Belite Bio's Tinlarebant Receives FDA Breakthrough Therapy Status for Stargardt Disease

Belite Bio, Inc., a clinical-stage biopharmaceutical company, has announced a significant advancement for its lead candidate, Tinlarebant, in the treatment of Stargardt disease (STGD1). The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to Tinlarebant based on interim data from the ongoing Phase 3 DRAGON trial. Stargardt disease, a genetic eye disorder causing progressive vision loss, currently lacks approved treatments, highlighting the importance of this development.

Dr. Tom Lin, Chairman and CEO of Belite Bio, emphasized the significance of the Breakthrough Therapy Designation, which acknowledges Tinlarebant’s potential to meet the unmet medical needs of STGD1 patients. He expressed the company’s commitment to the Stargardt community and its intention to advance Tinlarebant as it prepares for further data analysis by the end of the year. The designation follows an interim analysis of the global Phase 3 DRAGON trial, which demonstrated Tinlarebant's efficacy and favorable safety profile.

The DRAGON trial is a pivotal, randomized, double-masked, placebo-controlled study designed to assess Tinlarebant's safety and efficacy in adolescent STGD1 patients. The study enrolled 104 participants across 11 regions, including the United States, Europe, and Asia, with a 2:1 randomization favoring Tinlarebant over placebo. The primary endpoint of the trial focuses on the growth rate of atrophic lesions, alongside evaluations of safety and tolerability.

According to Dr. Nathan L. Mata, Chief Scientific Officer at Belite Bio, this designation represents a major achievement, reinforcing the therapeutic approach of targeting the underlying mechanisms of STGD1 to delay or halt disease progression. Dr. Mata highlighted the progress made in understanding the disease, partly through the development and study of a STGD1 mouse model over the past 26 years. This research has advanced knowledge on targeting retinol binding protein 4 (RBP4) to reduce harmful byproducts of vitamin A, bringing the scientific community closer to an approved treatment for STGD1.

The FDA's Breakthrough Therapy Designation aims to expedite the development of drugs for serious or life-threatening conditions. This designation is supported by preliminary clinical evidence showing substantial improvement over existing therapies on significant clinical endpoints. Benefits of this designation include eligibility for rolling review, priority review, and increased regulatory engagement to facilitate faster development, aiming to bring therapies to patients more swiftly.

Tinlarebant is administered orally as a daily tablet, positioned as an early intervention for maintaining retinal health in patients with Stargardt disease and Geographic Atrophy (GA) due to advanced dry age-related macular degeneration (AMD). If approved, Tinlarebant would be a novel oral therapeutic option addressing unmet medical needs in these areas, with no current FDA-approved treatments for STGD1 or orally administered therapies for GA.

Belite Bio focuses on developing novel therapeutics for degenerative retinal diseases with significant unmet needs, including STGD1 and GA, in addition to certain metabolic diseases. The company is progressing with its clinical trials, with Tinlarebant being evaluated in the Phase 3 DRAGON study for adolescent STGD1 patients and the Phase 3 PHOENIX study for GA patients.

Overall, the Breakthrough Therapy Designation for Tinlarebant marks a pivotal step in advancing potential treatments for Stargardt disease, indicating promising clinical progress and a commitment to improving the quality of life for those affected by this condition.