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Our Pipeline

Our goal is to discover and develop potentially life-changing therapeutics across a range of neurologic diseases.1

The Ionis Neurology pipeline includes investigational RNA-targeted therapeutics for severe and debilitating neurologic diseases that affect a wide range of patients with unmet needs.1,2

 

Our RNA-targeted drug discovery and development platform provides a fast, efficient path to identify first-in-class and/or best-in-class therapeutics.2-7

 

With 3 FDA-approved neurologic therapeutics and more than 10 investigational RNA-targeted therapeutics in mid- or late-stage development, Ionis is potentially transforming the trajectory of serious neurologic diseases.1,2,8

 

 

Ionis’ Pipeline Includes Early- and Late-Stage Investigational Therapeutics Designed to Target Genetic Sequences Associated With a Range of Rare and Prevalent Neurologic Conditions1,9-21,a,b

 

Investigational RNA-Targeted Therapeutic Disease State Gene/Target Phase 1 Phase 2 Phase 3
Ionis-owned
Zilganersen
Alexander disease GFAP

ION582c

Angelman syndrome UBE3A-ATS

ION717d

Prion disease PRNP

ION440

MECP2 duplication syndrome

MECP2

ION356

Pelizaeus-Merzbacher disease

PLP1

ION859e

Parkinson’s disease LRRK2

ION464e

Multiple system atrophy and
Parkinson's disease

SNCA

ION269d

Alzheimer's disease
in Down syndrome

APP
Investigational RNA-Targeted Therapeutic Disease State Gene/Target Phase 1 Phase 2 Phase 3
Ionis-Biogen

Tofersen

Superoxide dismutase 1 
amyotrophic lateral schlerosis

SOD1

IONIS-MAPTRxe

Alzheimer’s disease TAU

Salanersen

Spinal muscular atrophy SMN2
Investigational RNA-Targeted Therapeutic Disease State Gene/Target Phase 1 Phase 2 Phase 3
Ionis-Otsuka
Ulefnersen

Fused in sarcoma amyotrophic
lateral sclerosis

FUS
Investigational RNA-Targeted Therapeutic Disease State Gene/Target Phase 1 Phase 2 Phase 3
Ionis-Roche
Tominersen
Huntington’s disease HTT

 

aContent in the table subject to change pending updates to Ionis pipeline. bSafety and efficacy have not been evaluated by any regulatory authorities for the indications described. cThe US Food and Drug Administration has granted both orphan drug designation and rare pediatric disease designation for its investigational drug ION582.15 dThis investigational antisense therapeutic is in a Phase 1/2a study.1 eThis investigational antisense therapeutic is in a Phase 1 study. The primary purpose of the study is the evaluation of the therapeutic’s safety profile. It is listed here as Phase 2 because the therapeutic is being tested in patients and not healthy volunteers. This study may be categorized by partners or on regulatory sites, such as ClinicalTrials.gov, as a Phase 1 study.1 

 

 

 

FUS, fused in sarcoma protein gene; GFAP, glial fibrillary acidic protein gene; HTT, huntingtin gene; LRRK2, leucine-rich-repeat kinase 2 gene; MECP2, methyl-CpG-binding protein 2 gene (human); PLP1, proteolipid protein 1 gene; PRNP, prion protein gene; SCNA, synuclein alpha gene; SMN2, survival of motor neuron 2 gene; SOD1, superoxide dismutase 1 gene; TAU, tau protein gene; UBE3A-ATS, ubiquitin protein ligase E3A antisense transcript.

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Zilganersen (Alexander disease) clinical study

Zilganersen (Alexander disease)

Zilganersen
(Alexander disease)

Phase:

Recruitment status:

Active, not recruiting
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ION582 (Angelman syndrome) clinical study

ION582 (Angelman syndrome)

ION582c
(Angelman syndrome)

Phase:

Recruitment status:

Currently recruiting
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ION717 (Prion disease) clinical study

ION717 (Prion disease)

ION717d
(Prion disease)

Phase:

Recruitment status:

Active, not recruiting
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ION440 (MECP2 duplication syndrome) clinical study

ION440 (MECP2 duplication syndrome) 

ION440
(MECP2 duplication syndrome)

Phase:

Recruitment status:

Currently recruiting
Image
ION356 (Pelizaeus-Merzbacher disease) clinical study

ION356 (Pelizaeus-Merzbacher disease)

ION356
(Pelizaeus-Merzbacher disease)

Phase:

Recruitment status:

Currently recruiting
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ION859 (Parkinson’s disease) clinical study

ION859 (Parkinson’s disease)

ION859e
(Parkinson’s disease)

Phase:

Recruitment status:

Completed
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ION464 (Multiple system atrophy) clinical study

ION464 (Multiple system atrophy)

ION464e
(Multiple system atrophy)

Phase:

Recruitment status:

Currently recruiting
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Tofersen icon

Tofersen (Superoxide dismutase 1 amyotrophic lateral sclerosis)

Tofersen
(Superoxide dismutase 1
amyotrophic lateral sclerosis)

Phase:

Recruitment status:

Completed
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IONIS-MAPT (Alzheimer’s disease) clinical study

IONIS-MAPTRx (Alzheimer’s disease)

IONIS-MAPTRxe
(Alzheimer’s disease)

Phase:

Recruitment status:

Completed
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ION306 (Spinal muscular atrophy) clinical study

Salanersen (Spinal muscular atrophy)

Salanersen
(Spinal muscular atrophy)

Phase:

Recruitment status:

Active, not recruiting
Image
Ulefnersen (Fused in sarcoma amyotrophic lateral sclerosis)

Ulefnersen (Fused in sarcoma amyotrophic lateral sclerosis)

Ulefnersen
(Fused in sarcoma
amyotrophic lateral sclerosis)

Phase:

Recruitment status:

Currently recruiting
Image
Tominersen (Huntington’s disease) clinical study

Tominersen (Huntington’s disease)

Tominersen
(Huntington’s disease)

Phase:

Recruitment status:

Active, not recruiting
Image
Multiple System Atrophy (MSA) disease state education download

ION269
(Alzheimer's diseasein Down syndrome) 

ION269d
Alzheimer's disease in Down
syndrome 

Phase:

Recruitment status:

Currently recruiting

 

aContent in the table subject to change pending updates to Ionis pipeline. bSafety and efficacy have not been evaluated by any regulatory authorities for the indications described. cThe US Food and Drug Administration has granted both orphan drug designation and rare pediatric disease designation for its investigational drug ION582.15 dThis investigational antisense therapeutic is in a Phase 1/2a study.1 eThis investigational antisense therapeutic is in a Phase 1 study. The primary purpose of the study is the evaluation of the therapeutic’s safety profile. It is listed here as Phase 2 because the therapeutic is being tested in patients and not healthy volunteers. This study may be categorized by partners or on regulatory sites, such as ClinicalTrials.gov, as a Phase 1 study.1 

 

 

 

FUS, fused in sarcoma protein gene; GFAP, glial fibrillary acidic protein gene; HTT, huntingtin gene; LRRK2, leucine-rich-repeat kinase 2 gene; MECP2, methyl-CpG-binding protein 2 gene (human); PLP1, proteolipid protein 1 gene; PRNP, prion protein gene; SCNA, synuclein alpha gene; SMN2, survival of motor neuron 2 gene; SOD1, superoxide dismutase 1 gene; TAU, tau protein gene; UBE3A-ATS, ubiquitin protein ligase E3A antisense transcript.

Learn more about the current clinical trials for Ionis’ RNA-targeted therapeutics.

Learn more about the neurologic diseases in Ionis’ pipeline.

 
 

References