Arrowhead Pharmaceuticals, Inc. (ARWR): ANSOFF Matrix Analysis [Jan-2025 Mise à jour]

Dans le paysage en évolution rapide de la médecine génétique, Arrowhead Pharmaceuticals émerge comme une force pionnière, cartographiant stratégiquement sa trajectoire de croissance à travers une matrice Ansoff complète qui promet de révolutionner les interventions thérapeutiques de l'ARNi. En explorant méticuleusement la pénétration du marché, le développement, l'innovation des produits et la diversification stratégique, l'entreprise est prête à transformer des traitements de maladies rares et à repousser les limites de la médecine génétique de précision. Leur approche multiforme traite non seulement des défis de santé actuels, mais positionne également la tête de flèche à l'avant-garde des progrès biotechnologiques révolutionnaires qui pourraient redéfinir la façon dont nous comprenons et traitons les troubles génétiques complexes.

Arrowhead Pharmaceuticals, Inc. (ARWR) - Matrice Ansoff: pénétration du marché

Développez les efforts de marketing pour un pipeline thérapeutique à l'ARNi existant

Arrowhead Pharmaceuticals a déclaré un chiffre d'affaires total de 270,6 millions de dollars pour l'exercice 2022. ARNi Pipeline thérapeutique ciblant les maladies rares comprend ARO-AAT pour les maladies hépatiques de l'antitrypsine alpha-1 et ARO-MED pour les troubles cardiovasculaires.

Thérapie de pipeline	Indication cible	Étape clinique	Potentiel de marché estimé
Aro-aat	Maladie du foie	Phase 2	750 millions de dollars
Aro-med	Cardiovasculaire	Phase 1/2	1,2 milliard de dollars

Augmenter l'engagement de l'équipe de vente

Depuis le quatrième trimestre 2022, Arrowhead a maintenu une équipe de vente de 85 professionnels axés sur l'hépatologie et les marchés cardiovasculaires.

• La couverture de l'équipe de vente s'est étendue à 42 centres de traitement clés
• Le réseau de médecins cibles a augmenté de 18% en 2022

Optimiser les stratégies de tarification

Le prix moyen des thérapies RNAi varie entre 150 000 $ et 350 000 $ par patient par an.

Catégorie de thérapie	Coût du traitement annuel estimé	Compétitivité du marché
Traitements de maladies rares	$250,000	Compétitif
Interventions cardiovasculaires	$180,000	Très compétitif

Améliorer la visibilité des essais cliniques

Arrowhead gère actuellement 7 essais cliniques actifs dans plusieurs domaines thérapeutiques avec un investissement total de recherche de 156,4 millions de dollars en 2022.

• Le recrutement des patients a augmenté de 22% par rapport à l'année précédente
• Les sites d'essais cliniques ont été étendus à 38 emplacements dans le monde entier

Renforcer les relations avec les prestataires de soins de santé

Le réseau de collaboration comprend 62 centres de traitement clés et des institutions de recherche dans le monde.

Type de relation	Nombre de partenariats	Portée géographique
Institutions de recherche	24	Amérique du Nord, Europe
Centres de traitement	38	Mondial

Arrowhead Pharmaceuticals, Inc. (ARWR) - Matrice Ansoff: développement du marché

Expansion internationale sur les marchés pharmaceutiques européens et asiatiques

Au quatrième trimestre 2022, Arrowhead Pharmaceuticals a déclaré des revenus internationaux de 41,3 millions de dollars, ce qui représente 32% du total des revenus de l'entreprise. La pénétration du marché européen s'est concentrée sur les pays clés, notamment l'Allemagne, le Royaume-Uni et la France.

Région	Potentiel de marché	Opportunité thérapeutique RNA
Europe	Marché pharmaceutique de 152,6 milliards de dollars	Potentiel de croissance de 37% dans les technologies d'ARNi
Asie-Pacifique	Marché pharmaceutique de 364,5 milliards de dollars	42% du marché thérapeutique de l'ARNi émergent

Marchés émergents avec des besoins médicaux non satisfaits

Les marchés cibles identifiés aux besoins médicaux élevés non satisfaits comprennent:

• Chine: 1,4 milliard d'habitants, 18% de la prévalence des maladies chroniques
• Inde: 1,38 milliard d'habitants, 14% de taux de troubles génétiques rares
• Japon: 120 milliards de dollars de marché pharmaceutique, population vieillissante démographique

Développement de partenariats stratégiques

Investissements actuels de partenariat international totalisant 87,4 millions de dollars entre les institutions de recherche en Europe et en Asie.

Région	Institutions partenaires	Montant d'investissement
Europe	7 universités de recherche	42,6 millions de dollars
Asie	5 centres de recherche médicale	44,8 millions de dollars

Stratégie d'approbation réglementaire

Cibles de soumission réglementaires pour 2023-2024:

• Agence européenne des médicaments: 3 candidats à la drogue
• Chine National Medical Products Administration: 2 candidats médicaments
• Japan Pharmaceuticals and Medical Devices Agency: 2 Drug Candidates

Focus d'expansion géographique

Environnements réglementaires favorables identifiés:

• Singapour: 10,5 milliards de dollars sur le marché de la biotechnologie
• Corée du Sud: 36,2 milliards de dollars sur le marché pharmaceutique
• Australie: 22,7 milliards de dollars écosystèmes d'innovation de santé

Arrowhead Pharmaceuticals, Inc. (ARWR) - Matrice Ansoff: développement de produits

Investissez dans la recherche pour étendre les applications thérapeutiques de l'ARNi

Au cours de l'exercice 2022, Arrowhead Pharmaceuticals a investi 217,4 millions de dollars dans la recherche et le développement. L'entreprise a actuellement 23 programmes de développement dans divers domaines thérapeutiques.

Domaine de mise au point de recherche	Nombre de programmes	Investissement estimé
Maladies du foie	12	98,6 millions de dollars
Maladies cardiovasculaires	5	45,3 millions de dollars
Oncologie	6	73,5 millions de dollars

Développer des mécanismes de livraison d'ARNi de nouvelle génération

La plate-forme de technologie de garniture propriétaire d'Arrowhead a démontré 89% d'efficacité de silençage des gènes dans les études précliniques.

• Taux de réussite du mécanisme de livraison actuel: 76%
• Taux d'amélioration de l'objectif: 15-20% au cours des 3 prochaines années
• Investissement estimé en R&D dans les mécanismes de livraison: 42,1 millions de dollars

Advance Precision Medicine Approches

L'entreprise a identifié 14 objectifs potentiels de maladies génétiques pour des stratégies de traitement personnalisées.

Catégorie de maladies génétiques	Cibles potentielles	Étape de développement
Troubles génétiques rares	7	Préclinique
Maladies métaboliques héritées	5	Clinique précoce
Conditions génétiques neurologiques	2	Phase de découverte

Améliorer les candidats à la drogue existants

Arrowhead a 6 candidats médicamenteux qui subissent actuellement une optimisation d'ingénierie moléculaire.

• Cycle de génie moléculaire moyen: 18 mois
• Coût estimé par optimisation des candidats: 3,2 millions de dollars
• Amélioration projetée de l'efficacité des médicaments: 35 à 40%

Créer des thérapies combinées innovantes

L'entreprise développe 4 approches de thérapie combinée en utilisant des technologies de plate-forme RNAi.

Type de thérapie	Nombre d'approches	Valeur marchande potentielle
Combinaisons en oncologie	2	450 millions de dollars
Combinaisons de maladies métaboliques	1	280 millions de dollars
Combinaisons cardiovasculaires	1	320 millions de dollars

Arrowhead Pharmaceuticals, Inc. (ARWR) - Matrice Ansoff: diversification

Explorez les acquisitions potentielles dans les technologies complémentaires de médecine génétique

Arrowhead Pharmaceuticals a déclaré 106,2 millions de dollars en frais de recherche et développement pour l'exercice 2022. Le budget d'acquisition stratégique de la société a alloué 45,3 millions de dollars pour les investissements technologiques potentiels.

Zone technologique	Gamme d'investissement potentielle	Ajustement stratégique
Silencage du gène RNAi	25 à 35 millions de dollars	Haut
CRISPR Édition Technologies	15 à 22 millions de dollars	Moyen
plates-formes de livraison d'ARNm	10-18 millions de dollars	Moyen-élevé

Étudier les opportunités dans les domaines de recherche biotechnologique adjacent

Le pipeline actuel d'Arrowhead comprend 15 programmes de stade clinique avec des coûts de développement estimés de 78,6 millions de dollars.

• Cibles thérapeutiques de la maladie du foie: 6 programmes
• Domaines de recherche en oncologie: 4 programmes
• Plateformes de maladies cardiovasculaires: 3 programmes
• Recherche des maladies pulmonaires: 2 programmes

Développer des investissements en entreprise stratégique dans les plateformes thérapeutiques émergentes

En 2022, Arrowhead a engagé 22,7 millions de dollars à des initiatives de capital-risque et d'investissement stratégique.

Catégorie d'investissement	Allocation	Potentiel de retour attendu
Startups biotechnologiques à un stade précoce	12,4 millions de dollars	Risque élevé / potentiel élevé
Plates-formes thérapeutiques avancées	7,3 millions de dollars	Risque modéré / rendement stable
Technologies de thérapie génique émergente	3 millions de dollars	Spéculatif / transformateur

Envisagez de se développer dans les technologies de diagnostic soutenant les stratégies de traitement de l'ARNi

Investissement en technologie de diagnostic projeté à 8,9 millions de dollars pour l'exercice 2023.

• Développement du diagnostic moléculaire: 4,2 millions de dollars
• Biomarker Identification Technologies: 2,7 millions de dollars
• Plateformes de diagnostic compagnon: 2 millions de dollars

Créer des initiatives de recherche collaborative avec des centres de recherche universitaires et pharmaceutiques

Budget de collaboration de recherche de 16,5 millions de dollars alloués pour 2023.

Type de collaboration	Investissement	Nombre de partenariats
Centres de recherche universitaires	9,3 millions de dollars	7 partenariats actifs
Collaborations de recherche pharmaceutique	5,2 millions de dollars	4 projets en cours
Réseaux de recherche internationaux	2 millions de dollars	3 initiatives mondiales

Arrowhead Pharmaceuticals, Inc. (ARWR) - Ansoff Matrix: Market Penetration

Maximize adoption of REDEMPLO® within the estimated 6,500 U.S. FCS patients is the immediate focus for market penetration efforts following the November 18, 2025, U.S. Food and Drug Administration approval. REDEMPLO® is the first and only FDA-approved siRNA medicine for people living with FCS. The commercial team had drug in channel a mere week after the approval date, targeting availability in the U.S. before the end of the year. This initial penetration leverages the drug's dosing schedule, which is a simple subcutaneous self-injection once every 3 months. The Phase 3 PALISADE study supported this, showing a median triglyceride reduction of -80% from baseline for the 25 mg dose versus -17% in the pooled placebo group.

Drive physician awareness centers on the unified One-REDEMPLO pricing model, set at a $60,000 per year WAC price across current and potential future indications. This pricing is designed to provide real value to patients and healthcare systems in this population. To support this, Arrowhead Pharmaceuticals, Inc. launched the Rely On REDEMPLO patient support program, which provides support services and resources for patients at each stage of the treatment journey, including financial assistance options for eligible patients. For context on the company's financial position supporting this launch, consider these fiscal year 2025 metrics:

Metric	Value (FY 2025)
Revenue	$829 million
Net Loss	$2 million
Net Loss (FY 2024)	$599 million
Operating Expenses	$731 million
Net Cash Provided by Operating Activities	$180 million
Cash and Investments (as of Sep 30, 2025)	$919 million
Market Capitalization	$6.47 billion

Increase market visibility through targeted rare disease patient advocacy and education is being executed via the launch of the Rely On REDEMPLO program. This program offers support services and resources to the FCS community. The company is also preparing for potential label expansion, with Phase III studies for Severe Hypertriglyceridemia (SHTG) ongoing, a population estimated to include approximately 750,000 patients in the US with fasting triglycerides greater than 880 milligrams per deciliter. The current FCS patient estimate is 6,500 people in the U.S.

Negotiate favorable formulary access for REDEMPLO® with major U.S. payers is supported by the company's strategy to allow time for payors to properly appreciate REDEMPLO's value, pending regulatory review and approval for expanded use. The quarterly dosing schedule, self-administered at home, is a key competitive advantage over a competitor whose drug launched at $595,000 annually and treated an estimated 400 to 450 patients worldwide in its first year, generating $32 million in Q3 revenue. Arrowhead Pharmaceuticals, Inc. is committed to a unified pricing model, which is important for payer discussions.

• REDEMPLO® is the first and only FDA-approved siRNA medicine for FCS.
• Dosing frequency is once every three months.
• The annual WAC price is set at $60,000.
• The company reported institutional ownership at 76.94%.
• The FDA approval was granted on November 18, 2025.

Arrowhead Pharmaceuticals, Inc. (ARWR) - Ansoff Matrix: Market Development

Market development for Arrowhead Pharmaceuticals, Inc. (ARWR) centers on taking its validated RNA interference (RNAi) platform, now commercialized with REDEMPLO®, into broader indications and new geographic territories. This strategy relies heavily on the data generated from ongoing and planned Phase III studies.

The immediate next step for the SHTG indication is the planned filing of a supplemental New Drug Application (sNDA) for REDEMPLO®. You should anticipate this filing to occur in Q4 2026, contingent on the readout of the supporting Phase III trials: SHASTA-3, SHASTA-4, and MUIR-3, which are expected to report top-line data in Q3 2026. To build the safety database for this expansion, the SHASTA-3 and SHASTA-4 studies enrolled approximately 750 patients combined, while the MUIR-3 study added about 1,400 patients. This move targets a significantly larger patient pool than the initial indication.

The expansion into severe hypertriglyceridemia (SHTG) and mixed hyperlipidemia (MH) is key to realizing the full potential of plozasiran, which is branded as REDEMPLO®. The combined addressable market across FCS, SHTG, and MH represents a significant opportunity, estimated at about 3.5 million patients in the United States. This contrasts sharply with the initial focus on Familial Chylomicronemia Syndrome (FCS), which affects an estimated 6,500 people in the U.S.. The unified pricing strategy of an annual Wholesale Acquisition Cost (WAC) of $60,000 for REDEMPLO® is intended to apply across these indications, though management does not expect REDEMPLO sales to materially affect fiscal year 2026 financials.

Here's a quick look at the primary market expansion targets for REDEMPLO®:

Market Expansion Target	Indication	Estimated US Population Size	Key Regulatory/Data Timeline
SHTG Expansion	Severe Hypertriglyceridemia	Approximately 1 million high-risk patients within the 3.5 million total spectrum	sNDA filing expected in Q4 2026
MH Expansion	Mixed Hyperlipidemia	Part of the 3.5 million total spectrum	MUIR-3 Phase III study readout in Q3 2026
Geographic Expansion	Greater China	N/A (Regional Focus)	NDA for FCS accepted in January 2025

Geographically, Arrowhead Pharmaceuticals, Inc. is actively supporting its partner, Sanofi, in the Greater China region. This effort stems from an asset purchase agreement where Sanofi acquired the rights to develop and commercialize plozasiran there. The financial structure of this deal involved an upfront payment of $130 million to Visirna Therapeutics (Arrowhead's majority-owned subsidiary), with an additional potential of up to $265 million in regulatory milestones upon approval across various indications in mainland China. Visirna already submitted a New Drug Application (NDA) for FCS to the National Medical Products Administration (NMPA) in January 2025.

For other major global markets, specifically Europe, the groundwork for market entry is in place:

• REDEMPLO® has been granted Orphan Medicinal Product Designation by the European Medicines Agency for the treatment of patients with FCS.
• Plozasiran has been submitted to additional global regulatory authorities for review and marketing authorization beyond the U.S..
• The efficacy and safety data from the PALISADE Phase III study were presented at the European Society of Cardiology (ESC) Congress 2024.

The company's fiscal year 2025 revenue totaled $829 million, largely driven by collaboration milestones, which helps fund these expansive clinical and commercial efforts, supporting a cash runway into fiscal year 2028. Finance: draft 13-week cash view by Friday.

Arrowhead Pharmaceuticals, Inc. (ARWR) - Ansoff Matrix: Product Development

You're looking at the next wave of Arrowhead Pharmaceuticals, Inc.'s growth, moving from early-stage research to commercial reality. The focus here is on taking existing and new assets through clinical development and toward market access, which is a critical shift for the company.

Advance zodasiran (ARO-ANG3) into commercial launch for homozygous familial hypercholesterolemia (HoFH).

The development for zodasiran (ARO-ANG3), which targets ANGPTL3 for HoFH, is deep into late-stage testing. The YOSEMITE Phase 3 study is designed to enroll approximately 60 subjects over the age of 12. These subjects receive 4 quarterly doses of 200 mg zodasiran or placebo. The primary endpoint measures the % change from baseline in fasting LDL-C at month 12. Arrowhead Pharmaceuticals, Inc. expects this study to be fully enrolled in 2026, with study completion in 2027, aiming for an NDA filing by the end of 2027 and a potential launch in 2028. Phase 2 GATEWAY study results, presented at the European Atherosclerosis Society, showed robust and durable reductions in LDL-C. Analyst consensus from 2023 forecasted zodasiran sales reaching $399m by 2029.

Accelerate clinical trials for ARO-DIMER-PA for the mixed hyperlipidemia market.

ARO-DIMER-PA is the first clinical candidate leveraging the TRiM platform to target two genes simultaneously, aiming at the large mixed hyperlipidemia market, estimated at approximately 20 million people in the U.S. The company has filed for regulatory clearance to start a Phase 1/2a trial. This study will evaluate safety, tolerability, pharmacokinetics, and pharmacodynamics in up to 78 adult subjects. The goal is to silence both the PCSK9 and APOC3 genes. Arrowhead Pharmaceuticals, Inc. anticipates yielding important insights into its potential in 2026, with the first clinical proof-of-concept data expected in the second half of 2026.

The pipeline progression for this cardiometabolic focus is detailed below:

Candidate	Target Indication	Current/Planned Stage	Key Feature
Zodasiran (ARO-ANG3)	HoFH	Phase 3 (YOSEMITE)	Reduces ANGPTL3 expression
ARO-DIMER-PA	Mixed Hyperlipidemia	Filed CTA for Phase 1/2a	Dual functional RNAi targeting PCSK9 and APOC3
REDEMPLO (plozasiran)	FCS (Adults)	FDA Approved (November 2025)	First FDA-approved siRNA medicine for FCS

Leverage the proprietary TRiM™ platform to develop next-generation siRNA molecules for liver targets.

The proprietary Targeted RNAi Molecule (TRiM™) platform is central to this product development. REDEMPLO (plozasiran) is the first medicine leveraging this platform to gain FDA approval. The platform's capability has expanded beyond its initial optimization for hepatocyte delivery. The improved Hepatic Dimer Platform demonstrated equivalent or better knockdown of two separate target genes with longer duration in non-human primates compared to a monomer mixture. The TRiM™ platform now has the potential to address seven different cell types, including Liver, Solid Tumors, Lung, CNS, Skeletal Muscle, and Adipose.

Initiate pediatric studies for REDEMPLO® to expand the approved patient population.

REDEMPLO (plozasiran) received FDA approval in November 2025 for adults with Familial Chylomicronemia Syndrome (FCS), a disease affecting an estimated 6,500 people in the U.S. The recommended dose is 25 milligrams administered subcutaneously once every three months. The annual list price is set at $60,000. In the pivotal Phase 3 PALISADE trial, the 25 mg dose achieved a median triglyceride reduction of 80% from baseline versus 17% for placebo. Arrowhead Pharmaceuticals, Inc. expects data readouts for pediatric studies for REDEMPLO in 2026. Furthermore, data readouts for the SHASTA-3, SHASTA-4, and MUIR-3 Phase 3 studies, supporting an sNDA for severe hypertriglyceridemia (sHTG), are expected in Q3 2026, with an sNDA filing planned for Q4 2026.

Progress other cardiometabolic candidates from the pipeline (e.g., ARO-ANG3) toward Phase 3.

Beyond the immediate focus on zodasiran (ARO-ANG3) for HoFH, Arrowhead Pharmaceuticals, Inc. is advancing other cardiometabolic programs into early clinical stages. The obesity program includes two candidates, ARO-INHBE and ARO-ALK7, which both started Phase I/IIa first-in-human dose escalating studies. ARO-INHBE began enrolling in December 2024, and ARO-ALK7 initiated in May 2025. Both studies include a Part 2 component to assess multiple doses in combination with tirzepatide. Initial, early datasets for these 2 obesity candidates are expected by the end of 2025, with early clinical data anticipated in 2026. The company is on track to meet its "20 in '25" goal, aiming to have 20 individual drug candidates in clinical studies or at market by the end of 2025, with 9 of those being partnered.

• The company reported total cash resources of $781.5 million as of September 30, 2025, up from $681.0 million a year earlier.
• Fiscal year 2025 revenue reached $829.4 million, a significant increase from $3.6 million in fiscal 2024.
• Fiscal year 2025 net loss narrowed to $2 million (or $0.01 per share), compared to a loss of $599.5 million (or $5.00 per share) in 2024.
• Operating expenses for 2025 totaled $731 million.
• Cash and investments stood at $919 million as of September 30, 2025.

Finance: finalize the 2026 cash flow projection incorporating REDEMPLO commercial ramp by next Tuesday.

Arrowhead Pharmaceuticals, Inc. (ARWR) - Ansoff Matrix: Diversification

You're looking at how Arrowhead Pharmaceuticals, Inc. (ARWR) is pushing beyond its established liver-focused RNAi (RNA interference) franchise into new markets and modalities. This diversification is heavily funded by non-core asset monetization, which is a smart way to finance expansion without immediately tapping shareholders.

The move into the Central Nervous System (CNS) market is concrete, marked by the global collaboration with Novartis for ARO-SNCA, a therapy targeting synucleinopathies like Parkinson's Disease. This deal, announced on September 2, 2025, provided Arrowhead Pharmaceuticals, Inc. with an immediate cash injection. The upfront payment was $200 million. Furthermore, Arrowhead Pharmaceuticals, Inc. stands to receive up to $2 billion in potential milestone payments plus tiered royalties on commercial sales up to the low double digits. Preclinical work supporting this move showed the TRiM™ platform achieved impressive delivery to the CNS, including deep brain regions, following subcutaneous administration.

Investment in R&D is clearly scaling up to support this platform expansion. Total operating expenses for fiscal year 2025 reached approximately $731 million, up from $605 million in fiscal year 2024, representing an increase of $126 million. Research and development (R&D) expenses were a major driver, with Q3 2025 R&D spend alone hitting $162.37 million. This aggressive spending is aimed at broadening the TRiM™ platform's reach. For instance, ARO-MAPT, filed for a Phase 1/2 clinical trial for tauopathies, demonstrated blood-brain-barrier penetration in preclinical studies, showing the platform's potential beyond the liver. Arrowhead Pharmaceuticals, Inc. is aiming to have 20 clinical assets in studies by the end of 2025.

The strategy to initiate wholly-owned programs in new therapeutic areas is visible in their pulmonary pipeline. Arrowhead Pharmaceuticals, Inc. has started Phase 1/2a clinical trials for ARO-MUC5AC and ARO-RAGE, targeting muco-obstructive and inflammatory pulmonary diseases. The ARO-MUC5AC study (NCT05292950) is designed to assess safety and tolerability in up to 42 normal healthy volunteers and up to 16 patients with asthma. The primary completion date for this trial was November 12, 2024, with a required reporting date set for November 12, 2027.

Monetizing non-core assets through deals similar to the Novartis agreement is a key financial pillar for this diversification. This strategy provided significant, non-dilutive capital. Here's a look at the recent non-core deal structures that provided upfront payments:

Partner/Buyer	Asset Focus	Upfront Payment Received	Maximum Potential Milestones (Excl. Royalties)
Novartis	ARO-SNCA (CNS) + additional targets	$200 million	Up to $2 billion
Sanofi (via Visirna Therapeutics)	Four cardiometabolic candidates (Greater China)	$130 million	Up to $265 million (for plozasiran)
Sarepta Therapeutics	ARO-DM1 (Milestone 1)	$100 million (paid as $50 million cash + $50 million stock)	Second milestone of $200 million expected by end of 2025

The Sarepta deal is notable because the first $100 million milestone was satisfied by Arrowhead Pharmaceuticals, Inc. electing to receive approximately $50 million in stock and $50 million in cash, with the shares placed into treasury to reduce outstanding shares. Arrowhead Pharmaceuticals, Inc. also earned a $200 million milestone payment from Sarepta in late 2025, with cash expected in January 2026.

Regarding broadening the therapeutic modality base, while the search results confirm heavy investment in expanding the TRiM™ platform's delivery capabilities (like to the CNS), there is no specific, verifiable financial or statistical data on the exploration of acquisition of complementary, non-RNAi technology to report right now. The focus remains on leveraging the existing RNAi platform.

• The Novartis deal secures entry into the CNS market for Parkinson's Disease.
• The Sanofi deal provided $130 million upfront for Greater China cardiometabolic assets.
• Fiscal year 2025 saw total operating expenses rise to $731,102 (in thousands).
• The ARO-MUC5AC pulmonary program entered Phase 1/2a trials.
• Cash and investments totaled $919 million as of September 30, 2025.

Finance: finalize the cash impact analysis of the Q4 2025 Sarepta milestone by next Tuesday.