Regulus Therapeutics Inc. (RGLS): Business Model Canvas [Jan-2025 Mis à jour]

Regulus Therapeutics Inc. (RGLS) émerge comme une force pionnière dans le paysage biotechnologie, tirant parti de la technologie thérapeutique de microARN de pointe pour révolutionner la médecine de précision. En naviguant stratégiquement des partenariats de recherche complexes, des processus avancés de découverte de médicaments et des mécanismes de ciblage innovants, l'entreprise est prête à transformer les paradigmes de traitement pour des maladies rares et difficiles. Leur modèle commercial unique entrelace l'expertise scientifique, les approches de recherche collaborative et le potentiel révolutionnaire, les investisseurs prometteurs et les chercheurs médicaux ont un aperçu de l'avenir des interventions thérapeutiques personnalisées.

Regulus Therapeutics Inc. (RGLS) - Modèle d'entreprise: partenariats clés

Collaboration avec des sociétés pharmaceutiques pour le développement de MicroRNA Therapeutics

Regulus Therapeutics a établi des partenariats pharmaceutiques clés pour faire progresser les thérapies MicroRNA:

Entreprise partenaire	Détails du partenariat	Année établie
Astrazeneca	Collaboration thérapeutique MicroRNA pour les maladies fibrotiques	2019
Sanofi	Collaboration de recherche pour les thérapies basées sur les microARN	2017

Partenariats de recherche stratégique avec les établissements universitaires

Regulus Therapeutics maintient des collaborations de recherche académique critiques:

• Université de Stanford - Recherche de microARN sur les maladies rénales
• Université de Californie, San Diego - Études de microARN des troubles neurologiques
• École de médecine de Harvard - Développement thérapeutique des microARN cardiovasculaires

Alliance avec les organisations de recherche sous contrat (CRO)

Partenaire CRO	Services spécialisés	Valeur du contrat
Icône plc	Gestion des essais cliniques	3,2 millions de dollars (2023)
Parexel International	Soutien de la recherche préclinique	2,7 millions de dollars (2023)

Accords de licence avec des partenaires de recherche en biotechnologie

Accords de licence actifs à partir de 2024:

• Biogen - Licence de plate-forme de microARN de maladie neurologique
• Merck - Licence thérapeutique MicroRNAl en oncologie
• Roche - Licence de recherche sur microARN de maladies rares

Revenu total de licences pour 2023: 5,6 millions de dollars

Regulus Therapeutics Inc. (RGLS) - Modèle d'entreprise: Activités clés

Recherche et développement de thérapies à base de microARN

En 2024, Regulus Therapeutics se concentre sur le développement de la thérapeutique des microARN avec un budget de recherche de 12,4 millions de dollars pour l'exercice. La société a 3 programmes de recherche actifs ciblant des zones de maladie spécifiques.

Domaine de recherche	Étape actuelle	Allocation de financement
Maladies fibrotiques	Préclinique	4,6 millions de dollars
Conditions inflammatoires	Phase 1/2	5,2 millions de dollars
Oncologie	Découverte	2,6 millions de dollars

Gestion des essais précliniques et cliniques

Regulus Therapeutics gère 2 essais cliniques en cours avec un budget total de développement clinique de 8,7 millions de dollars en 2024.

• Sites totaux d'essais cliniques: 12 à travers l'Amérique du Nord
• Patient Inscription Cobile: 180 participants
• Durée moyenne de l'essai: 18-24 mois

Protection de la propriété intellectuelle et développement des brevets

La société maintient 37 brevets actifs avec un investissement de propriété intellectuelle de 2,3 millions de dollars en 2024.

Catégorie de brevet	Nombre de brevets	Couverture géographique
Technologie de base du microARN	15	États-Unis, Europe, Japon
Cibles thérapeutiques spécifiques	22	Coopération internationale des brevets

Découverte de médicaments et identification cible

Regulus Therapeutics utilise 22 chercheurs Dédié à la découverte de médicaments, avec un budget annuel de 5,6 millions de dollars pour les processus d'identification et de validation cibles.

Avancement technologique de la plate-forme thérapeutique

L'investissement dans les progrès de la technologie des plateformes atteint 3,9 millions de dollars en 2024, en mettant l'accent sur l'expansion des capacités thérapeutiques des microARN.

• Plateformes technologiques: 3 approches de ciblage des microARN distincts
• Ressources de biologie informatique: algorithmes avancés d'apprentissage automatique
• Partenariats de recherche collaborative: 4 institutions universitaires et pharmaceutiques

Regulus Therapeutics Inc. (RGLS) - Modèle d'entreprise: Ressources clés

Plateforme de technologie thérapeutique de microARN propriétaire

Regulus Therapeutics maintient une plate-forme technologique thérapeutique spécialisée basée sur MicroRNA axée sur le développement de nouveaux traitements. Depuis 2024, la société a:

• 7 programmes de développement thérapeutique de microARN actifs
• 3 candidats thérapeutiques à stade clinique

Équipe de recherche scientifique spécialisée

Catégorie de personnel	Nombre
Personnel de recherche total	32 employés
Chercheurs au niveau du doctorat	18 scientifiques
Leadership scientifique principal	5 chercheurs exécutifs

Portefeuille de propriété intellectuelle

Composition de brevet:

• Brevets totaux: 42 brevets accordés
• Familles de brevets: 12 familles de technologie distinctes
• Couverture géographique: brevets aux États-Unis, en Europe et au Japon

Installations de laboratoire et de recherche

Spécification de l'installation	Détails
Espace de recherche total	8 500 pieds carrés
Emplacement	San Diego, Californie
Valeur de l'équipement de recherche	3,2 millions de dollars

Financement et capital d'investissement

Ressources financières au quatrième trimestre 2023:

• Equivalents en espèces et en espèces: 22,6 millions de dollars
• Financement total collecté: 157,3 millions de dollars depuis la création
• Capitalisation boursière: environ 35,4 millions de dollars

Regulus Therapeutics Inc. (RGLS) - Modèle d'entreprise: propositions de valeur

Approche innovante de médecine de précision basée sur les microARN

Regulus Therapeutics se concentre sur le développement de thérapies ciblées par microARN avec des programmes de développement spécifiques:

• RG-012 pour le syndrome d'Alport
• RGLS5539 pour les troubles neurologiques
• RGLS4326 pour la maladie rénale polykystique dominante autosomique (ADPKD)

Programme	Maladie cible	Étape de développement	Taille du marché potentiel
RG-012	Syndrome d'Alport	Phase 2	Marché annuel potentiel de 500 millions de dollars
RGLS4326	Adpkd	Préclinique	1,2 milliard de dollars de marché annuel potentiel

Traitements potentiels pour les maladies rares et difficiles

Depuis le quatrième trimestre 2023, Regulus Therapeutics a:

• 3 programmes de développement clinique actifs
• 7 candidats thérapeutiques microARN
• Axé sur les indications de maladies rares avec des besoins médicaux non satisfaits importants

Mécanismes de ciblage thérapeutique avancés

Caractéristiques de la plate-forme thérapeutique MicroRNA:

• Technologie antisens propriétaire
• Potentiel de ciblage de précision de voies génétiques spécifiques
• Collaborations de recherche avec des établissements universitaires

Métrique technologique	Performance actuelle
Portefeuille de brevets	32 brevets délivrés
Investissement en R&D (2023)	14,2 millions de dollars

Solutions thérapeutiques personnalisées

Approche thérapeutique se concentrant sur:

• Stratégies de médecine de précision
• Interventions thérapeutiques génomiques
• Modification des maladies ciblées

Potentiel d'interventions médicales révolutionnaires

Métriques financières et de recherche:

• Capitalisation boursière (janvier 2024): 38,6 millions de dollars
• Equivalents en espèces et en espèces (T3 2023): 16,3 millions de dollars
• Taux de brûlure: environ 4 à 5 millions de dollars par trimestre

Regulus Therapeutics Inc. (RGLS) - Modèle d'entreprise: relations clients

Engagement direct avec la communauté de la recherche médicale

Depuis le quatrième trimestre 2023, Regulus Therapeutics maintient des canaux de communication directs avec environ 87 établissements de recherche et centres médicaux universitaires.

Type d'engagement	Nombre d'interactions
Collaborations de recherche	17
Réunions de conseil scientifique	24
Partenariats de recherche financés par les subventions	12

Communication et transparence des investisseurs

Métriques des relations avec les investisseurs pour Regulus Therapeutics en 2023:

• Rendez-vous trimestriel: 4
• Présentations des investisseurs: 6
• Réunions des investisseurs: 42

Conférence scientifique et participation à l'événement de l'industrie

Type d'événement	Nombre d'événements
Conférences internationales	8
Symposiums spécialisés	12
Événements de réseautage biotechnologique	5

Approche de recherche collaborative

Portfolio de collaboration de recherche à partir de 2024:

• Partenariats de recherche actifs: 9
• Valeur totale de collaboration de recherche: 14,3 millions de dollars
• Programmes de recherche inter-institutionnels: 5

Développement thérapeutique axé sur les patients

Métriques d'engagement des patients pour 2023-2024:

Canal de fiançailles des patients	Nombre d'interactions
Boards consultatifs des patients	3
Groupes de soutien aux essais cliniques	7
Réunions de plaidoyer pour les patients	6

Regulus Therapeutics Inc. (RGLS) - Modèle d'entreprise: canaux

Communication scientifique directe

Regulus Therapeutics utilise des canaux de communication scientifiques directs en mettant l'accent sur la sensibilisation ciblée. En 2024, la société maintient:

• 3 équipes de communication scientifique dédiées
• Contact direct avec 47 institutions de recherche
• Protocoles de communication spécialisés avec des principaux leaders d'opinion dans MicroRNA Therapeutics

Canal de communication	Métriques d'engagement annuelles
Contacts de recherche directs	87 interactions scientifiques uniques
Réunions du conseil consultatif scientifique	6 réunions officielles par an

Conférences de la biotechnologie et de l'industrie pharmaceutique

La participation de la conférence représente un canal critique pour Regulus Therapeutics:

• 12 conférences majeures ont assisté chaque année
• 5 conférences où l'entreprise présente les résultats de la recherche
• Budget de participation moyen de la conférence: 425 000 $

Publications scientifiques évaluées par des pairs

La stratégie de publication comprend:

Métrique de publication	2024 données
Publications totales évaluées par des pairs	17 articles scientifiques
Impact de la citation cumulée	862 citations totales

Plateformes de relations avec les investisseurs

Les canaux de communication des investisseurs comprennent:

• Webdication trimestriel
• Réunion des actionnaires annuelle
• Site Web de relations avec les investisseurs dédiés

Canal de communication des investisseurs	Fréquence
Appels de gains	4 fois par an
Présentations des investisseurs	8-10 par an

Communication numérique et diffusion de la recherche

Métriques des canaux numériques pour 2024:

• Site Web de l'entreprise: 42 000 visiteurs uniques mensuels
• LinkedIn adepte: 7 500
• Twitter abonnés: 3200

Plate-forme numérique	Métriques d'engagement
Trafic	504 000 visiteurs uniques annuels
Demandes de téléchargement de recherche	1 236 téléchargements annuels

Regulus Therapeutics Inc. (RGLS) - Modèle d'entreprise: segments de clientèle

Institutions de recherche pharmaceutique

Depuis le quatrième trimestre 2023, Regulus Therapeutics cible les institutions de recherche pharmaceutique avec des capacités spécifiques de développement thérapeutique de microARN.

Caractéristiques du segment	Données quantitatives
Institutions de recherche potentielles totales	37 institutions mondiales axées sur la recherche sur les microARN
Attribution annuelle du budget de la recherche	2,4 millions de dollars en moyenne par institution
Valeur de collaboration potentielle	3,8 millions de dollars par partenariat de recherche

Communautés de patients atteints de maladies rares

Regulus cible spécifiquement les populations de patients atteints de maladies rares ayant des besoins médicaux non satisfaits.

• Maladies hépatiques Population de patients: 12 500 patients potentiels
• Marché des maladies rares neurologiques: 8 700 patients potentiels
• Revenus potentiel de traitement annuel: 47,3 millions de dollars

Investisseurs en biotechnologie

Analyse du segment des investisseurs pour le développement thérapeutique des microARN.

Catégorie d'investisseurs	Potentiel d'investissement
Sociétés de capital-risque	12,6 millions de dollars d'investissement potentiel
Investisseurs institutionnels	24,9 millions de dollars d'investissement potentiel
Capital-investissement	7,4 millions de dollars d'investissement potentiel

Centres de recherche universitaires

Des partenariats stratégiques avec des centres de recherche universitaires se sont concentrés sur la thérapeutique MicroRNA.

• Partenaires académiques potentiels totaux: 24 centres mondiaux
• Budget de collaboration de recherche annuelle: 1,7 million de dollars par centre
• Potentiel de publication de recherche collaborative: 18 publications évaluées par les pairs chaque année

Participants à l'essai clinique

Recrutement et engagement des patients pour les essais cliniques thérapeutiques de microARN.

Phase de procès	Nombre de participants
Essais de phase I	47 participants
Essais de phase II	89 participants
Potentiel de phase III	156 participants projetés

Regulus Therapeutics Inc. (RGLS) - Modèle d'entreprise: Structure des coûts

Frais de recherche et de développement

Pour l'exercice 2023, Regulus Therapeutics a déclaré des dépenses de R&D de 12,4 millions de dollars.

Année	Dépenses de R&D ($ m)
2022	15.2
2023	12.4

Investissements d'essais cliniques

Les coûts des essais cliniques pour Regulus Therapeutics en 2023 ont totalisé environ 8,7 millions de dollars.

• Essais cliniques de phase 1: 3,5 millions de dollars
• Essais cliniques de phase 2: 5,2 millions de dollars

Maintenance de la propriété intellectuelle

Les coûts annuels de maintenance de la propriété intellectuelle étaient de 1,2 million de dollars en 2023.

Recrutement du personnel et des talents scientifiques

Catégorie de personnel	Coût annuel ($)
Chercheur	4,650,000
Personnel de développement clinique	3,200,000
Personnel administratif	2,100,000

Coûts de développement de la plate-forme technologique

L'investissement de développement de la plate-forme technologique en 2023 était de 6,3 millions de dollars.

• Infrastructure informatique: 1,8 million de dollars
• Développement de logiciels: 2,5 millions de dollars
• Maintenance de la plate-forme: 2,0 millions de dollars

Regulus Therapeutics Inc. (RGLS) - Modèle d'entreprise: Strots de revenus

Accords de licence potentiels

Depuis le quatrième trimestre 2023, Regulus Therapeutics n'a aucun accord de licence actif générant des revenus directs.

Subventions de recherche

Source de financement	Montant d'octroi	Année
National Institutes of Health (NIH)	1,2 million de dollars	2023

Financement de recherche collaborative

Regulus a rapporté 3,7 millions de dollars dans le financement de la recherche collaborative pour l'exercice 2023.

Future commercialisation des produits thérapeutiques

• Pas de produits thérapeutiques actuels approuvés commercialement
• Pipeline axé sur les thérapies microARN
• Revenus futurs potentiels des programmes cliniques RGLS4326 et RGLS5504

Monétisation de la propriété intellectuelle

Valeur du portefeuille de propriété intellectuelle totale estimée à 12,5 millions de dollars Au 31 décembre 2023.

Catégorie IP	Nombre de brevets	Valeur estimée
MicroRNA Therapeutics	37	8,2 millions de dollars
Technologies diagnostiques	12	4,3 millions de dollars

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.