> Trialight public data. Index of pages: https://trialight.com/llms.txt
> Web page: https://trialight.com/data/asset/tominersen-ad27b249-1f74-46e0-90f9-4c17f452879d
> Record changed: 2026-10-13

# Tominersen

Highest phase reached

Phase 3

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297581)

Drug type

Oligonucleotide

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297581)

## Development status

**Listing at source**

Listed

**Highest phase reached**

Phase 3

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297581)

## Names and chemistry

**Drug type**

Oligonucleotide

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297581)

**Drug name**

Tominersen

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297581)

### Other names (31)

- ionis httrx
- Ionis httrx
- isis 443139
- Isis 443139
- isis 443139 10 mg
- ISIS 443139 10 mg
- ISIS 443139 120 mg
- ISIS 443139 30 mg
- ISIS 443139 60 mg
- ISIS 443139 90 mg
- isis 443139 free acid
- ISIS-443139 FREE ACID
- rg 6042 free acid
- RG-6042 FREE ACID
- rg6042
- Rg6042
- RG6042
- rg6042 free acid
- RG6042 FREE ACID
- ro7234292
- Ro7234292
- RO7234292
- RO7234292 (RG6042)
- tominersen
- Tominersen

And 6 more, listed on the interactive page.

## Identifiers

**CAS registry numbers**

- 1709886-74-7

**ChEMBL ID**

CHEMBL4297581

Source: [www.ebi.ac.uk](https://www.ebi.ac.uk/chembl/explore/compound/CHEMBL4297581)

**FDA ingredient codes (UNII)**

- 7QI41X9QWC

## Regulatory information

### Other regulatory facts

**Patents**

0

**Designations**

2

**First in class**

No

**Fast track**

No

**Breakthrough designation**

No

**Orphan designation**

No

**Priority review**

No

**Orphan exclusivity**

No

**Accelerated approval**

No

## Pharmacology

### Drug classification

**Drug name stem**

\-rsen

### Mechanisms of action (2)

- **Mechanism**  
Huntingtin mRNA antisense inhibitor

**Action type**  
ANTISENSE\_INHIBITOR

**Target**  
Huntingtin mRNA

**Protein identifier**  
ENSG00000197386
- **Mechanism**  
Huntingtin mRNA antisense inhibitor

**Action type**  
Inhibitor

**Target**  
Huntingtin mRNA

**Protein identifier**  
ENSG00000197386

### Open Targets mechanisms

- **Mechanism**  
Huntingtin mRNA antisense inhibitor

**Action type**  
ANTISENSE INHIBITOR

**Target**  
Huntingtin mRNA

### Investigated indications (5)

- **Condition**  
Pyoderma Gangrenosum

**Highest recorded phase**  
1

**Condition identifier**  
EFO:0006835

**Subject identifier**  
D017511
- **Condition**  
Hidradenitis Suppurativa

**Highest recorded phase**  
1

**Condition identifier**  
EFO:1000710

**Subject identifier**  
D017497
- **Condition**  
Huntington Disease

**Highest recorded phase**  
3

**Condition identifier**  
MONDO:0007739

**Subject identifier**  
D006816
- **Condition**  
Anti-Neutrophil Cytoplasmic Antibody-Associated Vasculitis

**Highest recorded phase**  
1

**Condition identifier**  
MONDO:0015492

**Subject identifier**  
D056648
- **Condition**  
Inflammation

**Highest recorded phase**  
1

**Condition identifier**  
MP:0001845

**Subject identifier**  
D007249

### Open Targets indications (6)

- **Condition identifier**  
EFO\_0006835
- **Condition identifier**  
EFO\_1000710
- **Condition identifier**  
MONDO\_0006559
- **Condition identifier**  
MONDO\_0007739

**Highest recorded phase**  
3
- **Condition identifier**  
MONDO\_0015492
- **Condition identifier**  
MP\_0001845

### Trial references (5)

- NCT02519036  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT02519036)
- NCT03342053  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03342053)
- NCT03761849  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03761849)
- NCT03842969  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT03842969)
- NCT04000594  
Source: [clinicaltrials.gov](https://clinicaltrials.gov/study/NCT04000594)

## Chemical properties

**Prodrug**

No

**Chirality**

Single enantiomer

**Inorganic**

No

**Natural product**

No

### Sequences (6)

- **Component**  
Light chain

**Type**  
Protein

**Sequence**  
DIVLTQSPASLAVSLGQRATISCKASQSVDYDGDSYMKWYQQKPGQPPKLLIYAASNLQSGIPARFSGSGSGTDFTLNIHPVEEEDAATYYCQQSNEDPYTFGGGTKLEIKRTVAAPSVFIFPPSDEQLKSGTASVVCLLNNFYPREAKVQWKVDNALQSGNSQESVTEQDSKDSTYSLSSTLTLSKADYEKHKVYACEVTHQGLSSPVTKSFNRGEC
- **Component**  
Heavy chain

**Type**  
Protein

**Sequence**  
QVQLQQSGPQLVRPGTSVKISCKASGYSFTTFWMDWVKQRPGQGLEWIGRIDPSDSESRLDQRFKDRATLTVDKSSSTVYMQLSSPTSEDSAVYYCARGNDGYYGFAYWGQGTLVTVSSASTKGPSVFPLAPCSRSTSESTAALGCLVKDYFPEPVTVSWNSGALTSGVHTFPAVLQSSGLYSLSSVVTVPSSSLGTKTYTCNVDHKPSNTKVDKRVESKYGPPCPSCPAPEFLGGPSVFLFPPKPKDTLMISRTPEVTCVVVDVSQEDPEVQFNWYVDGVEVHNAKTKPREEQFNSTYRVVSVLTVLHQDWLNGKEYKCKVSNKGLPSSIEKTISKAKGQPREPQVYTLPPSQEEMTKNQVSLTCLVKGFYPSDIAVEWESNGQPENNYKTTPPVLDSDGSFFLYSRLTVDKSRWQEGNVFSCSVMHEALHNHYTQKSLSLSLGK
- **Component**  
Glycosylation sites (N)

**Sequence**  
Asn-296 $ Asn-296''''
- **Component**  
Sequence

**Type**  
Nucleic Acid

**Sequence**  
(3'-5') mC=mU-mC-A-G=d(T=A=A=mC=A=T=T=G=A=mC)=A-mC-mC-A=mC
- **Component**  
Disulfide bridges

**Sequence**  
22-96 $ 22''-96'' $ 23'-92' $ 23'''-92''' $ 133-218' $ 133''-218''' $ 138'-198' $ 138'''-198''' $ 146-202 $ 146''-202'' $ 225-225'' $ 228-228'' $ 260-320 $ 260''-320'' $ 366-424 $ 366''-424''
- **Component**  
Glycosylation sites (N)

**Sequence**  
Asn-296 $ Asn-296''

## Related records

### Trials (6)

- A Study to Evaluate the Efficacy and Safety of Intrathecally Administered RO7234292 (RG6042) in Participants With Manifest Huntington's Disease
- A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7234292 (ISIS 443139) in Huntington's Disease Patients Who Participated in Prior Investigational Studies of RO7234292 (ISIS 443139)
- A Study to Investigate the Pharmacokinetics and Pharmacodynamics of RO7234292 (RG6042) in CSF and Plasma, and Safety and Tolerability Following Intrathecal Administration in Patients With Huntington's Disease
- An Open-Label Extension Study to Evaluate Long-Term Safety and Tolerability of RO7234292 (RG6042) in Huntington's Disease Participants Who Participated in Prior Roche and Genentech Sponsored Studies
- [GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's Disease](https://trialight.com/data/trial/generation-hd2-a-study-to-evaluate-the-safety-biomarkers-and-2a8e154d-ad7a-4801-b441-c73a9d4453f2.md)
- Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ISIS 443139 in Participants With Early Manifest Huntington's Disease

### In development for (4)

- [Anti-neutrophil cytoplasmic antibody-associated vasculitis](https://trialight.com/data/condition/anti-neutrophil-cytoplasmic-antibody-associated-vasculitis-a59f9899-4e99-54af-98e3-b03dbdb0192e.md) Phase 1
- [Hidradenitis suppurativa](https://trialight.com/data/condition/hidradenitis-suppurativa-c1030a8d-4971-5d31-b030-aa19715eb7a2.md) Phase 1
- [Huntington disease](https://trialight.com/data/condition/huntington-disease-fe2d46ec-080a-5259-a901-cad1d6e07061.md) Phase 3
- Pyoderma gangrenosum Phase 1

### Targets (1)

- [HTT](https://trialight.com/data/target/htt-bf3c6b9d-3906-5cad-ad8c-c4b4c7761722.md) Huntingtin mRNA antisense inhibitor

---

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