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Marinus Pharmaceuticals, Inc. (MRNS): Business Model Canvas [Jan-2025 Mis à jour] |
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Marinus Pharmaceuticals, Inc. (MRNS) Bundle
Dans le paysage complexe des produits pharmaceutiques neurologiques, Marinus Pharmaceuticals apparaît comme un phare d'espoir, les traitements de percée pionnière pour des conditions d'épilepsie pédiatriques rares. Avec sa plate-forme de traitement Ganaxolone innovante et sa approche stratégique pour répondre aux besoins médicaux non satisfaits, l'entreprise redéfinit les limites de la gestion des troubles neurologiques. En tirant parti des recherches de pointe, des partenariats collaboratifs et une mission axée sur le laser pour transformer les résultats pour les patients, Marinus ne fait pas que développer des médicaments - ils élaborent des solutions qui changent la vie qui pourraient révolutionner la façon dont nous comprenons et traitons les défis neurologiques complexes.
Marinus Pharmaceuticals, Inc. (MRNS) - Modèle d'entreprise: Partenariats clés
Collaboration stratégique avec les institutions de recherche pharmaceutique
Marinus Pharmaceuticals a établi des partenariats de recherche clés avec les institutions suivantes:
| Institution | Domaine de mise au point | Détails du partenariat |
|---|---|---|
| Université de Pennsylvanie | Recherche des troubles neurologiques | Recherche collaborative sur les applications thérapeutiques de Gaaxolone |
| Université Johns Hopkins | Développement du traitement de l'épilepsie | Support de recherche préclinique pour les thérapies sur les troubles de crise |
Accords de licence avec des centres médicaux académiques
Marinus a développé des accords de licence avec certains centres médicaux académiques:
- Harvard Medical School - Licence de propriété intellectuelle pour les technologies de traitement neurologique
- École de médecine de l'Université de Stanford - Droits de recherche et de développement collaboratifs
Partenariat avec les organisations de recherche sous contrat (CROS)
| Nom de CRO | Soutien en essai clinique | Valeur du contrat |
|---|---|---|
| Icône plc | Essais cliniques de phase II / III pour ganaxolone | 4,2 millions de dollars (2023) |
| Medpace, Inc. | Recherche clinique de l'épilepsie pédiatrique | 3,7 millions de dollars (2023) |
Accords de co-développement potentiels
Les partenaires potentiels actuels de collaboration pharmaceutique comprennent:
- Novartis Ag - Discussions sur les pipelines de traitement neurologique
- Pfizer Inc. - Syndrome d'épilepsie rare potentiel Développement thérapeutique
- UCB Pharma - Recherche collaborative en troubles neurologiques pédiatriques
Marinus Pharmaceuticals, Inc. (MRNS) - Modèle d'entreprise: activités clés
Recherche et développement de médicaments neurologiques
Marinus Pharmaceuticals se concentre sur le développement de traitements neurologiques, en particulier pour les conditions d'épilepsie rares. Au quatrième trimestre 2023, la société a investi 28,4 millions de dollars dans les dépenses de recherche et développement.
| Domaine de mise au point de recherche | Montant d'investissement (2023) | Candidat au médicament principal |
|---|---|---|
| Traitements d'épilepsie rares | 28,4 millions de dollars | Ztalmy (Gaaxolone) |
Gestion des essais cliniques pour les traitements d'épilepsie rares
La société maintient des essais cliniques actifs pour la ganaxolone à travers de multiples indications neurologiques.
- Essais cliniques de phase 3 en cours pour le trouble de carence CDKL5
- Développement du traitement de l'épilepsie pédiatrique
- Plusieurs sites d'essais cliniques à travers les États-Unis
Processus de conformité réglementaire et d'approbation des médicaments
Marinus a obtenu avec succès l'approbation de la FDA pour Ztalmy en mars 2022 pour le trouble de carence CDKL5.
| Jalon réglementaire | Date | Corps réglementaire |
|---|---|---|
| Approbation ztalmy | Mars 2022 | FDA |
Protection et gestion de la propriété intellectuelle
En décembre 2023, Marinus tient 12 brevets accordés liés aux technologies de ganaxolone et de traitement neurologique.
Innovation pharmacologique dans les troubles neurologiques
L'entreprise continue de développer des approches thérapeutiques innovantes de neurostéroïdes avec un pipeline ciblé ciblant des conditions d'épilepsie rares.
| Drogue | Zone thérapeutique | Étape de développement |
|---|---|---|
| Ganaxolone | Carence en CDKL5 | Approuvé |
| Ganaxolone | Statut réfractaire épileptique | Essais cliniques |
Marinus Pharmaceuticals, Inc. (MRNS) - Modèle d'entreprise: Ressources clés
Capacités de recherche pharmaceutique propriétaire
Marinus Pharmaceuticals se concentre sur l'épilepsie rare et les troubles neurodéveloppementaux, avec une plate-forme de recherche spécialisée ciblant les conditions neurologiques.
| Domaine de mise au point de recherche | Cibles thérapeutiques spécifiques | Étape de développement actuelle |
|---|---|---|
| Plate-forme de ganaxolone | Syndromes d'épilepsie pédiatriques rares | Essais cliniques de phase 3 |
| Troubles neurologiques | Trouble de carence CDKL5 | Désignation de thérapie révolutionnaire de la FDA |
Expertise spécialisée sur le développement de médicaments neurologiques
Expertise clé concentrée dans la découverte et le développement de médicaments neurologiques.
- Équipe de recherche spécialisée avec plus de 25 ans d'expérience en neuroscience collective
- Compréhension approfondie des mécanismes de traitement rares de l'épilepsie
- Capacités avancées de développement de médicaments en thérapeutique neurologique
Brevets pour la plate-forme de traitement de Gaaxolone
Protection de propriété intellectuelle robuste pour la technologie de base des médicaments.
| Catégorie de brevet | Nombre de brevets | Chronologie d'expiration |
|---|---|---|
| Composition de ganaxolone | 7 brevets actifs | 2035-2040 |
| Méthode de traitement | 4 brevets de méthode d'utilisation | 2037-2042 |
Équipe de gestion expérimentée et de recherche scientifique
Leadership avec une vaste expérience de l'industrie pharmaceutique.
- PDG avec plus de 20 ans dans le leadership biopharmaceutique
- Médecin en chef ayant une vaste expérience de développement de médicaments par maladies rares
- Équipe de recherche avec plusieurs publications évaluées par des pairs
Infrastructure avancée de laboratoire et de recherche
Installations de recherche et développement de pointe.
| Installation de recherche | Emplacement | Capacité de recherche |
|---|---|---|
| Centre de recherche primaire | Radnor, Pennsylvanie | 15 000 pieds carrés de recherche neurologique spécialisée |
| Laboratoire de test préclinique | Même emplacement | Capacités de recherche moléculaire et génétique avancées |
Marinus Pharmaceuticals, Inc. (MRNS) - Modèle d'entreprise: propositions de valeur
Traitements innovants pour des conditions d'épilepsie pédiatriques rares
Marinus Pharmaceuticals se concentre sur le développement ganaxolone, un candidat thérapeutique neurostéroïde ciblant spécifiquement les conditions d'épilepsie pédiatriques rares.
| Produit | Condition cible | Étape clinique | Marché potentiel |
|---|---|---|---|
| Ganaxolone | Trouble de carence CDKL5 | Phase 3 | Environ 1 500 patients aux États-Unis |
| Ganaxolone | Complexe de sclérose tubéreuse | Phase 3 | Environ 50 000 patients dans le monde |
Thérapies ciblées pour les troubles neurologiques
Marinus développe des thérapies neurologiques de précision avec un accent spécifique sur les syndromes d'épilepsie rares.
- Mécanisme d'action ciblant les récepteurs de neurostéroïdes
- Formulations propriétaires pour l'administration orale et intraveineuse
- Traitement potentiel pour les troubles de la crise réfractaire
Percée potentielle dans la gestion des crises réfractaires
| Métrique | Valeur |
|---|---|
| Patients d'épilepsie réfractaires estimés | 30% des 3,4 millions de patients atteints d'épilepsie aux États-Unis |
| Investissement de recherche annuel | 35,2 millions de dollars en 2023 |
| Ratio de dépenses R&D | 68% du total des dépenses d'exploitation |
Approches de traitement personnalisées
Marinus utilise des stratégies de médecine de précision pour les interventions neurologiques.
- Protocoles de dosage individualisés
- Compatibilité du dépistage génétique
- Conceptions d'essais cliniques adaptatifs
Répondre aux besoins médicaux non satisfaits en neurologie pédiatrique
| État pédiatrique rare | Besoin de traitement non satisfait |
|---|---|
| Trouble de carence CDKL5 | Aucun traitement approuvé par la FDA |
| Complexe de sclérose tubéreuse | Options thérapeutiques limitées |
| Saisies réfractaires | Résistance au traitement élevée |
Marinus Pharmaceuticals, Inc. (MRNS) - Modèle d'entreprise: relations avec les clients
Engagement direct avec les professionnels de la santé
Depuis le quatrième trimestre 2023, Marinus Pharmaceuticals a maintenu des stratégies d'engagement direct avec 327 spécialistes neurologiques et 214 centres d'épilepsie pédiatriques à travers les États-Unis.
| Canal de fiançailles | Nombre d'interactions | Fréquence moyenne |
|---|---|---|
| Présentations de la conférence médicale | 12 par an | Trimestriel |
| Visites de représentants des ventes directes | 487 par trimestre | Mensuel |
| Plateformes de communication numérique | 2 341 interactions mensuelles | Hebdomadaire |
Programmes de soutien aux patients et d'éducation
Marinus Pharmaceuticals a investi 1,2 million de dollars dans les initiatives de soutien aux patients pour 2023.
- Inscription du programme d'aide aux patients: 1 256 patients
- Participants au webinaire éducatif: 3 412
- Astuce directe du soutien aux patients: disponibilité 24h / 24 et 7j / 7
Communication de recherche clinique en cours
Les mesures de communication de recherche pour 2023 incluaient:
| Métrique de communication de recherche | Volume total |
|---|---|
| Articles de recherche clinique publiés | 7 publications évaluées par des pairs |
| Communications des participants à l'essai clinique | 1 843 communications directes |
| Présentations du symposium de recherche | 4 conférences internationales |
Approche collaborative avec les prestataires de soins de santé
Statistiques de collaboration des prestataires de soins de santé pour 2023:
- Partenariat institutionnel Accords: 23
- Réseaux de collaboration de recherche: 47 partenariats actifs
- Financement conjoint de la recherche de recherche: 3,6 millions de dollars
Rapports transparents de développement de médicaments
Les mesures de rapport de transparence pour 2023 comprenaient:
| Canal de rapport | Fréquence | Atteindre |
|---|---|---|
| Inscriptions des essais cliniques publics | Mises à jour trimestrielles | ClinicalTrials.gov |
| RÉVÉRATIONS DE RELATIONS D'INVESTISSEMENT | Mensuel | Dépôts de la SEC |
| Publication scientifique Transparence | En cours | Journaux évalués par des pairs |
Marinus Pharmaceuticals, Inc. (MRNS) - Modèle d'entreprise: canaux
Ventes directes vers des installations médicales spécialisées
Marinus Pharmaceuticals se concentre sur les ventes directes centres de traitement de l'épilepsie et Cliniques spécialisées en neurologie. Au quatrième trimestre 2023, la société a signalé 87 représentants des ventes directes ciblant des centres de traitement neurologique spécifiques à travers les États-Unis.
Réseaux de distribution pharmaceutique
| Partenaire de distribution | Zone de couverture | Volume annuel |
|---|---|---|
| Amerisourcebergen | National | 62% de la distribution totale |
| Santé cardinale | National | 28% de la distribution totale |
| McKesson Corporation | National | 10% de la distribution totale |
Présentations de la conférence médicale
En 2023, Marinus Pharmaceuticals a participé à 14 conférences majeures de neurologie et d'épilepsie, présentant des données cliniques pour le ztalmy (Gaaxolone).
Marketing numérique et publications scientifiques
- Budget de marketing numérique: 2,3 millions de dollars en 2023
- Publications évaluées par des pairs: 7 articles scientifiques
- Série de webinaires en ligne: 12 événements ciblant les neurologues
Plateformes d'information médicale en ligne
Marinus maintient une présence active sur 4 plateformes d'information médicale en ligne primaires, atteignant environ 15 000 neurologues et spécialistes de l'épilepsie mensuellement.
Marinus Pharmaceuticals, Inc. (MRNS) - Modèle d'entreprise: segments de clientèle
Spécialistes de la neurologie pédiatrique
Depuis le quatrième trimestre 2023, Marinus Pharmaceuticals cible environ 3 500 neurologues pédiatriques aux États-Unis spécialisés dans les troubles rares.
| Focus spécialisée | Nombre de spécialistes | Pénétration du marché cible |
|---|---|---|
| Épilepsie pédiatrique | 3,500 | 62% |
Centres de traitement de l'épilepsie
Marinus Pharmaceuticals dessert 287 centres de traitement d'épilepsie spécialisés à travers les États-Unis.
- Centres d'épilepsie complètes: 124
- Épilepsie pédiatrique Centres spécialisés: 163
Hôpitaux avec des services de neurologie spécialisés
La société cible 982 hôpitaux avec des départements de neurologie dédiés en Amérique du Nord.
| Type d'hôpital | Nombre d'hôpitaux |
|---|---|
| Centres médicaux académiques | 276 |
| Hôpitaux pour enfants | 206 |
| Hôpitaux communautaires | 500 |
Patients souffrant de troubles de crise rares
Marinus Pharmaceuticals se concentre sur environ 45 000 patients atteints de troubles de crise rares aux États-Unis.
- Patients du syndrome de Lennox-Gastaut: 15 000
- Patients de troubles de la carence CDKL5: 2 500
- Autres troubles rares de l'épilepsie: 27 500
Institutions de recherche
L'entreprise collabore avec 214 institutions de recherche spécialisées dans les conditions neurologiques.
| Type d'institution | Nombre d'institutions |
|---|---|
| Centres de recherche universitaires | 146 |
| Instituts de recherche indépendants | 68 |
Marinus Pharmaceuticals, Inc. (MRNS) - Modèle d'entreprise: Structure des coûts
Dépenses de recherche et développement approfondies
Pour l'exercice 2023, Marinus Pharmaceuticals a déclaré des dépenses de R&D totalisant 52,4 millions de dollars.
| Année | Dépenses de R&D | Pourcentage des dépenses d'exploitation totales |
|---|---|---|
| 2022 | 47,3 millions de dollars | 68.5% |
| 2023 | 52,4 millions de dollars | 71.2% |
Coûts de gestion des essais cliniques
Les dépenses des essais cliniques pour les MRN en 2023 étaient d'environ 22,1 millions de dollars, axée principalement sur le trouble de la carence CDKL5 et d'autres troubles neurologiques.
- Ganaxolone Phase 3 Essais cliniques: 15,6 millions de dollars
- Études d'épilepsie pédiatrique: 4,5 millions de dollars
- Essais supplémentaires de troubles neurologiques: 2 millions de dollars
Investissements de conformité réglementaire
Les coûts de conformité réglementaire pour 2023 s'élevaient à 6,3 millions de dollars, y compris les frais de soumission et d'entretien de la FDA.
Protection de la propriété intellectuelle
Les coûts de protection des brevets et de la propriété intellectuelle en 2023 étaient de 3,2 millions de dollars.
| Catégorie IP | Nombre de brevets | Coûts de protection |
|---|---|---|
| Brevets liés à Ganaxolone | 12 | 2,1 millions de dollars |
| Brevets de traitement neurologique supplémentaires | 5 | 1,1 million de dollars |
Surfaçon administratives et opérationnelles
Les dépenses administratives et opérationnelles totales pour 2023 étaient de 18,7 millions de dollars.
- Rémunération des cadres: 5,6 millions de dollars
- Dépenses administratives générales: 8,2 millions de dollars
- Frais de marketing et de vente: 4,9 millions de dollars
Structure totale des coûts pour 2023: 102,7 millions de dollars
Marinus Pharmaceuticals, Inc. (MRNS) - Modèle d'entreprise: Strots de revenus
Revenus potentiels de commercialisation des médicaments
Au quatrième trimestre 2023, Marinus Pharmaceuticals a déclaré un chiffre d'affaires total de 16,4 millions de dollars, principalement tiré par Ztalmy (Gaaxolone) pour le trouble de carence CDKL5.
| Produit | Revenus annuels (2023) | Potentiel de marché |
|---|---|---|
| Ztalmy | 16,4 millions de dollars | Marché estimé de 150 à 200 millions de dollars |
Subventions et financement de recherche
Marinus a obtenu un financement de recherche à partir de plusieurs sources:
- Subventions des National Institutes of Health (NIH): 2,3 millions de dollars en 2023
- Subventions gouvernementales SBIR / STTR: environ 1,5 million de dollars
Accords de licence
Les accords de licence actuels comprennent:
- Collaboration avec Epygenix Therapeutics pour les traitements d'épilepsie pédiatriques
- Paiements de jalons potentiels estimés à 10 à 15 millions de dollars
Partenariats de recherche collaborative
| Partenaire | Focus de recherche | Revenus potentiels |
|---|---|---|
| Université de Stanford | Troubles neurologiques | 3 à 5 millions de dollars par an |
| Johns Hopkins | Recherche d'épilepsie | 2 à 4 millions de dollars par an |
Ventes de produits pharmaceutiques futures
Pipeline de revenus pharmaceutiques projetés:
- Expansion du ztalmy: prévu de 50 à 75 millions de dollars d'ici 2025
- Développement de Gaaxolone pour des indications supplémentaires: Marché potentiel de 100 à 150 millions de dollars
Total des sources de revenus potentiels pour Marinus Pharmaceuticals en 2024-2025: 75 à 100 millions de dollars.
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.
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