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Tominersen

Phase 3

Huntington Disease | RNA therapy | Neurology |Roche Holding AG|Trials Updated: Aug 10, 2026

Tominersen development status

Highest phase Phase 3 (NCT03842969)
Phase scored for RHHBYPhase 2
Registered trials 4 across 1 sponsor since Nov 2017

Tominersen target and mechanism

Molecular targetHTT
Target classAntisense Inhibitor
ModalityRNA therapy
ChEMBLCHEMBL4297581

Also known as RG6042, RO7234292 (RG6042)

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment301

FDA Designations

No designations recorded

Tominersen clinical trials

Tominersen · 1 trial · 1 indication

Phase 2 1
NCT05686551GENERATION HD2. A Study to Evaluate the Safety, Biomarkers, and Efficacy of Tominersen Compared With Placebo in Participants With Prodromal and Early Manifest Huntington's DiseaseHuntington Disease
ACTIVE NOT_RECRUITING301 Analytics
PHASE2ACTIVE NOT_RECRUITING
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
Huntington Disease
NCT05686551301
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Study Endpoints

Primary Endpoints

DB Period: Incidence and Severity of Adverse Events (AEs), With Severity Determined According to the AE Severity Grading Scale
Up to approximately 36 months
DB Period: Change From Baseline in Clinical Laboratory Results - Cerebrospinal Fluid (CSF) White Blood Cell (WBC)
Baseline visit (Day 1), and Months 4, 8, 9, 12, 16
DB Period: Change From Baseline in Clinical Laboratory Results - CSF Protein
Baseline visit (Day 1), and Months 4, 8, 9, 12, 16
DB Period: Change From Baseline in Structural Magnetic Resonance Imaging (MRI) Assessing Any New Abnormalities Including Radiographic Features Consistent With Hydrocephalus and Other Relevant MRI Safety Findings
Baseline, Months 4, 8, 12, 16 and up to approximately 36 months
DB Period: Percentage Change From Baseline in Geometric Means of CSF Mutant Huntingtin (mHTT) Protein Levels at Month 9
Baseline, Month 9
DB Period: Change From Baseline in Composite Unified Huntington's Disease Rating Scale (cUHDRS) Scores (non-U.S. Sites) at 16 Months
Baseline to 16 months

Change in scores on the scale.

DB Period: Change From Baseline in Total Functional Capacity (TFC) Scores (U.S. Sites) at 16 Months
Baseline to 16 months

Change in scores on the scale.

OLE Period: Incidence and Severity of AEs, With Severity Determined According to the AE Severity Grading Scale
Up to approximately 29 months
OLE Period: Change Over Time in Clinical Laboratory Results - CSF WBC
Up to approximately 24 months
OLE Period: Change Over Time in Clinical Laboratory Results - CSF Protein
Up to approximately 24 months
OLE Period: Change From Baseline in Structural MRI Assessing Any New Abnormalities, Including Radiographic Features Consistent With Hydrocephalus and Other Relevant MRI Safety Findings
Up to approximately 29 months

Secondary Endpoints

DB Period: Change From Baseline in Montreal Cognitive Assessment (MoCA) Scores
Baseline, Months 4, 8, 12, 16 and up to approximately 36 months
DB Period: Percentage of Participants With Suicidal Ideation or Behavior (I/B), as Assessed by C-SSRS Score at Each Visit, Including Detailed Focus on Any Individual Cases Identified as Having Severe I/B During the Study Conduct
Up to approximately 36 months
DB Period: Change From Baseline at 16 Months in TFC (non-U.S. Sites) Scores
Baseline to 16 months
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Tominersen 60 milligrams (mg)EXPERIMENTAL60 mg tominersen administered intrathecally (IT) every 16 weeks (Q16W). Tominersen will be administered in the DB period and the OLE period.
PlaceboPLACEBO_COMPARATORPlacebo will be administered IT, Q16W in the DB period.
Tominersen 100 mgEXPERIMENTAL100 mg tominersen administered IT, Q16W. Tominersen will be administered in the DB period and the OLE period.

Interventions

NameTypeDescription
TominersenDRUGTominersen will be administered at the dose and schedule specified in the protocol.
PlaceboDRUGMatching placebo administered IT, Q16W during the DB period.
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Eligibility Criteria

Age Range25 Years to 50 Years
SexALL
Healthy VolunteersNo
Study Sites70

Inclusion Criteria: DB Period: * HD gene expansion mutation carrier status with a cytosine-adenine-guanine-age product (CAP) score of 400-500 inclusive * Either: * Prodromal HD (defined as Diagnostic Confidence Level (DCL) 2 to 3, Independence Scale (IS) ≥70, and TFC ≥8); Or * Early manifest ...

Countries:United StatesArgentinaAustraliaAustriaCanadaDenmarkFranceGermanyItalyNew ZealandPolandPortugalSpainSwitzerlandUnited Kingdom
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Competitive Landscape -Huntington's Disease 15 trials (matched to "Huntington Disease")

CompanyTickerTrialsLead PhaseDrugs
Novartis AG Sponsored ADRNVS2PHASE3Votoplam
Neurocrine Biosciences, Inc.NBIX1PHASE3Valbenazine
Hoffmann-La RocheRHHBY3PHASE2Tominersen, RG6496, SPK-10001
Alnylam Pharmaceuticals, IncALNY1PHASE1ALN-HTT02
uniQure N.V.QURE2PHASE1AMT-130
Sarepta Therapeutics, Inc.SRPT1PHASE1SRP-1005
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Recent Changes (Last 90 Days)

LOWAug 11, 2026NCT05686551Completion: 2027-04-01 → 2026-10-31
LOWAug 11, 2026NCT05686551Completion: 2027-04-01 → 2026-10-31
LOWAug 11, 2026NCT05686551Completion: 2027-04-01 → 2026-10-31

Frequently asked questions about Tominersen

What is Tominersen used for in Huntington Disease?

Tominersen is an investigational RNA therapy being developed for Huntington disease, a genetic neurological disorder. It is being studied in people with prodromal and early manifest Huntington disease, meaning those with early signs or a confirmed diagnosis. It is not approved for any use and remains in clinical development.

What does Tominersen target?

Tominersen targets huntingtin, or HTT, the protein produced by the mutated gene that causes Huntington disease. It is an antisense inhibitor designed to reduce production of huntingtin. By lowering HTT, the therapy aims to address the underlying cause of the disease rather than only its symptoms.

Who is developing Tominersen?

Tominersen is being developed by Roche Holding AG, which trades under the ticker RHHBY. Roche is running the clinical program for the drug in Huntington disease.

What phase is Tominersen in?

Tominersen is in Phase 2 clinical development. It has not been approved by the FDA or any other regulatory agency. The ongoing Phase 2 study is evaluating the drug in participants with prodromal and early manifest Huntington disease.

What clinical trials is Tominersen in?

Tominersen is being studied in the GENERATION HD2 trial, registered as NCT05686551. This Phase 2 study is evaluating the safety, biomarkers, and efficacy of tominersen compared with placebo in participants with prodromal and early manifest Huntington disease. It has enrolled 301 participants and is currently active but not recruiting.

Other drugs targeting HTT

PTC518 PTCT· PHASE2 SRP-1005 SRPT· PHASE1

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