Belite Bio Inc ADR (NASDAQ:BLTE) leapt 17.1% in premarket trading Monday after unveiling strong Phase 3 results for Tinlarebant — the first pivotal clinical trial to show efficacy in Stargardt disease, a rare inherited condition that causes progressive vision loss and currently has no approved therapies.
The late-stage DRAGON study achieved its main goal, showing Tinlarebant reduced the rate of retinal lesion growth by 36% versus placebo (p=0.0033). The disease affects more than 50,000 people in the U.S., and until now, patients have had no available treatment options.
Tinlarebant demonstrated a favorable safety profile, with only four participants discontinuing due to treatment-related effects. The most common ocular side effects reported — xanthopsia and delayed dark adaptation — were generally mild and resolved over time.
“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.
A total of 104 patients aged 12 to 20 with Stargardt disease type 1 participated in the trial. Beyond the primary endpoint, Tinlarebant also achieved a 33.6% reduction in lesion growth in the fellow eye (p=0.041) and delivered strong performance across multiple secondary outcomes.
Belite Bio expects to submit a New Drug Application to the FDA in the first half of 2026. Tinlarebant already carries several key regulatory designations in the U.S., including Breakthrough Therapy, Fast Track, and Rare Pediatric Disease status, and is recognized as an orphan drug in the U.S., Europe, and Japan.
The company will hold a conference call at 8:00 a.m. ET today to review the study findings.