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.
8
Pipeline Programs↗
Distinct drugs in development (Preclinical–Phase 3)
6
Indications↗
Distinct diseases across all programs
125
Clinical Trials↗
All sponsored trials (all statuses)
8
Orange Book Patents
Across 2 drugs with upcoming expirations
Pipeline
Pipeline Snapshot
By Phase
8 drugs in development
Phase 34
Phase 21
Phase 12
Preclinical1
By Therapeutic Area
top 1
Neurology
338%
R&D Momentum
60/100
Pipeline Velocity
22/100
Trial Completion Rate
92/100
Pipeline Concentration
53/100
Pipeline Freshness
70/100
Activity
Recent Activity
Last 14 days
Sep 19, 2026
Active, not recruiting
A Phase 1/2a Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION717 in Patients With Prion Disease
Sep 15, 2026
Completed
The CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-study
Sep 12, 2026
Completed
CARDIO-TTRansform ION-682884-CS2-Scintigraphy Sub-study
Earlier
Sep 5, 2026TrialDonidalorsen Expanded Access Program for Patients With Hereditary Angioedema (HAE)
Sep 4, 2026TrialA Phase 2, Open-label Trial Evaluating the Safety, Tolerability and Efficacy of ION775 in Adults With Hypertriglyceridemia
Sep 4, 2026TrialA Phase 3 Global, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ION-682884 in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
Sep 4, 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
Sep 3, 2026TrialAn Open-Label Study of Donidalorsen in Pediatric Patients Age 2 to Less Than 12 Years Old With Hereditary Angioedema
4 more events
Aug 29, 2026TrialPhase 3 Study of the Efficacy and Safety of ION582 in Children and Adults With Angelman Syndrome
Aug 25, 2026TrialPhase 3 Study of the Efficacy and Safety of ION582 in Children and Adults With Angelman Syndrome Due to Paternal Uniparental Disomy or Imprinting Defects
Aug 22, 2026TrialA Phase 1-3, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION373 in Patients With Alexander Disease
Aug 21, 2026TrialHALOS: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients With Angelman Syndrome
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
NeurologyOtherMetabolic & EndocrineImmunologyRare Disease
Pipeline
Drug Pipeline
Indication
Preclinical
Phase 1
Phase 2
Phase 3
Approved
Neurology
Prion Disease
ION717Preclinical
Unknown / Other
Alexander Disease
zilganersenPhase 3
Angelman Syndrome
ION582Phase 3
obudanersenPhase 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)