Belite Bio has finalised the design for the Phase III clinical trial of Tinlarebant (LBS-008) to treat advanced dry age-related macular degeneration (AMD).

The latest move comes following discussions with the US Food and Drug Administration (FDA).

The two-year prospective, placebo-controlled, double-masked, randomised study has been designed for evaluating the safety and efficacy of daily oral Tinlarebant in geographic atrophy (GA) associated dry AMD patients.

At least 300 GA patients in multiple centres across the world are expected to be enrolled in the study.

Enrolment is anticipated to commence in the first half of next year.

The change in GA lesion size from baseline to month 24 compared to a placebo will be the study’s primary endpoint.

At the mid-point of the study, an interim analysis of Tinlarebant’s efficacy and safety is anticipated to be conducted.

At present, Tinlarebant is being assessed in a two-year Phase II study and a global Phase III study in individuals with Stargardt Disease (STGD1).

Belite Bio CEO Dr Tom Lin said: “We are very excited to initiate our Phase III advanced dry AMD study to bring forward an early intervention, once-a-day oral treatment for patients suffering from visual loss due to dry AMD.

“The safety and efficacy of tinlarebant is currently being evaluated in a Phase II study and a Phase III study in patients affected by STGD1.

“Considering that a common cause of disease progression in both GA and STGD1 is characterised by the aberrant accumulation of cytotoxic byproducts of vitamin A, we are optimistic for a positive outcome for Tinlarebant in GA patients.”

The new oral therapy Tinlarebant has been developed as an early intervention for preventing the accumulation of vitamin A-based toxins (bisretinoids), which cause STGD1 and contribute to dry AMD pathogenesis.

It reduces the serum RBP4 level, a carrier protein that delivers retinol to the eye.

The therapy has already received Rare Pediatric Disease Designation and Fast Track Designation in the US, and Orphan Drug Designation in both Europe and the US for the treatment of STGD1.