Dose-ranging study of FWY003 in geographic atrophy secondary to age-related macular degeneration: randomized, double-masked, placebo-controlled trial in adults

The primary objective of this study is to evaluate the dose response relationship of FWY003 on the geographic atrophy lesion area in patients with age-related macular degeneration. This assessment is clinically significant to determine the optimal dosage for managing lesion progression.

Secondary objectives include:

• Assessment of the safety and tolerability of FWY003.
• Evaluation of the effect of FWY003 on visual function in the study eye from baseline through Month 18.
• Assessment of the effect of FWY003 on photoreceptor parameters from baseline through Month 18.
• Evaluation of pharmacokinetics of FWY003 from baseline through Month 18.
• Assessment of the rate of change in the geographic atrophy lesion area from baseline through Month 18.
• Evaluation of the effect of FWY003 on the geographic atrophy lesion area from baseline through Month 00.

This clinical trial involves a total of 130 participants diagnosed with geographic atrophy secondary to age-related macular degeneration. The study population consists of male and female patients within specific age ranges. Inclusion requires a diagnosis of the condition in at least one eye, with the lesion size measured by fundus autofluorescence. The total lesion area must be between 2.5 and 17.5 mm², and the lesion must be visualized on the macula-centered image without being contiguous with peripapillary atrophy. Additionally, participants must possess an equal to or greater than etDRS best-corrected visual acuity of 35 letters in the study eye. The primary objective is to evaluate the dose-response relationship of FWY003 on the lesion area.

This Phase 2, randomized, double-masked, placebo-controlled, multicenter, dose-range finding study is designed to evaluate the efficacy and safety of FWY003 tosilate in patients with geographic atrophy secondary to age-related macular degeneration. The main objective is to assess the dose response relationship regarding the lesion area. Participants undergo a screening visit to confirm eligibility based on age, diagnosis, and specific fundus autofluorescence lesion size requirements. The primary endpoint is the change in the lesion area from baseline to Month 18. Secondary endpoints include the incidence of adverse events, changes in best corrected visual acuity, and modifications in the ellipsoid zone attenuation. The study involves oral administration of a capsule. Participant involvement is expected to continue through the 18-month assessment period, with the trial estimated to conclude by August 2029.

The investigational medicinal product is FWY003 tosilate, administered in a capsule pharmaceutical form. The administration is via the oral route at a dosage of 00 DF dosage form.

A placebo to FWY003 is utilized as a comparator in this study.

The efficacy of FWY003 in patients with geographic atrophy secondary to age-related macular degeneration will be assessed through several parameters. The primary endpoint is the change in GA lesion area from baseline to month 18. Secondary efficacy endpoints include the rate of change of the GA lesion area, expressed as a square-root transformed value, from baseline to month 18 and from baseline to month 00. Additionally, the change in the area of total and partial ellipsoid zone attenuation in the macula will be evaluated from baseline through month 18.

Changes in visual function in the study eye will be measured from baseline through month 18 using the following methods:

• Best Corrected Visual Acuity (BCVA) under regular luminance using the Early treatment diabetic retinopathy study (ETDRS) scale.
• ETDRS low luminance visual acuity.
• Quantitative contrast sensitivity function (qCSF) under regular luminance.
• Low Contrast quantitative Visual Acuity (LCqVA) under regular luminance and low luminance.
• The proportion of participants experiencing a loss of ≥15 letters from baseline.