Aug 25, 2022

Belite Bio Launching Phase 3 Clinical Trial for Stargardt Disease Drug

Eye On the Cure Research News

The company’s drug is designed to reduce accumulation of toxins that cause vision loss.

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The San Diego-based biopharmaceutical company Belite Bio is enrolling adolescent patients (ages 12-18) with Stargardt disease in DRAGON, its Phase 3 clinical trial for Tinlarebant, an emerging oral medication designed to slow disease progression and vision loss.

The company has initiated the Phase 3 clinical trial in the US, UK, Germany, Belgium, Switzerland, Hong Kong, Taiwan, and Australia. Approximately 60 patients are targeted for enrollment in the placebo-controlled study. To participate, subjects must have been clinically diagnosed with Stargardt disease and have at least one mutation identified in the ABCA4 gene.

Tinlarebant is designed to inhibit a protein known as retinol binding protein 4 (RBP4) to reduce the uptake of vitamin A to the retina, thereby decreasing the production and accumulation of toxic vitamin A byproducts, which are the hallmark of Stargardt disease.

Stargardt disease is the leading cause of inherited macular degeneration, affecting approximately 30,000 people in the US and tens of thousands more around the world. The condition causes central vision loss and is usually diagnosed in children and young adults.

Belite Bio also plans to launch a Phase 2/3 clinical trial for Tinlarebant in the 4th quarter of 2022.