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Tominersen

Phase 2

Huntington Disease | Small molecule | Neurology |Roche Holding AG|Last Updated: Aug 10, 2026

Target and mechanism

Molecular targetHTT
Target classAntisense Inhibitor
ModalitySmall molecule
ChEMBLCHEMBL4297581

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

Clinical trial landscape

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 8 trials (matched to "Huntington Disease")

CompanyTickerTrialsLead PhaseDrugs
Novartis AG Sponsored ADRNVS2PHASE3Votoplam
Neurocrine Biosciences, Inc.NBIX1PHASE3Valbenazine
Alnylam Pharmaceuticals, IncALNY1PHASE1ALN-HTT02
uniQure N.V.QURE2PHASE1intra-striatal rAAV5-miHTT
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?

Tominersen is an investigational small molecule being developed for Huntington disease. It is currently being studied in patients with prodromal and early manifest Huntington's disease, a progressive neurological condition. The drug is administered via intrathecal injection and is designed to target the underlying cause of the disease.

What does Tominersen target?

Tominersen is an antisense oligonucleotide (ASO) that targets the huntingtin gene. It is designed to reduce the production of the huntingtin protein, which is the cause of Huntington disease. By lowering the levels of this protein, Tominersen aims to slow or halt the progression of the disease.

Who is developing Tominersen?

Tominersen is being developed by Roche Holding AG, a multinational healthcare company. Roche is conducting clinical trials to evaluate the safety, biomarkers, and efficacy of Tominersen in patients with Huntington disease. The company is responsible for the drug's development and regulatory strategy.

What phase is Tominersen in?

Tominersen is currently in Phase 2 clinical development. It is being evaluated in a randomized, double-blind, placebo-controlled study in patients with prodromal and early manifest Huntington's disease. The drug is not yet approved by regulatory authorities and remains investigational.

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's disease. The trial is active but not recruiting, with an enrollment of 301 participants.

Is Tominersen the same as other Huntington disease drugs?

Tominersen is a unique antisense oligonucleotide designed specifically for Huntington disease. It is not the same as other drugs in development for this condition, as it targets the huntingtin gene directly. Its mechanism of action and clinical development program are distinct from other investigational therapies.