Ionis Pharmaceuticals

Ionis Pharmaceuticals(IONS)

CARLSBAD, CA
Pharmaceutical
WebsiteLinkedInCareers Page

Focus: RNA

Ionis Pharmaceuticals is a life sciences company focused on RNA.

NeurologyGene Therapy
Funding Stage
PUBLIC
Open Jobs
0

Products & Portfolio (2)

DAWNZERA (AUTOINJECTOR)
donidalorsen
Launch
RNASUBCUTANEOUS · SOLUTION
ASO‑GalNAc conjugate that causes ribonuclease H1 (RNase H1)‑mediated degradation of PKK mRNA through binding to PKK mRNA, which results in reduced production of PKK protein. PKK is a pro‑enzyme for plasma kallikrein, which results in the release of bradykinin, a potent vasodilator causing swelling and pain in HAE. In patients with HAE, C1‑inhibitor (C1‑INH) deficiency or dysfunction leads to excessive plasma kallikrein activity, bradykinin generation, and angioedema attacks. Donidalorsen lowers PKK concentration, preventing excessive bradykinin production in patients with HAE.
2025
0
TRYNGOLZA (AUTOINJECTOR)
olezarsen sodium
Growth
SUBCUTANEOUS · SOLUTION
2024
0

Pipeline & Clinical Trials

Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl C
Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
N/A
Clinical Trials (1)
NCT06014541Observational Study to Characterize Biomarkers and Disease Progression in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome
N/A
The Myelin Disorders Biorepository Project
Leukodystrophy
N/A
Fomivirsen sodium
Cytomegalovirus Retinitis
N/A
Clinical Trials (1)
NCT00002187A Study of ISIS 2922 in the Treatment of Advanced Cytomegalovirus Retinitis
N/A
N/A
Evaluation of Outcome Metrics in Alexander Disease
Alexander Disease
N/A
Clinical Trials (1)
NCT02714764Evaluation of Outcome Metrics in Alexander Disease
N/A
N/A
Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Peliza
Pelizaeus-Merzbacher Disease
N/A
Clinical Trials (1)
NCT05659901Rocket Study: A Study to Characterize Biomarkers and Disease Progression in Participants With Pelizaeus-Merzbacher Disease
N/A
N/A
Clinical Trials (1)
NCT06360237Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)
N/A
Natural History and Functional Status Study of Patients With Lafora Disease
Lafora Disease
N/A
Clinical Trials (1)
NCT03876522Natural History and Functional Status Study of Patients With Lafora Disease
N/A
Angelman Syndrome Video Assessment (ASVA) Source Material Study
Angelman Syndrome
N/A
Clinical Trials (1)
NCT05637697Angelman Syndrome Video Assessment (ASVA) Source Material Study
N/A
N/A
N/A
Clinical Trials (1)
NCT03400098ATTR Expanded Access Program (EAP) by Ionis
N/A
Fomivirsen sodium
Cytomegalovirus Retinitis
N/A
Clinical Trials (1)
NCT00002355A Comparison of ISIS 2922 Used Immediately or Later in Patients With Cytomegalovirus (CMV) of the Eyes
N/A
N/A
Clinical Trials (1)
NCT06415448Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema
N/A
99m-technetium pyrophosphate scintigraphy
Amyloidosis
N/A
Clinical Trials (1)
NCT05259072carDIo-ttranSSfOrm nucLEar Imaging Study
N/A
N/A
Phase 1
Clinical Trials (1)
NCT02900027Safety, Tolerability, PK, and Pharmacodynamics(PD) of IONIS APOCIII-LRx in Healthy Volunteers With Elevated Triglycerides
Phase 1
Ionis AGT-LRx
Healthy Volunteers
Phase 1
Clinical Trials (1)
NCT03101878Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Ionis AGT-LRx in Healthy Volunteers
Phase 1
Phase 1
Clinical Trials (1)
NCT04934891A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ION547
Phase 1
Phase 1
Clinical Trials (1)
NCT06673069Hero: 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)
Phase 1
IONIS ANGPTL3-LRx
Hypertriglyceridemia
Phase 1
Clinical Trials (1)
NCT02709850Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS ANGPTL3-LRx in Healthy Volunteers With Elevated Triglycerides and Participants With Familial Hypercholesterolemia
Phase 1
ISIS 681257
Renal Impairment
Phase 1
Clinical Trials (1)
NCT03506854Study of ISIS 681257 in Patients With Renal Impairment Compared to Healthy Patients
Phase 1
Phase 1
Clinical Trials (1)
NCT00734240Safety and Tolerability of Single and Multiple Doses of ISIS 353512 in Healthy Volunteers
Phase 1
Phase 1
Clinical Trials (1)
NCT01414881Study to Assess the Effects of Mipomersen on Lipid and Lipoprotein Metabolism in Healthy Subjects
Phase 1
Phase 1
Clinical Trials (1)
NCT00836225Safety, Tolerability and Activity Study of Multiple Doses of ISIS-SGLT2Rx in Healthy Volunteers
Phase 1
Phase 1
Clinical Trials (1)
NCT04659096A Study of ION537 in Patients With Molecularly Selected Advanced Solid Tumors
Phase 1
Phase 1
Clinical Trials (1)
NCT05337878A Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pelacarsen (ISIS 681257) in Healthy Japanese Participants
Phase 1
Phase 1
Clinical Trials (1)
NCT05579860A Study Comparing Two Subcutaneous Formulations: Vial and Autoinjector (AI) With Olezarsen, at Two Dose Levels, in Healthy Adult Participants
Phase 1
Phase 1
Clinical Trials (1)
NCT04731623A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION904
Phase 1
Phase 1
Clinical Trials (1)
NCT03263507Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen (IONIS-PKK-LRx) Administered Subcutaneously to Healthy Volunteers
Phase 1
Phase 1
Clinical Trials (1)
NCT03582462A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single and Multiple Doses of IONIS FXI-LRx in up to 84 Healthy Volunteers
Phase 1
warfarin sodium
Healthy
Phase 1
Clinical Trials (1)
NCT01133366A Study to Determine the Effects of Multiple Doses of Mipomersen (200 mg SC) on the Pharmacodynamics and Pharmacokinetics of Single-dose Warfarin
Phase 1
Phase 1
Clinical Trials (1)
NCT00365781Examination of Dermatologic Effects From Subcutaneous Injections of ISIS 113715
Phase 1
Phase 1
Clinical Trials (1)
NCT04302064A Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending and Multiple Doses of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Healthy Japanese Participants
Phase 1
Phase 1
Clinical Trials (1)
NCT06150716Orbit Study: A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Intrathecally Administered ION356 in Participants With Pelizaeus Merzbacher Disease (PMD)
Phase 1
IONIS TMPRSS6-Lrx
Thalassemia
Phase 1
Clinical Trials (1)
NCT03165864Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Healthy Volunteers
Phase 1
Phase 1
Phase 1
Clinical Trials (1)
NCT04165486Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of ION464 Administered to Adults With Multiple System Atrophy (HORIZON)
Phase 1
Phase 1
Clinical Trials (1)
NCT00519727Safety Study of ISIS 325568 in Healthy Volunteers
Phase 1
mipomersen sodium
Healthy
Phase 1
Clinical Trials (1)
NCT01090661A Study to Evaluate the Effect of Mipomersen on Cardiac Repolarization Conducted in Healthy Subjects
Phase 1
Phase 1
Clinical Trials (1)
NCT01299298A Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Single Doses of Mipomersen in Japanese Healthy Volunteers
Phase 1
Phase 1
Clinical Trials (1)
NCT03976349A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease
Phase 1
Phase 1
Clinical Trials (1)
NCT01041222Safety, Tolerability, and Activity Study of ISIS SOD1Rx to Treat Familial Amyotrophic Lateral Sclerosis (ALS) Caused by SOD1 Gene Mutations
Phase 1
Phase 1
Clinical Trials (1)
NCT03186989Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-MAPTRx in Patients With Mild Alzheimer's Disease
Phase 1
Phase 1
Clinical Trials (1)
NCT02910635A Study to Evaluate the Effect of Volanesorsen on Cardiac Repolarization Conducted in Healthy Volunteers
Phase 1
IONIS-ENaCRx
Healthy Subjects
Phase 1
Clinical Trials (1)
NCT03647228A Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of IONIS-ENaCRx in Healthy Volunteers and Patients With Cystic Fibrosis
Phase 1
Phase 1
Clinical Trials (1)
NCT01061814A Study to Evaluate 3 Different Dosing Regimens of Mipomersen Administered Via Subcutaneous Injections to Healthy Volunteers
Phase 1
Phase 1
Clinical Trials (1)
NCT02414594Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS APO(a)-LRx in Healthy Volunteers With Elevated Lipoprotein(a)
Phase 1
Phase 1
Clinical Trials (1)
NCT04398485A Study of ION251 Administered to Patients With Relapsed/Refractory Multiple Myeloma
Phase 1
IONIS-STAT3Rx
Advanced Cancers
Phase 1/2

Open Jobs (0)

No open positions listed yet. Check their careers page directly.

Interview Prep Quick Facts
Founded: 1989
Portfolio: 2 approved products, 138 clinical trials
Top TAs: Metabolic Diseases, Cardiovascular, Neurology
SEC Filings: 2 available
Portfolio Health
Launch1 (50%)
Growth1 (50%)
2 total products
Therapeutic Area Focus
Cardiovascular
12 pipeline
Neurology
11 pipeline
Oncology
6 pipeline
Immunology
6 pipeline
Rare Diseases
4 pipeline
Endocrinology
3 pipeline
Marketed
Pipeline

Financials (FY2025)

Revenue
$227M30%
R&D Spend
$900M(397%)
Net Income
-$366M
Cash
$242M
Source: SEC 10-K Filing