Belite Bio, Inc. (NASDAQ: BLTE) has announced advancements in its clinical programs for Tinlarebant, an oral therapy targeting retinal degenerative diseases. The company is focused on addressing unmet medical needs in Stargardt disease (STGD1) and geographic atrophy (GA).
Clinical Trial Updates
The company dosed the first patient in the Phase 2/3 DRAGON II trial of Tinlarebant for STGD1. This trial aims to evaluate the pharmacokinetics and pharmacodynamics of Tinlarebant in Japanese adolescent STGD1 subjects, with a target enrollment of approximately 60 subjects aged 12 to 20 years, including around 10 Japanese subjects. The primary efficacy endpoint is the slowing of atrophic lesion growth rate.
The pivotal global Phase 3 PHOENIX trial of Tinlarebant in GA subjects is ongoing, with more than 280 subjects enrolled as of November 11, 2024. This 24-month, randomized, double-masked, placebo-controlled trial is evaluating the slowing of atrophic lesion growth rate as its primary efficacy endpoint.
Interim analysis from the pivotal global Phase 3 DRAGON trial of Tinlarebant in adolescent STGD1 subjects is anticipated by the end of 2024 or early 2025. The DRAGON trial has completed enrollment with 104 subjects in 11 countries. The primary efficacy endpoint is the slowing of atrophic lesion growth rate.
Tinlarebant (LBS-008) Mechanism
Tinlarebant (LBS-008) is an oral, potent, once-daily retinol binding protein 4 (RBP4) antagonist. It works by decreasing RBP4 levels in the blood, reducing vitamin A (retinol) delivery to the eye without disrupting systemic retinol delivery to other tissues. Accumulation of cytotoxic vitamin A byproducts (bisretinoids) has been implicated in the onset and progression of STGD1 and GA.
Leadership Appointment
Hendrik P. N. Scholl, MD, MA, a globally recognized leader in ophthalmology and the coordinating principal investigator of the largest natural history study of Stargardt disease, has been appointed as Chief Medical Officer. Dr. Scholl's expertise in treating retinal diseases, including Stargardt disease and age-related macular degeneration, is expected to be invaluable to Belite Bio.
Financial Position
As of September 30, 2024, Belite Bio had $109.0 million in cash, money market funds, time deposits, and U.S. treasury bills. The company recently received approximately $28.75 million from the exercise of warrants, which is expected to be used for general corporate purposes.
About Stargardt Disease and Geographic Atrophy
Stargardt disease type 1 (STGD1) is a genetic eye disease that causes progressive vision loss, often beginning in childhood or adolescence. Geographic atrophy (GA) is an advanced form of dry age-related macular degeneration (AMD) that leads to retinal cell death and vision loss in the elderly. There are currently no FDA-approved, orally administered treatments for GA.
