Ionis Pharmaceuticals
NASDAQ:IONSBiotechCarlsbad, California, United States · Est. 1978 · 1,100 employees
Ionis Pharmaceuticals is a biopharmaceutical company that discovers and develops antisense oligonucleotide medicines to treat diseases caused by mutant genes or genes that produce harmful proteins.
3
Pipeline Programs↗
Distinct drugs in development (Preclinical–Phase 3)
3
Indications↗
Distinct diseases across all programs
113
Clinical Trials↗
All sponsored trials (all statuses)
8
Orange Book Patents
Across 2 drugs with upcoming expirations
Pipeline
Pipeline Snapshot
By Phase
3 drugs in development
Phase 32
Phase 11
By Therapeutic Area
top 1
Neurology
267%
R&D Momentum
54/100
Pipeline Velocity
11/100
Trial Completion Rate
92/100
Pipeline Concentration
56/100
Pipeline Freshness
66/100
Strategic Signals
- ↗Active in Neurology but thin pipeline — potential licensing opportunity
Activity
Recent Activity
Last 14 days
Jul 10, 2026
Completed
A Phase 2a, Randomized, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered to Patients With Phlebotomy Dependent Polycythemia Vera (PD-PV)
Jul 1, 2026
Recruiting
Donidalorsen Treatment in Children With Hereditary Angioedema
Earlier
Jun 24, 2026TrialPhase 1-2, Open-Label, Single and Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION337 in Patients With Dravet Syndrome
Jun 24, 2026TrialPhase 3 Study of the Efficacy and Safety of ION582 in Children and Adults With Angelman Syndrome
Mar 3, 2026TrialWAYLIVRA-LT: Volanesorsen Long-Term Extension
Aug 21, 2025FDADawnzera (autoinjector) — DAWNZERA (AUTOINJECTOR)
Dec 20, 2024TrialHero: A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics (PK) and Pharmacodynamics (PD) of ION269 in Participants With Down Syndrome (DS) at Risk for Alzheimer's Disease (AD)
Dec 19, 2024FDATryngolza (autoinjector) — TRYNGOLZA (AUTOINJECTOR)
4 more events
Oct 21, 2024TrialATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS)
Apr 10, 2024TrialOrbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
Jan 4, 2024TrialPrProfile: A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION717
Dec 13, 2022TrialCORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)
Details
Company Info
Company Overview
$5.5B
Mkt Cap
$1.2B
Revenue
—
Cash
CEOKristina Burow
Founded1978
Employees1,100
HQCarlsbad, California, United States
ExchangeNASDAQ:IONS
Websiteionisph.com
LinkedInCompany Page
Therapeutic Focus
NeurologyOncologyMetabolic & EndocrineHematologyCardiovascularInfectious DiseaseImmunologyOtherGastroenterologyNephrologyOphthalmologyRare DiseaseRespiratory
Pipeline
Drug Pipeline
Indication
Preclinical
Phase 1
Phase 2
Phase 3
Approved
Unknown / Other
Neurology
Unknown / Other
Angelman Syndrome
ION582Phase 3
Regulatory
Regulatory Timeline
20241 event
FDADec 19
Tryngolza (autoinjector) — TRYNGOLZA (AUTOINJECTOR)
Application NDA218614 (NDA)
20251 event
FDAAug 21
Dawnzera (autoinjector) — DAWNZERA (AUTOINJECTOR)
Application NDA219407 (NDA)
Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)