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Sefaxersen

Phase 3

Primary IgA Nephropathy | Small molecule | Nephrology |Roche Holding AG|Last Updated: Aug 14, 2026

Target and mechanism

Target class-Sen (Aso)
ModalitySmall molecule

Also known as Sefaxersen (RO7434656)

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials1
Total Enrollment459

FDA Designations

No designations recorded

Clinical trial landscape

Sefaxersen · 1 trial · 1 indication

Phase 3 1
NCT05797610A Study to Evaluate the Efficacy and Safety of Sefaxersen (RO7434656) in Participants With Primary Immunoglobulin A (IgA) Nephropathy at High Risk of ProgressionPrimary IgA Nephropathy
ACTIVE NOT_RECRUITING459 Analytics
PHASE3ACTIVE NOT_RECRUITING
A Study to Evaluate the Efficacy and Safety of Sefaxersen (RO7434656) in Participants With Primary Immunoglobulin A (IgA) Nephropathy at High Risk of Progression
Primary IgA NephropathyUnlock trial analytics

Study Endpoints

Primary Endpoints

Change From Baseline in the Urine Protein-to-Creatinine Ratio (UPCR) at Week 37
Baseline, Week 37

UPCR will be assessed in urine sampled over 24 hours.

Secondary Endpoints

Estimated Glomerular Filtration Rate (eGFR) Slope at Week 105 from Baseline
Baseline, Week 105
Percentage of Participants Achieving Hematuria Resolution at Week 37
At Week 37
Time to the Composite Kidney Failure Endpoint
Up to approximately 36 months
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Sefaxersen (RO7434656)EXPERIMENTALParticipants will receive subcutaneous (SC) doses of sefaxersen (RO7434656) on Days 1, 15, and 29 followed by once every 4 weeks (Q4W) until Week 105. Participants may be eligible to switch to open-label treatment after Week 105 at the investigator's discretion or after the common-close timepoint, whichever occurs first. The common-close timepoint is defined as the data cut-off date for the primary analysis, when approximately 230 participants in the primary cohort have completed the Week 105 visit.
PlaceboPLACEBO_COMPARATORParticipants will receive SC doses of sefaxersen (RO7434656) matching placebo on Days 1, 15, and 29 followed by once Q4W until Week 105. Participants may be eligible to switch to open-label treatment after Week 105 at the investigator's discretion or after the common-close timepoint, whichever occurs first. The common-close timepoint is defined as the data cut-off date for the primary analysis, when approximately 230 participants in the primary cohort have completed the Week 105 visit.

Interventions

NameTypeDescription
Sefaxersen (RO7434656)DRUGSefaxersen (RO7434656) will be administered as SC injection per schedule as specified.
PlaceboDRUGMatching placebo will be administered as SC injection per schedule as specified.
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites178

Inclusion Criteria: * Primary IgAN, as evidenced by a kidney biopsy performed within 10 years prior to or during screening, without known secondary cause * Treatment with maximum tolerated doses of angiotensin-converting enzyme (ACE) inhibitors or angiotensin II receptor blockers (ARBs) for at leas...

Countries:United StatesArgentinaAustraliaBrazilCanadaChinaCzechiaFranceGermanyGreeceHong KongItalyJapanMalaysiaMexicoPolandSingaporeSouth KoreaSpainTaiwanUnited Kingdom
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Recent Changes (Last 90 Days)

MEDIUMAug 14, 2026NCT05797610Status: RECRUITING → ACTIVE_NOT_RECRUITING
MEDIUMAug 14, 2026NCT05797610Status: RECRUITING → ACTIVE_NOT_RECRUITING

Frequently asked questions about Sefaxersen

What is Sefaxersen used for?

Sefaxersen is an investigational small molecule antisense oligonucleotide being developed for the treatment of primary IgA nephropathy, a kidney disease. It is currently in Phase 3 clinical development and has not been approved by regulatory authorities.

What does Sefaxersen target?

Sefaxersen is an antisense oligonucleotide (ASO), a class of drugs that work by targeting and modulating the expression of specific genes. As an ASO, it is designed to interfere with the production of disease-related proteins, though the specific molecular target has not been disclosed.

Who makes Sefaxersen?

Sefaxersen is being developed by Roche Holding AG, a multinational healthcare company. Roche's stock is traded under the ticker symbol RHHBY on the OTC market.

What phase is Sefaxersen in?

Sefaxersen is currently in Phase 3 clinical development. It is an investigational drug, meaning it has not yet been approved by regulatory authorities and is still being studied for safety and efficacy in patients with primary IgA nephropathy.

What clinical trials is Sefaxersen in?

Sefaxersen is being studied in a Phase 3 clinical trial with the identifier NCT05797610. This randomized, double-blind, placebo-controlled study evaluates the efficacy and safety of Sefaxersen in participants with primary IgA nephropathy at high risk of progression. The trial is active but not recruiting and has an enrollment of 459 participants.

Is Sefaxersen the same as RO7434656?

Yes, Sefaxersen is also known as RO7434656. Both names refer to the same investigational drug being developed by Roche for the treatment of primary IgA nephropathy.