IONIS-FB-LRx for Age-Related Macular Degeneration

No longer recruiting at 117 trial locations

Overseen By Ionis Pharmaceuticals

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Ionis Pharmaceuticals, Inc.

Must not be taking: Steroids

Disqualifiers: Diabetic retinopathy, Diabetic macular edema, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 11

Duration: 45 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called IONIS-FB-LRx for individuals with age-related macular degeneration (AMD), a condition that damages the retina and affects vision. The goal is to determine how effectively the treatment slows the growth of damaged areas in the eye. Participants will receive either the treatment or a placebo (a substance with no active ingredient) via injection every four weeks. Individuals with clear signs of geographic atrophy (a specific type of eye damage) due to AMD, who can see at least 20/200 in the study eye, may be suitable for this trial. As a Phase 2 trial, this research focuses on measuring the treatment's effectiveness in an initial, smaller group, offering participants a chance to contribute to important advancements in AMD treatment.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but it does exclude those on chronic steroid treatment. It's best to discuss your specific medications with the trial team.

Is there any evidence suggesting that IONIS-FB-LRx is likely to be safe for humans?

Research has shown that IONIS-FB-LRx demonstrates promising safety results from earlier studies. In a study with 54 healthy volunteers, the treatment was well-tolerated. Participants experienced a decrease in plasma FB, a protein linked to eye problems, and no major safety issues were reported. These findings suggest that IONIS-FB-LRx is generally safe for humans, with few side effects. While these results are encouraging, further research is needed to confirm its safety in individuals with age-related macular degeneration (AMD). ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for AMD?

Unlike the standard treatments for age-related macular degeneration, which often include anti-VEGF injections like ranibizumab (Lucentis) or aflibercept (Eylea), IONIS-FB-LRx works by targeting a different pathway. This innovative treatment uses antisense technology to specifically reduce the production of a protein called Factor B, which is involved in the inflammation process that contributes to this eye condition. Researchers are excited about this because it offers a new mechanism of action that could potentially provide benefits to patients who might not respond well to existing therapies. Additionally, IONIS-FB-LRx is administered subcutaneously every four weeks, which could offer a more convenient dosing regimen compared to the more frequent injections required by some current options.

What evidence suggests that IONIS-FB-LRx might be an effective treatment for age-related macular degeneration?

Research has shown that IONIS-FB-LRx is being developed to treat geographic atrophy (GA), a condition caused by age-related macular degeneration (AMD). This treatment targets Factor B, a component of the immune system. Early results suggest that IONIS-FB-LRx might slow the progression of GA, a major issue in AMD. However, some results have been mixed, and further research is needed to confirm its effectiveness. While promising, the treatment remains under study in this trial, where participants will receive either IONIS-FB-LRx or a placebo to fully understand its potential benefits. ^{1 2 3 6 7}

Are You a Good Fit for This Trial?

This trial is for adults with Geographic Atrophy due to Age-Related Macular Degeneration. Participants must have a certain level of vision, be non-pregnant, non-lactating, and not recently treated with steroids or other investigational drugs. They should also be vaccinated against specific infections.

Inclusion Criteria

My eyes can be clearly imaged for the study.

I am not pregnant or breastfeeding, and I cannot have children due to surgery or menopause.

Best-corrected visual acuity (BCVA) letter score of 35 letters (approx. 20/200 Snellen equivalent) or better on the ETDRS chart

Exclusion Criteria

You have previously been treated with an experimental medication, biological product, or medical device.

I am on long-term steroid treatment, including creams or eye injections.

I have had diabetic eye problems.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive IONIS-FB-LRx or placebo subcutaneously every 4 weeks, completing the treatment period at Week 45

45 weeks

11 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• IONIS-FB-LRx

Trial Overview The study tests IONIS-FB-LRx's ability to slow down the expansion of damaged areas in the eye caused by AMD. It compares this new treatment against a placebo using high-quality imaging techniques to measure changes over time.

How Is the Trial Designed?

Group I: Placebo Experimental Treatment 1 Intervention

Stage 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.

Group II: IONIS-FB-LRx Experimental Treatment 1 Intervention

Stage 1 participants will receive IONIS-FB-LRx randomized to 1 of 3 dose levels, administered subcutaneously every 4 weeks, completing the treatment period at Week 45. Stage 2 will expand 2 of the dosing cohorts in a new randomized group of participants based on the Stage 1 interim analysis. Participants will be randomized to 1 of 2 of the expanded cohorts, administered subcutaneously every 4 weeks, completing the treatment period at Week 45.

Who Is Running the Clinical Trial?

Ionis Pharmaceuticals, Inc.

Lead Sponsor

Trials

151

Recruited

27,800+

Dr. Brett P. Monia

Ionis Pharmaceuticals, Inc.

Chief Executive Officer since 2020

PhD in Pharmacology from the University of Pennsylvania, BSc in Molecular Biology and Analytical Chemistry from Stockton State College

Dr. Eric Bastings

Ionis Pharmaceuticals, Inc.

Chief Medical Officer

MD

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03815825

NCT03815825 | GOLDEN STUDY: A Study to Assess ... Evaluate the effect of IONIS-FB-LRx on the rate of change of the area of geographic atrophy (GA) secondary to age-related macular degeneration (AMD) measured by ...

2. ir.ionis.com ir.ionis.com/news-releases/news-release-details/ionis-enters-new-collaboration-partner-develop-ionis-fb-l-rx

Ionis Enters New Collaboration with Partner to Develop ... This collaboration will leverage Ionis' leadership in RNA-targeted therapeutics to develop IONIS-FB-LRx targeting Factor B (FB) for a broad range of diseases.

3. retinaroundup.com retinaroundup.com/2025/02/11/angiogenesis-2025-results-of-the-golden-and-verona-studies/

Angiogenesis 2025 – Results of the Golden and Verona ... Efficacy and Safety of Complement Factor B Antisense, IONIS-FB-LRx, in Geographic Atrophy due to Age-Related Macular Degeneration: Results ...

4. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC9414333/

Recent Advances in Age-Related Macular Degeneration ... Development of IONIS-FB-LRx to Treat Geographic Atrophy Associated with AMD. IOVS. 2020;61:4305. [Google Scholar]; 192. Safety and Efficacy of IONIS-FB-Lrx ...

5. fiercebiotech.com fiercebiotech.com/biotech/ionis-axes-eye-disease-targets-roche-partnered-prospect-after-data-disappoint

Ionis axes eye disease from targets of Roche-partnered ... Ionis axes eye disease from targets of Roche-partnered prospect after data disappoint ... Another of Ionis Pharmaceuticals' key midphase readouts ...

6. cdek.pharmacy.purdue.edu cdek.pharmacy.purdue.edu/trial/NCT03815825/

Trial | NCT03815825 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 ...

7. ctv.veeva.com ctv.veeva.com/study/safety-and-efficacy-of-ionis-fb-lrx-in-up-to-120-patients-55-and-older-with-geographic-atrophy-ga

Safety and Efficacy of IONIS-FB-Lrx in up to 120 Patients 55 ... The purpose of this study is to assess the Safety and Efficacy of IONIS-FB-Lrx for up to 120 patients with Geographic Atrophy secondary to Age Related Macular ...