Ionis Pharmaceuticals

Name

Ionis Pharmaceuticals

Legal name

Ionis Pharmaceuticals, Inc.

Website

https://ionispharma.com

Company type

Public

Founded year

1989

Operating status

Operating

Headcount range

1,001–5,000 employees

Ownership category

akta.pro rank

Product category

RNA-Targeted Antisense Oligonucleotide Therapeutics

NAICS

Pharmaceutical Preparation Manufacturing (325412), Biological Product (except Diagnostic) Manufacturing (325414), Research and Development in Biotechnology (except Nanobiotechnology) (541714)

SIC

Pharmaceutical Preparations (2834), Biological Products, (No Disgnostic Substances) (2836)

akta.pro primary industry

Antisense Oligonucleotides (ASO) Therapeutics (splice modulation/knockdown) (HLAAADAC)

akta.pro secondary industries

RNA Manufacturing & CMC (oligo synthesis, IVT, purification, formulation, QC) (HLAAADAF), RNA Therapeutics Clinical Development (trial design, endpoints, patient stratification) (HLAAADAH), RNA Therapeutics Platforms & Design (sequence design, target ID/validation, AI/bioinformatics) (HLAAADAG), RNA Chemistry & Oligonucleotide Engineering (backbones, modifications, stereochemistry) (HLAAADAE), RNA Therapeutics Safety & Immunogenicity (innate immune activation, tox, off-target) (HLAAADAI), RNA Therapeutics Regulatory, Quality & Pharmacovigilance (GxP, comparability, post-market) (HLAAADAJ)

Keywords

• Antisense oligonucleotide therapeutics
• RNA-targeted medicines
• Rare disease drugs
• Biopharmaceutical development
• Oligonucleotide manufacturing

Headquarters

HQ city

Carlsbad

HQ country

United States

HQ region

North America

Offices1 record

Markets served

GTM type

B2B

Offering type

Hardware or Manufacturing

Cost components

Technology or R&D, Personnel, Supply Chain, Marketing or Sales, Operations

Revenue model

1. Commercial Product Sales: Revenue from direct sales of wholly owned commercial products including TRYNGOLZA for FCS and sHTG, and DAWNZERA for hereditary angioedema. Product sales growing significantly with Q1 2026 showing strong uptake.
2. Licensing Royalties: Tiered royalties from partnered products including 10-12% on GSK's bepirovirsen net sales and royalties up to mid-20% range from Recordati's zilganersen sales outside US. Existing royalty streams from SPINRAZA, TEGSEDI, and WAYLIVRA.
3. Milestone Payments: Upfront, development, regulatory, and commercial milestone payments from partnership agreements including $30M from Recordati for zilganersen, $15M from GSK for bepirovirsen EMA acceptance, and $80M combined from Roche and GSK in Q1 2026.
4. Strategic Financing: Capital raised through convertible senior notes offerings including $700M in 2025 for 2030 maturity, $500M in 2023 for 2028 maturity, and $500.3M equity offering in September 2024.

Pricing tiers

Go-to-market motion2 records

Distribution channels6 records

Marketing channels6 records

Core offering

Ionis Pharmaceuticals discovers, develops, and commercializes RNA-targeted antisense oligonucleotide (ASO) therapeutics for serious and rare diseases. The company sells FDA-approved products including TRYNGOLZA (olezarsen) for severe hypertriglyceridemia and Dawnzera (donidalorsen) for hereditary angioedema, while licensing pipeline candidates to global pharmaceutical partners (GSK, Biogen, Ono, Recordati, Sobi) for development and ex-US commercialization in exchange for royalties and milestone payments.

Product overview

Ionis Pharmaceuticals is a biotechnology company focused on RNA-targeted medicines. The company operates a diversified business model combining independently commercialized products with partnered programs. Its core commercial products include TRYNGOLZA (olezarsen) for severe hypertriglyceridemia and Dawnzera (donidalorsen) for hereditary angioedema. The pipeline includes zilganersen for Alexander disease pending FDA approval. Partnered programs include bepirovirsen (with GSK for hepatitis B), salanersen (with Biogen for SMA), and sapablursen (with Ono for polycythemia vera). The company also has earlier-stage programs in cardiovascular, neurological, and rare diseases.

Differentiator

Problem solved

Functional benefit

Brands

• Tryngolza: RNA-targeted medicine for severe hypertriglyceridemia (sHTG) and familial chylomicronemia syndrome (FCS)
• Dawnzera
• Zilganersen

Products and services

• TRYNGOLZA (olezarsen) FDA-approved antisense oligonucleotide therapy for adults with severe hypertriglyceridemia (sHTG) and Familial Chylomicronemia Syndrome (FCS), reducing triglycerides and the risk of acute pancreatitis. Self-administered monthly via autoinjector in 50mg or 80mg doses.
• Dawnzera (donidalorsen) FDA-approved antisense oligonucleotide therapy for hereditary angioedema (HAE), Ionis' second independent commercial product launch as part of the company's transition to a full-ownership strategy.
• Zilganersen Investigational antisense oligonucleotide for Alexander disease (AxD), a rare progressive neurodegenerative condition. Under FDA Priority Review with PDUFA date of September 22, 2026; Phase 3 data showed 33.3% improvement in gait speed. Ionis retains US commercial rights while Recordati holds ex-US rights.
• Bepirovirsen Antisense oligonucleotide licensed to GSK for chronic hepatitis B that achieved a 19% functional cure rate in Phase 3 B-Well trials versus 0% with placebo. Under FDA Priority Review with October 2026 PDUFA date and EMA filing accepted. GSK projects peak sales exceeding £2 billion.
• Salanersen (BIIB115) Antisense oligonucleotide for spinal muscular atrophy, licensed to Biogen for global development and commercialization. Received FDA Breakthrough Therapy Designation for SMA.
• Sapablursen Antisense oligonucleotide for polycythemia vera licensed to Ono Pharmaceutical. In pivotal Phase 3 INTREPID study with Fast Track, Orphan Drug, and Breakthrough Therapy designations; first patient dosed June 2026.

Quantifiable outcome

• 91% reduction in acute pancreatitis events with Tryngolza treatment at 12 months
• +4 more outcomes

Named customers2 records

Segments6 records

Ideal customer profiles2 records

Technology focussed No

API detail

Has API

No

API docs

API detail

Core technology

AI maturity

App detail

Feature3 records

Partnerships

Nine partnerships are on record, tiered core, major and minor.

• RecordaticoreOEM/ Whitelabel/ Licensing Partner · 25 June 2026Ionis granted Recordati exclusive rights to develop and commercialize zilganersen for Alexander disease (AxD) in all countries outside the United States. Ionis receives $30 million upfront payment plus additional milestone payments and tiered royalties up to mid-20% range on annual net sales. Recordati brings deep rare disease expertise and handles international regulatory filings and commercialization. Ionis retains US commercial responsibility and global development leadership. FDA decision expected September 22, 2026.
• NovartismajorStrategic or Co-development Partner · 1 June 2026Novartis partnered with Ionis on a lipoprotein(a) candidate program. Phase 3 clinical readout expected in 2026. Lipoprotein(a) is a cardiovascular risk factor described as 'diabolical molecule' nearing regulatory approval with potential to surpass statins in usage.
• AstraZenecamajorStrategic or Co-development Partner · 1 June 2026AstraZeneca partnered with Ionis on ATTR cardiomyopathy program. Phase 3 clinical readout expected in 2026. ATTR amyloidosis market projected to reach $6.3 billion by 2035 with 11.6% CAGR.
• RocheminorStrategic or Co-development Partner · 29 April 2026R&D collaboration generating milestone payments. Ionis received approximately $95 million in milestone payments from multiple partnerships including Roche and GSK in Q1 2026.
• Ono PharmaceuticalmajorOEM/ Whitelabel/ Licensing Partner · 1 March 2025Ono Pharmaceutical acquired exclusive global rights for development and commercialization of sapablursen for polycythemia vera from Ionis in March 2025. Sapablursen received Fast Track, Orphan Drug, and Breakthrough Therapy designations from FDA. Phase 3 INTREPID study initiated with first patient dosed June 2026.
• SobimajorChannel Partner/ Reseller/ Distributor · 6 June 2023Sobi holds rights to Tryngolza (olezarsen) commercialization in Europe. EMA validated indication extension application for severe hypertriglyceridemia in March 2026. Sobi presented pooled Phase 3 analysis at EAS 2026 Congress showing 85% reduction in acute pancreatitis and 66% triglyceride reduction.
• KardiganminorOEM/ Whitelabel/ Licensing Partner · 1 January 2023Kardigan licensed tonlamarsen (antisense oligonucleotide targeting angiotensinogen) from Ionis for hypertension treatment. Kardigan reported positive Phase 2 KARDINAL trial data at ACC.26 showing statistically significant 67% mean reduction in plasma angiotensinogen levels.
• BiogencoreStrategic or Co-development Partner · 10 January 2022Biogen licensed global development and commercialization rights from Ionis for multiple antisense programs including salanersen (BIIB115) for spinal muscular atrophy, diranersen (BIIB080) for Alzheimer's disease, and other neurological candidates. Salanersen received FDA Breakthrough Therapy Designation for SMA. Biogen advancing diranersen to registrational development for Alzheimer's based on Phase 2 CELIA trial showing tau pathology reduction and cognitive benefit.
• GSK (GlaxoSmithKline)coreStrategic or Co-development Partner · 12 August 2019GSK licensed bepirovirsen from Ionis in 2019 in a deal worth up to $260 million. GSK holds global rights for development and commercialization of the chronic hepatitis B treatment. Ionis receives tiered royalties of 10-12% on net sales. FDA granted Priority Review with PDUFA date of October 26, 2026. Phase 3 B-Well trials demonstrated 19% functional cure rate versus 0% with standard of care. EMA accepted regulatory filing. GSK projects peak sales exceeding £2 billion.

Scale indicators17 records

Recent moves6 records

Expansion highlights6 records

Direct peers

• BridgeBio Pharma: Clinical-stage biotech focused on genetic diseases with overlapping ATTR-CM cardiology programs. Comparable as a mid-cap rare disease biotech pursuing targeted therapeutics for genetically defined patient populations.
• Alnylam Pharmaceuticals: Leading RNA-targeted therapeutics company developing siRNA-based medicines for rare and prevalent diseases. Closest peer to Ionis as both pioneered RNA-targeted drug platforms, targeting similar hepatic and rare disease indications with comparable licensing/commercialization strategies.
• Arrowhead Pharmaceuticals: Direct competitor in RNA-targeted therapeutics with a competing sHTG therapy referenced in Ionis's pricing strategy. Develops RNAi-based medicines for cardiometabolic, liver, and rare diseases using TRiM platform, overlapping with Ionis's pipeline and target indications.
• Wave Life Sciences: Clinical-stage RNA therapeutics company developing stereopure oligonucleotides for neurological and rare diseases. Direct peer in oligonucleotide chemistry with overlapping CNS programs and similar partnering-led commercialization model.
• Sarepta Therapeutics: RNA-targeted therapeutics company specializing in treatments for Duchenne muscular dystrophy and rare neuromuscular diseases. Peer in developing RNA-modality medicines for rare genetic conditions with similar regulatory and pricing dynamics.

Broad incumbents

• Biogen: Large biotech with deep neurology focus and Ionis's commercial partner for salanersen (SMA) and diranersen (Alzheimer's). Comparable as a major neurology biotech and as Ionis's established commercialization partner for multiple antisense programs.
• BioNTech: Global RNA therapeutics leader with mRNA platform spanning oncology, infectious disease, and rare diseases. Comparable as a major RNA-modality biotech pursuing both wholly owned and partnered commercial strategies across multiple therapeutic areas.
• Regeneron Pharmaceuticals: Large biotech with RNA/antisense and gene editing programs through Regeneron Genetic Medicines. Comparable as a diversified biotech leveraging genetic insights for therapeutic development, including ophthalmology and cardiometabolic indications.
• Moderna: Major mRNA therapeutics company with broad pipeline including vaccines, oncology, and rare disease programs. RNA-modality peer though focused on mRNA versus Ionis's ASO platform; both target expansion of RNA medicines into prevalent and rare diseases.

Emerging players

• ProQR Therapeutics: Emerging RNA editing company developing Axiomer platform for rare diseases. Niche peer in RNA-modality therapeutics focused on liver and CNS indications with similar platform-based approach to genetic medicines.

Market position

Strengths5 records

Weaknesses5 records

Competitive moat4 records

Key risks6 records

Key highlights7 records

Customer concentration

• Xx.com/ionispharma
• Facebookfacebook.com/ionispharma
• LinkedInlinkedin.com/company/ionis-pharmaceuticals
• Instagraminstagram.com/ionis_pharma

Revenue estimate

Valuation estimate

Number of profiles

Profiles13 records

Funding overview

Funding rounds5 records

Investors

Funding detail is available on the Subscription and Enterprise plan.Contact sales →

M&A1 record

Investments4 records

M&A and investment is available on the Subscription and Enterprise plan.Contact sales →

Frequently asked questions about Ionis Pharmaceuticals