower the ongoing R&D burn rate. The company had projected combined Selling, General, and Administrative (SG&A) and R&D expenses for the full year 2024 to be in the range of approximately $135 to $138 million, including about $20 million in stock-based compensation expense. This projection reflected the cost reduction activities implemented in Q4 2024.

Selling, General, and Administrative (SG&A) Expenses for ZTALMY Commercialization

The SG&A component is driven by the commercialization efforts for ZTALMY (ganaxolone oral suspension CV), the company's approved therapy for seizures associated with CDKL5 deficiency disorder (CDD). For the nine months ended September 30, 2024, SG&A expenses were $47.9 million, an increase from $45.8 million in the prior year's comparable period, with the nine-month increase attributed to higher stock-based compensation expense and commercial expenses. The cost reduction plans initiated in Q4 2024 aimed to bring the combined SG&A and R&D spend down, with an expectation that the full impact of savings would be achieved in the third quarter of 2024.

Cost of Product Revenue for ZTALMY Manufacturing and Supply

Costs directly tied to producing and supplying ZTALMY are reflected in the Cost of Product Revenue. For the nine months ended September 30, 2024, this cost was $2.205 million. This compares to $1.047 million for the same nine-month period in 2023. The company was making investments to expand ZTALMY manufacturing capacity in preparation for potential global launches, such as the anticipated commercial launch in China in early 2025.

Interest Expense on Debt and Tiered Payments to Sagard Healthcare

Financing costs include traditional interest expense and the specific revenue interest payments related to the Sagard Healthcare Partners agreement. For the nine months ended September 30, 2024, the reported Interest expense was $12.806 million. The Sagard agreement, a $32.5 million upfront financing from October 2022, mandates tiered payments based on U.S. net sales of ganaxolone, including ZTALMY. The structure is:

• 7.5% on net sales through the first half of 2026.
• 15% on net sales less than $100 million annually thereafter.
• 7.5% on net sales greater than $100 million annually thereafter.

The total amount payable to Sagard is capped at 1.9x the upfront payment, meaning a hard cap of $65 million. This structure represents a significant variable cost tied directly to the commercial success of ZTALMY.

Restructuring Costs Associated with the Strategic Review and Downsizing

The strategic review process, initiated after the Q3 2024 clinical results, led to specific, one-time restructuring costs. For the nine months ended September 30, 2024, Marinus Pharmaceuticals reported Restructuring Costs of $1.950 million. Separately, restructuring costs of $2.0 million were recorded for the three months ended June 30, 2024, resulting from cost-saving initiatives implemented in the second quarter of 2024, which included the workforce reduction.

Here's a quick look at the key expense components from the nine months ended September 30, 2024, compared to the prior year:

Cost Category	Nine Months Ended Sep 30, 2024 (in millions)	Nine Months Ended Sep 30, 2023 (in millions)
Research and Development (R&D)	$61.3	$73.0
Selling, General, and Administrative (SG&A)	$47.9	$45.8
Cost of Product Revenue	$2.205	$1.047
Interest Expense	$12.806	N/A (Interest income was $6.366M)
Restructuring Costs	$1.950	$0

What this estimate hides is the impact of the January 2025 acquisition by Immedica Pharma, which was for $151 million and would fundamentally change the cost structure by absorbing operations, though the 2024 figures show the cost base being managed right before that transaction. Finance: draft pro-forma cash flow statement reflecting the Immedica acquisition impact by next Wednesday.

Marinus Pharmaceuticals, Inc. (MRNS) - Canvas Business Model: Revenue Streams

You're looking at the sources of cash flow for Marinus Pharmaceuticals, Inc. as of late 2025, which, honestly, is a bit complex given the recent strategic shift. The primary, ongoing stream was product sales, but the structure was heavily influenced by financing and partnership activity.

The core commercial revenue stream centered on ZTALMY (ganaxolone) oral suspension for seizures associated with CDKL5 deficiency disorder (CDD). For the full year 2024, Marinus Pharmaceuticals, Inc. had projected ZTALMY U.S. net product revenue to be narrowed to between $33 million and $34 million. This was an update from earlier 2024 guidance which had ranged up to $35 million.

Beyond product sales, Marinus Pharmaceuticals, Inc. had several non-recurring or partnership-dependent revenue components:

• Potential milestone payments from international partners for CDD launches.
• Upfront and tiered payments from future collaboration or licensing agreements.

Regarding international partners, Orion Corporation, which held an exclusive license for Europe, the UK, and Switzerland, was mentioned in connection with potential future launches in select European countries in the second half of 2024. However, Marinus Pharmaceuticals, Inc. terminated its collaboration and supply agreements with Orion Corporation near the end of 2024 as part of its strategic review. This termination relieved Orion from a pending €500,000 development cost payment for Q4 2024, but Marinus Pharmaceuticals, Inc. became obligated to pay Orion €1,500,000 under specific conditions related to a significant transaction. China's Tenacia Biotechnology was anticipating a commercial launch for ZTALMY in early 2025.

Federal contract revenue from the Biomedical Advanced Research and Development Authority (BARDA) was a diminishing stream. For the three and nine months ended September 30, 2024, Marinus Pharmaceuticals, Inc. recognized only $0.1 million and $0.3 million, respectively, in BARDA revenue. This was a significant drop from the $1.9 million and $10.8 million recognized in the same periods of 2023, primarily due to the completion of the BARDA base period funding in Q4 2023.

To bolster its cash position during the strategic review, Marinus Pharmaceuticals, Inc. secured a significant financing agreement in mid-2025. In July 2025, the company entered into a revenue interest financing agreement with Sagard Healthcare Partners for a total of $32.5 million upfront in return for payments based on U.S. net sales of ganaxolone. This is a key example of upfront payments from a financing/licensing-like arrangement. The company had also tapped Barclays to help explore strategic alternatives following a trial miss in October 2024.

The ultimate realization of value from the strategic alternatives process was the acquisition by Immedica Pharma AB. The transaction was structured as a cash tender offer to acquire all outstanding shares for USD 0.55 per share, corresponding to an implied enterprise value of approximately USD 151 Million. This acquisition, expected to close in Q1 2025, represents the potential proceeds from the sale of the company. The company's Board of Directors unanimously approved this deal.

Here's a quick look at the key financial figures related to these streams:

Revenue/Financing Component	Amount/Range	Period/Context
ZTALMY U.S. Net Product Revenue Guidance	$33 million to $34 million	Full Year 2024
BARDA Federal Contract Revenue	$0.3 million (YTD)	Nine Months Ended September 30, 2024
Sagard Financing Upfront Payment	$32.5 million	July 2025
Orion Termination Obligation (Contingent)	€1,500,000	Due upon a significant transaction
Immedica Acquisition Implied Enterprise Value	Approximately USD 151 Million	Transaction Value

The company's cash position as of June 30, 2024, was $64.7 million, expected to fund operations into the second quarter of 2025, before factoring in the July 2025 financing. Defintely, the revenue mix shifted dramatically from product sales and government grants to financing activities and, ultimately, an acquisition event.