Belite Bio has announced a historic milestone in the fight against Stargardt disease type 1 (STGD1). It has revealed topline results from its global Phase 3 DRAGON trial of Tinlarebant—the first pivotal trial to show meaningful benefit in this rare, vision-robbing condition.
STGD1, an inherited retinal disorder that causes progressive vision loss starting in childhood or young adulthood, has no approved treatments worldwide. DRAGON enrolled 104 patients and hit its primary endpoint, demonstrating a 36% reduction in the growth of retinal lesions compared with placebo. The results were statistically significant.
“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 Dr 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 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. We extend our sincere gratitude to the patients, families, and investigators whose dedication made this achievement possible.”
The therapy was well tolerated, with only four treatment-related discontinuations, and showed a favorable safety profile consistent with earlier studies.
“The significant lesion growth reduction observed in the DRAGON study, along with the favorable safety profile, provide important validation of our therapeutic approach and the mechanism of Tinlarebant," said Dr Nathan Mata, Chief Scientific Officer at Belite Bio.
"These results underscore the team’s commitment to addressing the unmet need in Stargardt disease and the potential to meaningfully improve the quality of life for those affected."
Leading investigators praised the trial’s results. “Seeing well-controlled Phase 3 data that shows a marked slowing of lesion growth in Stargardt disease is deeply encouraging,” said Professor Michel Michaelides, Consultant Ophthalmologist at Moorfields Eye Hospital.
“It is remarkable to recognize that with the robust results of the DRAGON trial, we may soon have Tinlarebant as the first treatment ever for Stargardt disease,” noted Quan Dong Nguyen, Professor of Ophthalmology at Stanford University. “It is only a matter of time before the observed reduction in lesion growth translates into measurable benefits in visual function.”
Dr. Hendrik Scholl, Chief Medical Officer of Belite Bio, emphasized the therapy’s potential to transform care.
“The DRAGON trial delivers the most compelling evidence to date that an oral therapy can alter the course of Stargardt disease. These results validate the scientific approach behind Tinlarebant’s development, demonstrating that reducing the accumulation of toxic byproducts in the retina can meaningfully slow disease progression… Collectively, these data reinforce Tinlarebant’s potential to change the treatment landscape for Stargardt disease and set a new benchmark for future research in inherited retinal disorders.”
Belite Bio plans to engage regulatory authorities and submit New Drug Applications for Tinlarebant in the first half of 2026. The drug has already received multiple designations, including Breakthrough Therapy, Fast Track, and Rare Pediatric Disease in the U.S., Orphan Drug status in the U.S., Europe, and Japan, and Pioneer Drug designation in Japan.
The DRAGON trial was a 24-month, randomized, double-masked, placebo-controlled, global Phase 3 study evaluating adolescent STGD1 patients.
