Ionis Pharmaceuticals
- Company typePublic
- Founded1989
- HeadquartersCarlsbad, United States
- Headcount1,001–5,000
- GTM typeB2B
- OfferingHardware or Manufacturing
Ionis Pharmaceuticals firmographics
Firmographics- 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
Ionis Pharmaceuticals industry classification
Industry- 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
Where Ionis Pharmaceuticals is headquartered
LocationHeadquarters
- HQ city
- Carlsbad
- HQ country
- United States
- HQ region
- North America
Offices1 record
Markets served
Ionis Pharmaceuticals business model
Business model- GTM type
- B2B
- Offering type
- Hardware or Manufacturing
- Cost components
- Technology or R&D, Personnel, Supply Chain, Marketing or Sales, Operations
Revenue model
- 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.
- 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.
- 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.
- 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
| Model | Billing | Price |
|---|---|---|
| Unit Pricing | Annual | TRYNGOLZA (olezarsen) for severe hypertriglyceridemia: $40,000 annual list price |
| Unit Pricing | Annual | TRYNGOLZA (olezarsen) for Familial Chylomicronemia Syndrome: $595,000 annual list price |
Go-to-market motion2 records
Distribution channels6 records
Marketing channels6 records
Ionis Pharmaceuticals product offering
Product offeringCore 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
Companies that use Ionis Pharmaceuticals
Customer profileNamed customers2 records
Segments6 records
Ideal customer profiles2 records
Ionis Pharmaceuticals technology and API
TechnologyTechnology focussed No
API detail
- Has API
- No
- API docs
- API detail
Core technology
AI maturity
App detail
Feature3 records
Ionis Pharmaceuticals partnerships and signals
Strategic signalPartnerships
Nine partnerships are on record, tiered core, major and minor.
- RecordaticoreIonis 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.
- NovartismajorNovartis 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.
- AstraZenecamajorAstraZeneca 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.
- RocheminorR&D collaboration generating milestone payments. Ionis received approximately $95 million in milestone payments from multiple partnerships including Roche and GSK in Q1 2026.
- Ono PharmaceuticalmajorOno 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.
- SobimajorSobi 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.
- KardiganminorKardigan 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.
- BiogencoreBiogen 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)coreGSK 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
Ionis Pharmaceuticals competitors and assessment
Company assessmentDirect 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
Ionis Pharmaceuticals social profiles
Digital presenceIonis Pharmaceuticals financial estimates
Financial estimateRevenue estimate
Valuation estimate
Ionis Pharmaceuticals leadership team
Management profileNumber of profiles
Profiles13 records
Ionis Pharmaceuticals funding detail
Funding detailFunding overview
Funding rounds5 records
Investors
Funding detail is available on the Subscription and Enterprise plan.Contact sales →
Ionis Pharmaceuticals M&A and investment
M&A and investmentM&A1 record
Investments4 records
M&A and investment is available on the Subscription and Enterprise plan.Contact sales →
Frequently asked questions about Ionis Pharmaceuticals
What does Ionis Pharmaceuticals do?
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.
Is Ionis Pharmaceuticals a public or private company?
Ionis Pharmaceuticals is a public company. It is classified as public and is currently operating.
When was Ionis Pharmaceuticals founded?
Ionis Pharmaceuticals was founded in 1989. It employs 1,001 to 5,000 people.
Where is Ionis Pharmaceuticals based?
Ionis Pharmaceuticals is headquartered in Carlsbad, United States, in the North America region.
How does Ionis Pharmaceuticals make money?
Four revenue lines are on record. Commercial Product Sales are the primary driver. The others are licensing Royalties, milestone Payments and strategic Financing.
Who are Ionis Pharmaceuticals's main competitors?
Direct peers on record are BridgeBio Pharma, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, Wave Life Sciences and Sarepta Therapeutics. Broad incumbents are Biogen, BioNTech, Regeneron Pharmaceuticals and Moderna. ProQR Therapeutics is listed as an emerging player.
Does Ionis Pharmaceuticals have an API?
No public API is recorded for Ionis Pharmaceuticals.
What industry is Ionis Pharmaceuticals in?
Ionis Pharmaceuticals's product category is RNA-Targeted Antisense Oligonucleotide Therapeutics. Its primary akta.pro industry code is HLAAADAC, Antisense Oligonucleotides (ASO) Therapeutics (splice modulation/knockdown), with a secondary code of HLAAADAF, RNA Manufacturing & CMC (oligo synthesis, IVT, purification, formulation, QC). Its NAICS code is 325412 and its SIC code is 2834.