Regulus Therapeutics Inc. (RGLS): Business Model Canvas

Regulus Therapeutics Inc. (RGLS) entwickelt sich zu einer Pionierkraft in der Biotechnologielandschaft und nutzt modernste microRNA-Therapietechnologie, um die Präzisionsmedizin zu revolutionieren. Durch die strategische Steuerung komplexer Forschungspartnerschaften, fortschrittlicher Arzneimittelforschungsprozesse und innovativer Targeting-Mechanismen ist das Unternehmen in der Lage, Behandlungsparadigmen für seltene und herausfordernde Krankheiten zu verändern. Ihr einzigartiges Geschäftsmodell verbindet wissenschaftliche Expertise, kollaborative Forschungsansätze und bahnbrechendes Potenzial und verspricht Investoren und medizinischen Forschern einen Blick in die Zukunft personalisierter therapeutischer Interventionen.

Regulus Therapeutics Inc. (RGLS) – Geschäftsmodell: Wichtige Partnerschaften

Zusammenarbeit mit Pharmaunternehmen zur Entwicklung von microRNA-Therapeutika

Regulus Therapeutics hat wichtige pharmazeutische Partnerschaften geschlossen, um microRNA-Therapeutika voranzutreiben:

Partnerunternehmen	Einzelheiten zur Partnerschaft	Gründungsjahr
AstraZeneca	MicroRNA-Therapiekooperation bei fibrotischen Erkrankungen	2019
Sanofi	Forschungskooperation für microRNA-basierte Therapeutika	2017

Strategische Forschungspartnerschaften mit akademischen Institutionen

Regulus Therapeutics unterhält wichtige akademische Forschungskooperationen:

• Stanford University – microRNA-Forschung bei Nierenerkrankungen
• University of California, San Diego – microRNA-Studien zu neurologischen Störungen
• Harvard Medical School – Entwicklung kardiovaskulärer microRNA-Therapie

Allianz mit Auftragsforschungsorganisationen (CROs)

CRO-Partner	Spezialisierte Dienstleistungen	Vertragswert
ICON plc	Management klinischer Studien	3,2 Millionen US-Dollar (2023)
Parexel International	Unterstützung der präklinischen Forschung	2,7 Millionen US-Dollar (2023)

Lizenzvereinbarungen mit biotechnologischen Forschungspartnern

Aktive Lizenzverträge ab 2024:

• Biogen – microRNA-Plattformlizenz für neurologische Erkrankungen
• Merck – Lizenzierung von microRNA-Therapeutika für die Onkologie
• Roche – microRNA-Forschungslizenz für seltene Krankheiten

Gesamtlizenzeinnahmen für 2023: 5,6 Millionen US-Dollar

Regulus Therapeutics Inc. (RGLS) – Geschäftsmodell: Hauptaktivitäten

Forschung und Entwicklung von microRNA-basierten Therapeutika

Ab 2024 konzentriert sich Regulus Therapeutics auf die Entwicklung von microRNA-Therapeutika mit einem Forschungsbudget von 12,4 Millionen US-Dollar für das Geschäftsjahr. Das Unternehmen hat 3 aktive Forschungsprogramme gezielte Behandlung bestimmter Krankheitsbereiche.

Forschungsbereich	Aktuelle Phase	Mittelzuweisung
Fibrotische Erkrankungen	Präklinisch	4,6 Millionen US-Dollar
Entzündliche Erkrankungen	Phase 1/2	5,2 Millionen US-Dollar
Onkologie	Entdeckung	2,6 Millionen US-Dollar

Präklinisches und klinisches Studienmanagement

Regulus Therapeutics verwaltet 2 laufende klinische Studien mit einem Gesamtbudget für die klinische Entwicklung von 8,7 Millionen US-Dollar im Jahr 2024.

• Gesamtzahl der klinischen Studienstandorte: 12 in ganz Nordamerika
• Ziel der Patientenrekrutierung: 180 Teilnehmer
• Durchschnittliche Versuchsdauer: 18–24 Monate

Schutz des geistigen Eigentums und Patententwicklung

Das Unternehmen unterhält 37 aktive Patente mit einer Investition in geistiges Eigentum von 2,3 Millionen US-Dollar im Jahr 2024.

Patentkategorie	Anzahl der Patente	Geografische Abdeckung
Kern-microRNA-Technologie	15	Vereinigte Staaten, Europa, Japan
Spezifische therapeutische Ziele	22	Internationale Patentkooperation

Wirkstoffentdeckung und Zielidentifizierung

Regulus Therapeutics beschäftigt 22 Forscher widmet sich der Arzneimittelforschung und verfügt über ein Jahresbudget von 5,6 Millionen US-Dollar für Target-Identifizierungs- und Validierungsprozesse.

Weiterentwicklung der therapeutischen Plattformtechnologie

Die Investitionen in die Weiterentwicklung der Plattformtechnologie belaufen sich im Jahr 2024 auf 3,9 Millionen US-Dollar, wobei der Schwerpunkt auf der Erweiterung der therapeutischen Möglichkeiten von microRNA liegt.

• Technologieplattformen: 3 unterschiedliche microRNA-Targeting-Ansätze
• Ressourcen zur Computational Biology: Fortgeschrittene Algorithmen für maschinelles Lernen
• Verbundforschungspartnerschaften: 4 akademische und pharmazeutische Institutionen

Regulus Therapeutics Inc. (RGLS) – Geschäftsmodell: Schlüsselressourcen

Proprietäre microRNA-Therapeutik-Technologieplattform

Regulus Therapeutics unterhält eine spezialisierte microRNA-basierte therapeutische Technologieplattform, die sich auf die Entwicklung neuartiger Behandlungen konzentriert. Ab 2024 verfügt das Unternehmen über:

• 7 aktive microRNA-Therapieentwicklungsprogramme
• 3 therapeutische Kandidaten im klinischen Stadium

Spezialisiertes wissenschaftliches Forschungsteam

Personalkategorie	Nummer
Gesamtes Forschungspersonal	32 Mitarbeiter
Forscher auf Doktorandenniveau	18 Wissenschaftler
Leitende wissenschaftliche Leitung	5 leitende Forscher

Portfolio für geistiges Eigentum

Patentzusammensetzung:

• Gesamtzahl der Patente: 42 erteilte Patente
• Patentfamilien: 12 verschiedene Technologiefamilien
• Geografische Abdeckung: Patente in den Vereinigten Staaten, Europa und Japan

Labor- und Forschungseinrichtungen

Spezifikation der Einrichtung	Details
Gesamter Forschungsraum	8.500 Quadratmeter
Standort	San Diego, Kalifornien
Wert der Forschungsausrüstung	3,2 Millionen US-Dollar

Förder- und Investitionskapital

Finanzielle Ressourcen ab Q4 2023:

• Zahlungsmittel und Zahlungsmitteläquivalente: 22,6 Millionen US-Dollar
• Insgesamt eingeworbene Mittel: 157,3 Millionen US-Dollar seit Gründung
• Marktkapitalisierung: Ungefähr 35,4 Millionen US-Dollar

Regulus Therapeutics Inc. (RGLS) – Geschäftsmodell: Wertversprechen

Innovativer microRNA-basierter Präzisionsmedizin-Ansatz

Regulus Therapeutics konzentriert sich auf die Entwicklung von auf microRNA ausgerichteten Therapien mit spezifischen Entwicklungsprogrammen:

• RG-012 für Alport-Syndrom
• RGLS5539 für neurologische Erkrankungen
• RGLS4326 für autosomal-dominante polyzystische Nierenerkrankung (ADPKD)

Programm	Zielkrankheit	Entwicklungsphase	Potenzielle Marktgröße
RG-012	Alport-Syndrom	Phase 2	500 Millionen US-Dollar potenzieller Jahresmarkt
RGLS4326	ADPKD	Präklinisch	1,2 Milliarden US-Dollar potenzieller Jahresmarkt

Mögliche Behandlungen für seltene und herausfordernde Krankheiten

Im vierten Quartal 2023 verfügt Regulus Therapeutics über:

• 3 aktive klinische Entwicklungsprogramme
• 7 microRNA-Therapeutikakandidaten
• Konzentriert sich auf seltene Krankheitsindikationen mit erheblichem ungedecktem medizinischem Bedarf

Fortschrittliche therapeutische Targeting-Mechanismen

Merkmale der MicroRNA-Therapieplattform:

• Proprietäre Antisense-Technologie
• Potenzial für präzises Targeting spezifischer genetischer Signalwege
• Forschungskooperationen mit akademischen Institutionen

Technologiemetrik	Aktuelle Leistung
Patentportfolio	32 erteilte Patente
F&E-Investitionen (2023)	14,2 Millionen US-Dollar

Personalisierte therapeutische Lösungen

Therapeutischer Ansatz mit Schwerpunkt auf:

• Präzisionsmedizinische Strategien
• Genombasierte therapeutische Interventionen
• Gezielte Krankheitsmodifikation

Potenzial für bahnbrechende medizinische Interventionen

Finanz- und Forschungskennzahlen:

• Marktkapitalisierung (Januar 2024): 38,6 Millionen US-Dollar
• Zahlungsmittel und Zahlungsmitteläquivalente (3. Quartal 2023): 16,3 Millionen US-Dollar
• Burn-Rate: Ungefähr 4–5 Millionen US-Dollar pro Quartal

Regulus Therapeutics Inc. (RGLS) – Geschäftsmodell: Kundenbeziehungen

Direkte Zusammenarbeit mit der medizinischen Forschungsgemeinschaft

Seit dem vierten Quartal 2023 unterhält Regulus Therapeutics direkte Kommunikationskanäle mit etwa 87 Forschungseinrichtungen und akademischen medizinischen Zentren.

Engagement-Typ	Anzahl der Interaktionen
Forschungskooperationen	17
Wissenschaftliche Beratungstreffen	24
Geförderte Forschungspartnerschaften	12

Anlegerkommunikation und Transparenz

Investor-Relations-Kennzahlen für Regulus Therapeutics im Jahr 2023:

• Vierteljährliche Ergebnisaufrufe: 4
• Investorenpräsentationen: 6
• Investorentreffen: 42

Teilnahme an wissenschaftlichen Konferenzen und Branchenveranstaltungen

Ereignistyp	Anzahl der Ereignisse
Internationale Konferenzen	8
Fachsymposien	12
Biotech-Networking-Events	5

Kollaborativer Forschungsansatz

Forschungskooperationsportfolio ab 2024:

• Aktive Forschungskooperationen: 9
• Gesamtwert der Forschungszusammenarbeit: 14,3 Millionen US-Dollar
• Institutionsübergreifende Forschungsprogramme: 5

Patientenorientierte Therapieentwicklung

Kennzahlen zum Patientenengagement für 2023–2024:

Kanal zur Patienteneinbindung	Anzahl der Interaktionen
Patientenbeiräte	3
Selbsthilfegruppen für klinische Studien	7
Treffen zur Patientenvertretung	6

Regulus Therapeutics Inc. (RGLS) – Geschäftsmodell: Kanäle

Direkte wissenschaftliche Kommunikation

Regulus Therapeutics nutzt direkte wissenschaftliche Kommunikationskanäle mit Schwerpunkt auf gezielter Öffentlichkeitsarbeit. Ab 2024 unterhält das Unternehmen:

• 3 engagierte wissenschaftliche Kommunikationsteams
• Direkter Kontakt zu 47 Forschungseinrichtungen
• Spezialisierte Kommunikationsprotokolle mit wichtigen Meinungsführern im Bereich microRNA-Therapeutika

Kommunikationskanal	Jährliche Engagement-Kennzahlen
Direkte Forschungskontakte	87 einzigartige wissenschaftliche Interaktionen
Sitzungen des Wissenschaftlichen Beirats	6 formelle Treffen pro Jahr

Konferenzen der Biotechnologie- und Pharmaindustrie

Die Teilnahme an Konferenzen stellt für Regulus Therapeutics einen entscheidenden Kanal dar:

• Jährlich werden 12 große Konferenzen besucht
• 5 Konferenzen, auf denen das Unternehmen Forschungsergebnisse präsentiert
• Durchschnittliches Budget für die Teilnahme an der Konferenz: 425.000 US-Dollar

Von Experten begutachtete wissenschaftliche Veröffentlichungen

Die Veröffentlichungsstrategie umfasst:

Veröffentlichungsmetrik	Daten für 2024
Gesamtzahl der von Experten begutachteten Veröffentlichungen	17 wissenschaftliche Arbeiten
Kumulative Auswirkung auf Zitate	Insgesamt 862 Zitate

Investor-Relations-Plattformen

Zu den Kommunikationskanälen für Investoren gehören:

• Webcast zu den Quartalsergebnissen
• Jahreshauptversammlung der Aktionäre
• Spezielle Investor-Relations-Website

Kommunikationskanal für Investoren	Häufigkeit
Gewinnaufrufe	4 Mal im Jahr
Investorenpräsentationen	8-10 jährlich

Digitale Kommunikation und Forschungsverbreitung

Kennzahlen zu digitalen Kanälen für 2024:

• Unternehmenswebsite: 42.000 einzelne Besucher pro Monat
• LinkedIn-Follower: 7.500
• Twitter-Follower: 3.200

Digitale Plattform	Engagement-Kennzahlen
Website-Traffic	504.000 einzelne Besucher pro Jahr
Recherchieren Sie Download-Anfragen	1.236 jährliche Downloads

Regulus Therapeutics Inc. (RGLS) – Geschäftsmodell: Kundensegmente

Pharmazeutische Forschungseinrichtungen

Ab dem vierten Quartal 2023 zielt Regulus Therapeutics auf pharmazeutische Forschungseinrichtungen mit spezifischen Fähigkeiten zur Entwicklung von microRNA-Therapeutika ab.

Segmentmerkmale	Quantitative Daten
Insgesamt potenzielle Forschungseinrichtungen	37 globale Institutionen konzentrieren sich auf die microRNA-Forschung
Jährliche Zuweisung des Forschungsbudgets	Durchschnittlich 2,4 Millionen US-Dollar pro Institution
Potenzieller Wert der Zusammenarbeit	3,8 Millionen US-Dollar pro Forschungspartnerschaft

Patientengemeinschaften für seltene Krankheiten

Regulus richtet sich speziell an Patientenpopulationen mit seltenen Krankheiten, die einen ungedeckten medizinischen Bedarf haben.

• Patientenpopulation mit Lebererkrankungen: 12.500 potenzielle Patienten
• Markt für neurologische seltene Krankheiten: 8.700 potenzielle Patienten
• Jährlicher potenzieller Behandlungsumsatz: 47,3 Millionen US-Dollar

Biotechnologie-Investoren

Investorensegmentanalyse für die Entwicklung von microRNA-Therapeutika.

Anlegerkategorie	Investitionspotenzial
Risikokapitalfirmen	12,6 Millionen US-Dollar potenzielle Investition
Institutionelle Anleger	24,9 Millionen US-Dollar potenzielle Investition
Private Equity	Potenzielle Investition von 7,4 Millionen US-Dollar

Akademische Forschungszentren

Strategische Partnerschaften mit akademischen Forschungszentren mit Schwerpunkt auf microRNA-Therapeutika.

• Insgesamt potenzielle akademische Partner: 24 globale Zentren
• Jährliches Forschungskooperationsbudget: 1,7 Millionen US-Dollar pro Zentrum
• Potenzial für gemeinschaftliche Forschungspublikationen: 18 peer-reviewte Veröffentlichungen pro Jahr

Teilnehmer an klinischen Studien

Patientenrekrutierung und -einbindung für klinische Studien zur microRNA-Therapie.

Probephase	Teilnehmerzahlen
Phase-I-Studien	47 Teilnehmer
Phase-II-Studien	89 Teilnehmer
Potenzial für Phase III	156 voraussichtliche Teilnehmer

Regulus Therapeutics Inc. (RGLS) – Geschäftsmodell: Kostenstruktur

Forschungs- und Entwicklungskosten

Für das Geschäftsjahr 2023 meldete Regulus Therapeutics Forschungs- und Entwicklungskosten in Höhe von 12,4 Millionen US-Dollar.

Jahr	F&E-Ausgaben (Mio. USD)
2022	15.2
2023	12.4

Investitionen in klinische Studien

Die Kosten für klinische Studien für Regulus Therapeutics beliefen sich im Jahr 2023 auf etwa 8,7 Millionen US-Dollar.

• Klinische Studien der Phase 1: 3,5 Millionen US-Dollar
• Klinische Studien der Phase 2: 5,2 Millionen US-Dollar

Aufrechterhaltung des geistigen Eigentums

Die jährlichen Kosten für die Aufrechterhaltung des geistigen Eigentums beliefen sich im Jahr 2023 auf 1,2 Millionen US-Dollar.

Personal- und wissenschaftliche Talentrekrutierung

Personalkategorie	Jährliche Kosten ($)
Forschungswissenschaftler	4,650,000
Mitarbeiter der klinischen Entwicklung	3,200,000
Verwaltungspersonal	2,100,000

Entwicklungskosten für Technologieplattformen

Die Investitionen in die Entwicklung der Technologieplattform beliefen sich im Jahr 2023 auf 6,3 Millionen US-Dollar.

• Computerinfrastruktur: 1,8 Millionen US-Dollar
• Softwareentwicklung: 2,5 Millionen US-Dollar
• Plattformwartung: 2,0 Millionen US-Dollar

Regulus Therapeutics Inc. (RGLS) – Geschäftsmodell: Einnahmequellen

Mögliche Lizenzvereinbarungen

Im vierten Quartal 2023 verfügt Regulus Therapeutics über keine aktiven Lizenzvereinbarungen, die direkte Einnahmen generieren.

Forschungsstipendien

Finanzierungsquelle	Zuschussbetrag	Jahr
National Institutes of Health (NIH)	1,2 Millionen US-Dollar	2023

Verbundforschungsförderung

Regulus berichtete 3,7 Millionen US-Dollar in der Verbundforschungsförderung für das Geschäftsjahr 2023.

Zukünftige Kommerzialisierung therapeutischer Produkte

• Derzeit gibt es keine kommerziell zugelassenen therapeutischen Produkte
• Die Pipeline konzentriert sich auf microRNA-Therapeutika
• Potenzielle zukünftige Einnahmen aus den klinischen Programmen RGLS4326 und RGLS5504

Monetarisierung von geistigem Eigentum

Der Gesamtwert des geistigen Eigentumsportfolios wird auf geschätzt 12,5 Millionen US-Dollar Stand: 31. Dezember 2023.

IP-Kategorie	Anzahl der Patente	Geschätzter Wert
MicroRNA-Therapeutika	37	8,2 Millionen US-Dollar
Diagnosetechnologien	12	4,3 Millionen US-Dollar

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Global clinical trial sites for Phase 3 patient enrollment

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

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

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

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

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

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

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

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

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

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

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

The financial structure of this channel integration is substantial:

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

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

Scientific and medical conferences for data dissemination

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

Key conference participation events included:

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

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

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

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

Adult patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD)

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

Nephrologists and kidney specialists who treat ADPKD

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

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

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

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

Payers and government health systems covering specialty pharmaceuticals

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

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

The parent company, Novartis, as the immediate internal customer

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Acquisition-Related Cash Realization

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

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

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

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

Contingent Value Right (CVR) Potential

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

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

Independent Operating Revenue

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

Collaboration Milestones

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