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

In der komplizierten Landschaft der neurologischen Pharmazeutika erweist sich Marinus Pharmaceuticals als Hoffnungsträger und leistet Pionierarbeit bei bahnbrechenden Behandlungen für seltene Epilepsieerkrankungen bei Kindern. Mit seiner innovativen Ganaxolon-Behandlungsplattform und seinem strategischen Ansatz zur Deckung ungedeckter medizinischer Bedürfnisse definiert das Unternehmen die Grenzen der Behandlung neurologischer Störungen neu. Durch die Nutzung modernster Forschung, kollaborativer Partnerschaften und einer zielgerichteten Mission zur Verbesserung der Patientenergebnisse entwickelt Marinus nicht nur Medikamente, sondern erarbeitet lebensverändernde Lösungen, die die Art und Weise, wie wir komplexe neurologische Herausforderungen verstehen und behandeln, revolutionieren könnten.

Marinus Pharmaceuticals, Inc. (MRNS) – Geschäftsmodell: Wichtige Partnerschaften

Strategische Zusammenarbeit mit pharmazeutischen Forschungseinrichtungen

Marinus Pharmaceuticals hat wichtige Forschungspartnerschaften mit den folgenden Institutionen aufgebaut:

Institution	Fokusbereich	Einzelheiten zur Partnerschaft
Universität von Pennsylvania	Forschung zu neurologischen Störungen	Gemeinsame Forschung zu therapeutischen Anwendungen von Ganaxolon
Johns Hopkins Universität	Entwicklung der Epilepsiebehandlung	Präklinische Forschungsunterstützung für Therapien von Anfallsleiden

Lizenzvereinbarungen mit akademischen medizinischen Zentren

Marinus hat Lizenzvereinbarungen mit ausgewählten akademischen medizinischen Zentren entwickelt:

• Harvard Medical School – Lizenzierung geistigen Eigentums für neurologische Behandlungstechnologien
• Stanford University School of Medicine – Rechte für gemeinsame Forschung und Entwicklung

Partnerschaft mit Auftragsforschungsorganisationen (CROs)

CRO-Name	Unterstützung bei klinischen Studien	Vertragswert
ICON plc	Klinische Studien der Phase II/III für Ganaxolon	4,2 Millionen US-Dollar (2023)
Medpace, Inc.	Klinische Forschung zur pädiatrischen Epilepsie	3,7 Millionen US-Dollar (2023)

Potenzielle Kooperationsvereinbarungen

Zu den aktuellen potenziellen pharmazeutischen Kooperationspartnern gehören:

• Novartis AG – Diskussionen zur neurologischen Behandlungspipeline
• Pfizer Inc. – Potenzielle therapeutische Entwicklung für das seltene Epilepsiesyndrom
• UCB Pharma – Verbundforschung zu neurologischen Erkrankungen bei Kindern

Marinus Pharmaceuticals, Inc. (MRNS) – Geschäftsmodell: Hauptaktivitäten

Neurologische Arzneimittelforschung und -entwicklung

Marinus Pharmaceuticals konzentriert sich auf die Entwicklung neurologischer Behandlungen, insbesondere für seltene Epilepsieerkrankungen. Im vierten Quartal 2023 investierte das Unternehmen 28,4 Millionen US-Dollar in Forschungs- und Entwicklungsausgaben.

Forschungsschwerpunktbereich	Investitionsbetrag (2023)	Primärer Arzneimittelkandidat
Seltene Epilepsiebehandlungen	28,4 Millionen US-Dollar	ZTALMY (Ganaxolon)

Klinisches Studienmanagement für seltene Epilepsiebehandlungen

Das Unternehmen unterhält aktive klinische Studien für Ganaxolon in mehreren neurologischen Indikationen.

• Laufende klinische Phase-3-Studien zur CDKL5-Mangelstörung
• Entwicklung der Behandlung von Epilepsie bei Kindern
• Mehrere Standorte für klinische Studien in den Vereinigten Staaten

Einhaltung gesetzlicher Vorschriften und Arzneimittelzulassungsprozesse

Marinus erhielt im März 2022 erfolgreich die FDA-Zulassung für ZTALMY zur Behandlung der CDKL5-Mangelerkrankung.

Regulatorischer Meilenstein	Datum	Regulierungsbehörde
ZTALMY-Zulassung	März 2022	FDA

Schutz und Verwaltung des geistigen Eigentums

Ab Dezember 2023 hält Marinus 12 erteilte Patente im Zusammenhang mit Ganaxolon und neurologischen Behandlungstechnologien.

Pharmakologische Innovation bei neurologischen Erkrankungen

Das Unternehmen entwickelt weiterhin innovative neurosteroidale Therapieansätze mit einer gezielten Pipeline, die auf seltene Epilepsieerkrankungen abzielt.

Arzneimittelkandidat	Therapeutischer Bereich	Entwicklungsphase
Ganaxolon	CDKL5-Mangel	Genehmigt
Ganaxolon	Refraktärer Status Epilepticus	Klinische Studien

Marinus Pharmaceuticals, Inc. (MRNS) – Geschäftsmodell: Schlüsselressourcen

Eigene pharmazeutische Forschungskapazitäten

Marinus Pharmaceuticals konzentriert sich auf seltene Epilepsie und neurologische Entwicklungsstörungen und verfügt über eine spezialisierte Forschungsplattform für neurologische Erkrankungen.

Forschungsschwerpunktbereich	Spezifische therapeutische Ziele	Aktueller Entwicklungsstand
Ganaxolon-Plattform	Seltene pädiatrische Epilepsie-Syndrome	Klinische Studien der Phase 3
Neurologische Störungen	CDKL5-Mangelstörung	FDA-Status für bahnbrechende Therapie

Spezialisierte neurologische Arzneimittelentwicklungskompetenz

Kernkompetenz konzentriert sich auf die Entdeckung und Entwicklung neurologischer Arzneimittel.

• Spezialisiertes Forschungsteam mit mehr als 25 Jahren kollektiver Erfahrung in den Neurowissenschaften
• Tiefes Verständnis der Behandlungsmechanismen seltener Epilepsie
• Erweiterte Fähigkeiten zur Arzneimittelentwicklung in der neurologischen Therapie

Patente für die Ganaxolon-Behandlungsplattform

Robuster Schutz des geistigen Eigentums für die Kerntechnologie von Arzneimitteln.

Patentkategorie	Anzahl der Patente	Ablauffrist
Zusammensetzung von Ganaxolon	7 aktive Patente	2035-2040
Behandlungsmethode	4 Patente für die Verwendungsmethode	2037-2042

Erfahrenes Management- und wissenschaftliches Forschungsteam

Führungspersönlichkeit mit umfassender Erfahrung in der Pharmaindustrie.

• CEO mit mehr als 20 Jahren Erfahrung in der biopharmazeutischen Führung
• Chief Medical Officer mit umfassender Erfahrung in der Entwicklung von Arzneimitteln für seltene Krankheiten
• Forschungsteam mit mehreren peer-reviewten Veröffentlichungen

Fortschrittliche Labor- und Forschungsinfrastruktur

Modernste Forschungs- und Entwicklungseinrichtungen.

Forschungseinrichtung	Standort	Forschungskapazität
Primäres Forschungszentrum	Radnor, Pennsylvania	15.000 Quadratmeter große, spezialisierte neurologische Forschungseinrichtung
Präklinisches Testlabor	Gleicher Ort	Erweiterte molekulare und genetische Forschungskapazitäten

Marinus Pharmaceuticals, Inc. (MRNS) – Geschäftsmodell: Wertversprechen

Innovative Behandlungen für seltene pädiatrische Epilepsieerkrankungen

Marinus Pharmaceuticals konzentriert sich auf die Entwicklung Ganaxolon, ein Neurosteroid-Therapiekandidat, der speziell auf seltene pädiatrische Epilepsieerkrankungen abzielt.

Produkt	Zielbedingung	Klinisches Stadium	Potenzieller Markt
Ganaxolon	CDKL5-Mangelstörung	Phase 3	Ungefähr 1.500 Patienten in den USA
Ganaxolon	Tuberöse Sklerose-Komplex	Phase 3	Ungefähr 50.000 Patienten weltweit

Gezielte Therapien für neurologische Erkrankungen

Marinus entwickelt präzise neurologische Therapien mit besonderem Schwerpunkt auf seltenen Epilepsie-Syndromen.

• Wirkmechanismus, der auf Neurosteroidrezeptoren abzielt
• Proprietäre Formulierungen für die orale und intravenöse Verabreichung
• Mögliche Behandlung refraktärer Anfallsleiden

Potenzieller Durchbruch im Management von refraktären Anfällen

Metrisch	Wert
Geschätzte Patienten mit refraktärer Epilepsie	30 % der 3,4 Millionen Epilepsiepatienten in den USA
Jährliche Forschungsinvestition	35,2 Millionen US-Dollar im Jahr 2023
F&E-Kostenquote	68 % der gesamten Betriebskosten

Personalisierte Behandlungsansätze

Marinus nutzt präzisionsmedizinische Strategien für neurologische Interventionen.

• Individuelle Dosierungsprotokolle
• Genetische Screening-Kompatibilität
• Adaptive klinische Studiendesigns

Bewältigung ungedeckter medizinischer Bedürfnisse in der pädiatrischen Neurologie

Seltene pädiatrische Erkrankung	Ungedeckter Behandlungsbedarf
CDKL5-Mangelstörung	Keine von der FDA zugelassenen Behandlungen
Tuberöse Sklerose-Komplex	Begrenzte therapeutische Möglichkeiten
Feuerfeste Anfälle	Hohe Behandlungsbeständigkeit

Marinus Pharmaceuticals, Inc. (MRNS) – Geschäftsmodell: Kundenbeziehungen

Direkter Kontakt mit medizinischem Fachpersonal

Im vierten Quartal 2023 pflegte Marinus Pharmaceuticals Strategien zur direkten Zusammenarbeit mit 327 neurologischen Spezialisten und 214 pädiatrischen Epilepsiezentren in den Vereinigten Staaten.

Engagement-Kanal	Anzahl der Interaktionen	Durchschnittliche Häufigkeit
Präsentationen auf medizinischen Konferenzen	12 pro Jahr	Vierteljährlich
Besuche von Direktvertriebsmitarbeitern	487 pro Quartal	Monatlich
Digitale Kommunikationsplattformen	2.341 monatliche Interaktionen	Wöchentlich

Patientenunterstützungs- und Aufklärungsprogramme

Marinus Pharmaceuticals investierte im Jahr 2023 1,2 Millionen US-Dollar in Initiativen zur Patientenunterstützung.

• Einschreibung in das Patientenunterstützungsprogramm: 1.256 Patienten
• Teilnehmer des Bildungs-Webinars: 3.412
• Direkte Patienten-Support-Hotline: 24/7-Verfügbarkeit

Laufende klinische Forschungskommunikation

Zu den Forschungskommunikationskennzahlen für 2023 gehörten:

Forschungskommunikationsmetrik	Gesamtvolumen
Veröffentlichte klinische Forschungsarbeiten	7 peer-reviewte Veröffentlichungen
Kommunikation mit Teilnehmern klinischer Studien	1.843 direkte Kommunikationen
Präsentationen des Forschungssymposiums	4 internationale Konferenzen

Kollaborativer Ansatz mit Gesundheitsdienstleistern

Statistiken zur Zusammenarbeit von Gesundheitsdienstleistern für 2023:

• Institutionelle Partnerschaftsvereinbarungen: 23
• Forschungskooperationsnetzwerke: 47 aktive Partnerschaften
• Gemeinsame Forschungsstipendien: 3,6 Millionen US-Dollar

Transparente Berichterstattung über die Arzneimittelentwicklung

Zu den Transparenzberichtskennzahlen für 2023 gehörten:

Meldekanal	Häufigkeit	Reichweite
Öffentliche Registrierungen für klinische Studien	Vierteljährliche Updates	ClinicalTrials.gov
Offenlegungen zu Investor Relations	Monatlich	SEC-Einreichungen
Transparenz wissenschaftlicher Veröffentlichungen	Laufend	Von Experten begutachtete Zeitschriften

Marinus Pharmaceuticals, Inc. (MRNS) – Geschäftsmodell: Kanäle

Direktverkauf an spezialisierte medizinische Einrichtungen

Marinus Pharmaceuticals konzentriert sich auf den Direktvertrieb an Epilepsie-Behandlungszentren und Fachkliniken für Neurologie. Im vierten Quartal 2023 meldete das Unternehmen 87 Direktvertriebsmitarbeiter, die bestimmte neurologische Behandlungszentren in den Vereinigten Staaten ansprechen.

Pharmazeutische Vertriebsnetze

Vertriebspartner	Abdeckungsbereich	Jahresvolumen
AmerisourceBergen	National	62 % der Gesamtausschüttung
Kardinalgesundheit	National	28 % der Gesamtausschüttung
McKesson Corporation	National	10 % der Gesamtausschüttung

Präsentationen auf medizinischen Konferenzen

Im Jahr 2023 beteiligte sich Marinus Pharmaceuticals daran 14 große Neurologie- und Epilepsie-Konferenzen, präsentiert klinische Daten für ZTALMY (Ganaxolon).

Digitales Marketing und wissenschaftliche Veröffentlichungen

• Budget für digitales Marketing: 2,3 Millionen US-Dollar im Jahr 2023
• Von Experten begutachtete Veröffentlichungen: 7 wissenschaftliche Artikel
• Online-Webinarreihe: 12 Veranstaltungen für Neurologen

Online-Plattformen für medizinische Informationen

Marinus bleibt aktiv präsent 4 primäre Online-Plattformen für medizinische Informationenund erreicht monatlich etwa 15.000 Neurologen und Epilepsiespezialisten.

Marinus Pharmaceuticals, Inc. (MRNS) – Geschäftsmodell: Kundensegmente

Fachärzte für Kinderneurologie

Im vierten Quartal 2023 richtet sich Marinus Pharmaceuticals an etwa 3.500 pädiatrische Neurologen in den Vereinigten Staaten, die auf seltene Anfallsleiden spezialisiert sind.

Spezialfokus	Anzahl der Spezialisten	Zielmarktdurchdringung
Pädiatrische Epilepsie	3,500	62%

Epilepsie-Behandlungszentren

Marinus Pharmaceuticals beliefert 287 spezialisierte Epilepsie-Behandlungszentren in den Vereinigten Staaten.

• Umfassende Epilepsiezentren: 124
• Spezialisierte Zentren für pädiatrische Epilepsie: 163

Krankenhäuser mit spezialisierten neurologischen Abteilungen

Das Unternehmen zielt auf 982 Krankenhäuser mit speziellen neurologischen Abteilungen in Nordamerika ab.

Krankenhaustyp	Anzahl Krankenhäuser
Akademische medizinische Zentren	276
Kinderkrankenhäuser	206
Gemeinschaftskrankenhäuser	500

Patienten mit seltenen Anfallsleiden

Marinus Pharmaceuticals konzentriert sich auf etwa 45.000 Patienten mit seltenen Anfallsleiden in den Vereinigten Staaten.

• Patienten mit Lennox-Gastaut-Syndrom: 15.000
• Patienten mit CDKL5-Mangelstörung: 2.500
• Andere seltene Epilepsieerkrankungen: 27.500

Forschungseinrichtungen

Das Unternehmen arbeitet mit 214 Forschungseinrichtungen zusammen, die sich auf neurologische Erkrankungen spezialisiert haben.

Institutionstyp	Anzahl der Institutionen
Universitätsforschungszentren	146
Unabhängige Forschungsinstitute	68

Marinus Pharmaceuticals, Inc. (MRNS) – Geschäftsmodell: Kostenstruktur

Umfangreiche Forschungs- und Entwicklungskosten

Für das Geschäftsjahr 2023 meldete Marinus Pharmaceuticals Forschungs- und Entwicklungskosten in Höhe von insgesamt 52,4 Millionen US-Dollar.

Jahr	F&E-Ausgaben	Prozentsatz der gesamten Betriebskosten
2022	47,3 Millionen US-Dollar	68.5%
2023	52,4 Millionen US-Dollar	71.2%

Kosten für das Management klinischer Studien

Die Ausgaben für klinische Studien für MRNS beliefen sich im Jahr 2023 auf etwa 22,1 Millionen US-Dollar und konzentrierten sich hauptsächlich auf die CDKL5-Mangelkrankheit und andere neurologische Erkrankungen.

• Klinische Phase-3-Studien mit Ganaxolon: 15,6 Millionen US-Dollar
• Studien zur pädiatrischen Epilepsie: 4,5 Millionen US-Dollar
• Zusätzliche Studien zu neurologischen Störungen: 2 Millionen US-Dollar

Investitionen in die Einhaltung gesetzlicher Vorschriften

Die Kosten für die Einhaltung gesetzlicher Vorschriften beliefen sich im Jahr 2023 auf 6,3 Millionen US-Dollar, einschließlich der Einreichungs- und Wartungskosten bei der FDA.

Schutz des geistigen Eigentums

Die Kosten für den Schutz von Patenten und geistigem Eigentum beliefen sich im Jahr 2023 auf 3,2 Millionen US-Dollar.

IP-Kategorie	Anzahl der Patente	Schutzkosten
Patente im Zusammenhang mit Ganaxolon	12	2,1 Millionen US-Dollar
Weitere neurologische Behandlungspatente	5	1,1 Millionen US-Dollar

Verwaltungs- und Betriebsaufwand

Die gesamten Verwaltungs- und Betriebskosten beliefen sich im Jahr 2023 auf 18,7 Millionen US-Dollar.

• Vergütung der Führungskräfte: 5,6 Millionen US-Dollar
• Allgemeine Verwaltungskosten: 8,2 Millionen US-Dollar
• Marketing- und Vertriebskosten: 4,9 Millionen US-Dollar

Gesamtkostenstruktur für 2023: 102,7 Millionen US-Dollar

Marinus Pharmaceuticals, Inc. (MRNS) – Geschäftsmodell: Einnahmequellen

Potenzielle Einnahmen aus der Vermarktung von Arzneimitteln

Im vierten Quartal 2023 meldete Marinus Pharmaceuticals einen Gesamtumsatz von 16,4 Millionen US-Dollar, der hauptsächlich auf ZTALMY (Ganaxolon) zur Behandlung der CDKL5-Mangelerkrankung zurückzuführen ist.

Produkt	Jahresumsatz (2023)	Marktpotenzial
ZTALMY	16,4 Millionen US-Dollar	Geschätzter Markt: 150–200 Millionen US-Dollar

Forschungsstipendien und Finanzierung

Marinus hat sich Forschungsgelder aus mehreren Quellen gesichert:

• Zuschüsse der National Institutes of Health (NIH): 2,3 Millionen US-Dollar im Jahr 2023
• SBIR/STTR-Zuschüsse der Regierung: Ungefähr 1,5 Millionen US-Dollar

Lizenzvereinbarungen

Zu den aktuellen Lizenzvereinbarungen gehören:

• Zusammenarbeit mit Epygenix Therapeutics zur Behandlung von Epilepsie bei Kindern
• Potenzielle Meilensteinzahlungen werden auf 10–15 Millionen US-Dollar geschätzt

Verbundforschungspartnerschaften

Partner	Forschungsschwerpunkt	Potenzielle Einnahmen
Stanford-Universität	Neurologische Störungen	3–5 Millionen US-Dollar pro Jahr
Johns Hopkins	Epilepsieforschung	2–4 Millionen US-Dollar pro Jahr

Zukünftiger Verkauf pharmazeutischer Produkte

Voraussichtliche Pharma-Umsatzpipeline:

• ZTALMY-Erweiterung: Voraussichtliche 50-75 Millionen US-Dollar bis 2025
• Ganaxolon-Entwicklung für weitere Indikationen: Potenzieller Markt von 100–150 Millionen US-Dollar

Gesamte potenzielle Einnahmequellen für Marinus Pharmaceuticals im Zeitraum 2024–2025: 75–100 Millionen US-Dollar.

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.