Belite Bio, Inc. (NASDAQ: BLTE) has announced significant progress in its clinical programs targeting degenerative retinal diseases. The company's lead candidate, Tinlarebant (LBS-008), is under evaluation in multiple Phase 3 and Phase 2/3 trials for Stargardt disease (STGD1) and geographic atrophy (GA). These advancements mark a crucial step forward in addressing unmet medical needs in these conditions.
Tinlarebant for Stargardt Disease
The first patient has been dosed in the Phase 2/3 DRAGON II trial of Tinlarebant for the treatment of Stargardt disease. This follows the completion of the Phase 1b portion of the trial in Japan, which evaluated the pharmacokinetics and pharmacodynamics of Tinlarebant in adolescent STGD1 subjects. The Phase 2/3 trial aims to enroll 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, with safety and tolerability also being assessed.
Belite Bio anticipates reporting interim analysis data from its pivotal global Phase 3 DRAGON trial by the end of 2024 or early 2025. The DRAGON trial is a 24-month, randomized, double-masked, placebo-controlled study involving 104 adolescent STGD1 subjects across 11 countries. The trial's primary endpoint focuses on the slowing of atrophic lesion growth rate.
Tinlarebant for Geographic Atrophy
The pivotal global Phase 3 PHOENIX trial of Tinlarebant in geographic atrophy is actively enrolling patients, with over 280 subjects enrolled as of November 11, 2024. The PHOENIX trial is a 24-month, randomized, double-masked, placebo-controlled study aiming to enroll approximately 430 subjects. The primary efficacy endpoint mirrors that of the DRAGON trial, focusing on slowing the rate of atrophic lesion growth. An interim analysis is planned at the midpoint of the trial.
Leadership Appointment
Belite Bio has appointed Hendrik P.N. Scholl, MD, MA, as Chief Medical Officer. Dr. Scholl is a globally recognized expert in Stargardt disease and age-related macular degeneration. His expertise is expected to be invaluable as Belite Bio continues to advance its clinical programs.
Financial Update
As of September 30, 2024, Belite Bio reported $109.0 million in cash, money market funds, time deposits, and U.S. treasury bills. Research and development expenses for the three months ended September 30, 2024, were $6.8 million, compared to $8.7 million for the same period in 2023. The company reported a net loss of $8.7 million for the three months ended September 30, 2024, compared to a net loss of $10.9 million for the same period in 2023.
About Tinlarebant
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. Vitamin A is critical to normal vision but can accumulate as toxic byproducts in individuals affected with STGD1 and GA, leading to retinal cell death and vision loss.
