December 02, 2025
2 min read
Key takeaways:
- Tinlarebant reduced lesion growth rate by 35.7%.
- The drug was well tolerated throughout the trial.
Belite Bio announced positive topline results from the phase 3 DRAGON trial of Tinlarebant in patients with Stargardt disease.
Tinlarebant (LBS-008) is an oral therapy designed to reduce the accumulation of cytoxic bisretinoids, toxins that cause retinal disease in Stargardt disease type 1. The drug has obtained breakthrough therapy, fast track and rare pediatric disease designations from the FDA, according to the release.
Belite Bio chief medical officer Hendrik Scholl, MD, told Healio that Tinlarebant has the potential to offer patients a treatment that can slow the progression of Stargardt disease.
“For a progressive disease with no current treatment options, gaining those extra years of better function is highly meaningful,” he said in an email. “For patients, that could mean more years of being able to read, recognize faces and participate fully in school or work. For eye care providers, having a therapy that targets the underlying biology of the disease would represent a meaningful shift in how Stargardt is managed today.”
The DRAGON trial was a 24-month, randomized, placebo-controlled trial including 104 patients aged between 12 and 20 years with Stargardt disease type 1. All patients had at least one mutation identified in the ABCA4 gene, an atrophic lesion size within three disc areas (7.62 mm2), and best corrected visual acuity of 20/200 or better, according to the release.
The company said Tinlarebant achieved the study’s primary endpoint by demonstrating a reduction in lesion growth rate of 35.7% versus placebo (P = .0033) measured by retinal imaging. A post-hoc analysis showed that the treatment effect remained consistent (P < .0001). Researchers also observed a 33.6% reduction in lesion growth in the fellow eye, according to the release.
Additionally, the therapy helped slow decreased autofluorescence lesion growth in the study eye (33.7%, P = .027) and fellow eye (32.7%, P = 0.017). The 5 mg dose reduced retinal binding protein 4 (RBP4) levels by a mean of 80% compared with baseline and RBP4 levels returned to 84% of baseline at the study’s conclusion, according to the release.
The company said the drug was well tolerated by patients. There were four drug discontinuations related to treatment. No drug or trial discontinuations were due to non-ocular adverse events. The most common drug-related ocular adverse event was xanthopsia which — along with delayed dark adaptation and night vision impairment — was mild and most cases were resolved during the trial, according to the release.
Belite Bio plans to file a new drug application with the FDA in the first half of 2026. Scholl said the trial results reinforce the drug’s potential as the company prepares to advance through the regulatory process.
“Belite also continues to progress the PHOENIX phase 3 trial in geographic atrophy, where the same biological pathway is thought to contribute to retinal degeneration,” he told Healio. “While each condition is distinct, the mechanistic consistency gives the company confidence in continuing to explore Tinlarebant’s broader therapeutic potential. Additional analyses from DRAGON and other ongoing studies will be shared at an upcoming medical meeting.”
Sources/Disclosures
Source:
Disclosures:
Scholl reports being an employee of Belite Bio.
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