Belite Bio Reports Positive Topline Results from Pivotal Stargardt Trial

Rare Daily Staff

Belite Bio reported positive topline results from its global Phase 3 DRAGON trial of its experimental therapy Tinlarebant to treat people with Stargardt disease, positioning the oral therapy as the first potential treatment for the rare eye disease.

Stargardt disease (STGD1) is the most common inherited retinal dystrophy, causing blurring or loss of central vision in both adults and children. The disease is caused by a dysfunctional retina-specific gene (ABCA4), which results in the massive accumulation of toxic vitamin A (retinol) byproducts (bisretinoids) in the retina, leading to retinal cell death and progressive loss of central vision.

Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4, the sole carrier protein for retinol transport from the liver to the eye. By modulating the amount of retinol entering the eye, Tinlarebant reduces the formation of bisretinoids.

The U.S. Food and Drug Administration has granted Tinlarebant Breakthrough Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations. Regulators in Europe and Japan have granted Orphan Drug designation. Tinlarebant also has been granted Sakigake designation in Japan.

The Phase 3 DRAGON trial enrolled 104 patients with STGD1 and met its primary efficacy endpoint, demonstrating a statistically significant and clinically meaningful 36 percent reduction in the growth rate of retinal lesions—measured as definitely decreased autofluorescence by fundus autofluorescence imaging—compared with placebo. Statistical significance was reached when applying the pre-specified analysis. Considering the progressive nature typically seen in STGD1, a post-hoc analysis providing specific data correlation showed that the treatment effect remained consistent.

“The final results from the DRAGON trial mark a historic breakthrough in Stargardt disease, paving the way for the first potential treatment for this devastating condition and bringing new hope to patients and families who have long faced a disease once considered untreatable,” said Tom Lin, chairman and CEO of Belite Bio. “Not only was Tinlarebant shown to be efficacious in slowing retinal degeneration, but this is also the first time that an oral treatment was able to demonstrate a clinically meaningful outcome in a retinal degenerative disease. With this data, we are advancing our regulatory interactions globally and moving closer to delivering the first approved treatment for people living with Stargardt disease.”

Photo: Tom Lin, chairman and CEO of Belite Bio