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IONIS-FB-LRx

Phase 2

Macular Degeneration | Small molecule | Ophthalmology |Ionis Pharmaceuticals, Inc.|Last Updated: Mar 7, 2025

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindCONTROLLED
Total Trials1
Total Enrollment332

FDA Designations

No designations recorded

Clinical trial landscape

IONIS-FB-LRx · 2 trials · 3 indications

Phase 2 2
NCT04014335A Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Participants With Primary IgA NephropathyPrimary IgA Nephropathy
COMPLETED23 Analytics
NCT03815825GOLDEN STUDY: A Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)Macular Degeneration
COMPLETED332 Analytics
PHASE2COMPLETED
A Study to Evaluate the Effectiveness and Safety of IONIS-FB-LRx, an Antisense Inhibitor of Complement Factor B, in Adult Participants With Primary IgA Nephropathy
Primary IgA NephropathyUnlock trial analytics
PHASE2COMPLETED
GOLDEN STUDY: A Study to Assess Safety and Efficacy of Multiple Doses of IONIS-FB-LRx in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration (AMD)
Macular DegenerationUnlock trial analytics

Study Endpoints

Primary Endpoints

Percent Reduction in 24-hour Urine Protein Excretion
Baseline to Week 29 (If participant discontinues Study Drug prior to Week 25, Baseline and 4 weeks after the last dose of Study Drug will be measured)
Rate of Change from Baseline in the Geographic Atrophy (GA) Area at Week 49, as Assessed by Retinal Imaging
Week 49

Secondary Endpoints

Absolute Reduction in 24-hour Urine Protein Excretion
Baseline to Week 29 (If participant discontinues Study Drug prior to Week 25, Baseline and 4 weeks after the last dose of Study Drug will be measured)
Absolute Reduction in Albuminuria (UACr Ratio)
Baseline to Week 29
Absolute Reduction in Proteinuria (UPCr Ratio)
Baseline to Week 29
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
IONIS-FB-LRxEXPERIMENTAL -
PlaceboEXPERIMENTALStage 1 participants will receive a placebo matching solution, administered subcutaneously every 4 weeks, completing the treatment period at Week 45. Stage 2 participants will receive a placebo matching solution, administered subcutaneously every 4 weeks, completing the treatment period at Week 45.

Interventions

NameTypeDescription
IONIS-FB-LRxDRUGParticipants will receive IONIS-FB-LRx, by subcutaneous injection (SC) at Week 1 and every 4 weeks through Week 25. Optional 48-week Extension, with drug dosing continuing every 4 weeks.
PlaceboDRUGPlacebo matching solution, administered subcutaneously every 4 weeks
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Eligibility Criteria

Age Range18 Years to 75 Years
SexALL
Healthy VolunteersNo
Study Sites7

Inclusion Criteria * Females must be non-pregnant and non-lactating, and either surgically sterile or post-menopausal OR use a highly effective method of birth control * Biopsy-proven primary immunoglobulin A (IgA) nephropathy * Hematuria * Proteinuria Exclusion Criteria * Clinically significant ...

Countries:AustraliaCanadaNew ZealandSingaporeUnited StatesAustriaCzechiaNetherlandsPolandSpain
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Frequently asked questions about IONIS-FB-LRx

What is IONIS-FB-LRx used for?

IONIS-FB-LRx is an investigational antisense drug being studied for macular degeneration, specifically geographic atrophy secondary to age-related macular degeneration, and for primary IgA nephropathy. It is in Phase 2 clinical development and is not yet approved by the FDA.

Who makes IONIS-FB-LRx?

IONIS-FB-LRx is being developed by Ionis Pharmaceuticals, Inc., which trades on NASDAQ under the ticker IONS. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with geographic atrophy and primary IgA nephropathy.

What phase is IONIS-FB-LRx in?

IONIS-FB-LRx is in Phase 2 clinical development. Two Phase 2 trials have been completed, one in geographic atrophy secondary to age-related macular degeneration and one in primary IgA nephropathy. The drug is investigational and has not received FDA approval.

What clinical trials is IONIS-FB-LRx in?

IONIS-FB-LRx has completed two Phase 2 trials. The GOLDEN STUDY (NCT03815825) enrolled 332 participants with geographic atrophy secondary to age-related macular degeneration. A second trial (NCT04014335) enrolled 23 adults with primary IgA nephropathy. Both trials were randomized, double-blind, and controlled.

How does IONIS-FB-LRx work?

IONIS-FB-LRx is an antisense inhibitor of complement factor B, a protein involved in the complement system. By targeting complement factor B, the drug aims to reduce inflammation and tissue damage in conditions like geographic atrophy and IgA nephropathy. Its mechanism is based on antisense technology.