Ionis Pharmaceuticals(IONS)

Focus: Ionis is an RNA-focused pharmaceutical company specializing in antisense oligonucleotide (ASO) therapeutics for neurology and gene therapy indications. The company is a public, mature biotech with two marketed products and a deep pipeline of 30+ programs across rare and metabolic diseases.

Neurology Gene Therapy

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Funding Stage

PUBLIC

Open Jobs

About Ionis Pharmaceuticals

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Why Work Here

Data completeness

6/9 — Partial

Company HealthStrong

Pipeline

Early-stage

50 total

LOE Risk

Protected

No near-term cliffs

Hiring

Stable

Revenue

$227M

Key Products

DAWNZERA

Hereditary Angioedema

Launch8.7yr

First-to-market ASO-GalNAc for HAE, recently launched; addresses unmet need in rare genetic disorder with 10-year exclusivity runway.

TRYNGOLZA

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Data Sources

Drug label: DailyMed / FDA
Clinical trials: ClinicalTrials.gov
Patent data: FDA Orange Book
Spending data: CMS Medicare

The information on this page is for informational purposes only and should not be used as a substitute for professional medical advice. Drug information is sourced from FDA, DailyMed, and other government databases. Adverse event data from FAERS does not establish causation. Always consult a healthcare professional for medical decisions.

Pipeline & Clinical Trials

Evaluation of Outcome Metrics in Alexander Disease

Alexander Disease

N/A

Clinical Trials (1)

NCT02714764Evaluation of Outcome Metrics in Alexander Disease

N/A

Donidalorsen

Hereditary Angioedema

N/A

Clinical Trials (1)

NCT06415448Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema

N/A

Scintigraphy scan

Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

N/A

Clinical Trials (1)

NCT06073587The CARDIO-TTRansform Scintigraphy Sub-study

N/A

Inotersen

Amyloidosis, Hereditary

N/A

Clinical Trials (1)

NCT03400098ATTR Expanded Access Program (EAP) by Ionis

N/A

The Myelin Disorders Biorepository Project

Leukodystrophy

N/A

neurofilament light chain

Hereditary Amyloidosis, Transthyretin-Related

N/A

Clinical Trials (1)

NCT05311488Early Detection of Neuropathy in ATTRv

N/A

99m-technetium pyrophosphate scintigraphy

Amyloidosis

N/A

Clinical Trials (1)

NCT05259072carDIo-ttranSSfOrm nucLEar Imaging Study

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

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

Olezarsen

Familial Chylomicronemia Syndrome

N/A

Clinical Trials (2)

NCT07727538A Study of Olezarsen for the Treatment of Familial Chylomicronemia Syndrome (FCS) in Pediatric Participants

Phase 3

NCT06360237Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)

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

Lumbar puncture

Spinocerebellar Ataxia Type 2

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

Fomivirsen sodium

Cytomegalovirus Retinitis

N/A

Clinical Trials (1)

NCT00002187A Study of ISIS 2922 in the Treatment of Advanced Cytomegalovirus Retinitis

N/A

Cardiac MRI

Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR-CM)

N/A

Clinical Trials (1)

NCT06073574CARDIO-TTRansform Magnetic Resonance Imaging (MRI) Sub-study

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

ION251

Relapsed Multiple Myeloma

PHASE_1

Clinical Trials (1)

NCT04398485A Study of ION251 Administered to Patients With Relapsed/Refractory Multiple Myeloma

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

ISIS 388626

Type 2 Diabetes Mellitus

PHASE_1

Clinical Trials (1)

NCT00836225Safety, Tolerability and Activity Study of Multiple Doses of ISIS-SGLT2Rx in Healthy Volunteers

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

Olezarsen

Healthy Participants

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

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

Eplontersen

Healthy Participants

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

mipomersen

Healthy Volunteer

PHASE_1

Clinical Trials (1)

NCT01061814A Study to Evaluate 3 Different Dosing Regimens of Mipomersen Administered Via Subcutaneous Injections to Healthy Volunteers

Phase 1

ISIS 353512

Inflammatory Diseases

PHASE_1

Clinical Trials (1)

NCT00734240Safety and Tolerability of Single and Multiple Doses of ISIS 353512 in Healthy Volunteers

Phase 1

ION356

Pelizaeus-Merzbacher Disease

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

ISIS 325568

Type 2 Diabetes Mellitus

PHASE_1

Clinical Trials (1)

NCT00519727Safety Study of ISIS 325568 in Healthy Volunteers

Phase 1

ION904

Healthy Volunteers

PHASE_1

Clinical Trials (1)

NCT04731623A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ION904

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

APOC-III-L-Rx

Elevated Triglycerides (TG)

PHASE_1

Clinical Trials (1)

NCT02900027Safety, Tolerability, PK, and Pharmacodynamics(PD) of IONIS APOCIII-LRx in Healthy Volunteers With Elevated Triglycerides

Phase 1

IONIS FXI-LRx

Healthy Participants

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

ION269

Alzheimer Disease

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

ISIS 333611

Familial Amyotrophic Lateral Sclerosis

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

ISIS 113715

Type 2 Diabetes Mellitus

PHASE_1

Clinical Trials (1)

NCT00365781Examination of Dermatologic Effects From Subcutaneous Injections of ISIS 113715

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

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

ION547

Healthy Volunteers

PHASE_1

Clinical Trials (1)

NCT04934891A Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of ION547

Phase 1

Volanesorsen

Abnormalities, Cardiovascular

PHASE_1

Clinical Trials (1)

NCT02910635A Study to Evaluate the Effect of Volanesorsen on Cardiac Repolarization Conducted in Healthy Volunteers

Phase 1

ION464

Multiple System Atrophy

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

IONIS-APO

Elevated Lipoprotein(a)

PHASE_1

Clinical Trials (1)

NCT02414594Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS APO(a)-LRx in Healthy Volunteers With Elevated Lipoprotein(a)

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

mipomersen

Healthy Volunteer

PHASE_1

Clinical Trials (1)

NCT01414881Study to Assess the Effects of Mipomersen on Lipid and Lipoprotein Metabolism in Healthy Subjects

Phase 1

IONIS MAPTRx

Mild Alzheimer's Disease

PHASE_1

Clinical Trials (1)

NCT03186989Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of IONIS-MAPTRx in Patients With Mild Alzheimer's Disease

Phase 1

Placebo

Healthy Participants

PHASE_1

Clinical Trials (1)

NCT05337878A Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pelacarsen (ISIS 681257) in Healthy Japanese Participants

Phase 1

Placebo

Healthy Volunteers

PHASE_1

Clinical Trials (1)

NCT03263507Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Donidalorsen (IONIS-PKK-LRx) Administered Subcutaneously to Healthy Volunteers

Phase 1

ION537

Advanced Solid Tumors

PHASE_1

Clinical Trials (1)

NCT04659096A Study of ION537 in Patients With Molecularly Selected Advanced Solid Tumors

Phase 1

AZD9150

Advanced Adult Hepatocellular Carcinoma

PHASE_1

Clinical Trials (1)

NCT01839604A Phase I/Ib Study of AZD9150 (ISIS-STAT3Rx) in Patients With Advanced/Metastatic Hepatocellular Carcinoma

Phase 1

mipomersen

Healthy Volunteer

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

BIIB094

Parkinson's Disease

PHASE_1

Clinical Trials (1)

NCT03976349A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB094 in Adults With Parkinson's Disease

Phase 1

IONIS-DMPKRx

Myotonic Dystrophy Type 1

PHASE_1_2

Clinical Trials (1)

NCT02312011A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1

Phase 1/2

Ionis Pharmaceuticals(IONS)

About Ionis Pharmaceuticals

Why Work Here

Key Products

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Pipeline & Clinical Trials

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