Belite Bio Inc. (NASDAQ:BLTE) on Monday released topline results from the global Phase 3 DRAGON trial of Tinlarebant, marking the first successful pivotal trial in patients with Stargardt disease type 1 (STGD1).
The Phase 3 DRAGON trial enrolled 104 patients.
STGD1 is an eye disease that leads to progressive vision loss, usually beginning in childhood or young adulthood.
Tinlarebant is intended to reduce the accumulation of vitamin A-based toxins (known as bisretinoids) that cause retinal disease in STGD1.
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Bisretinoids are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), 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.
Tinlarebant achieved the primary efficacy endpoint, demonstrating a statistically significant reduction in lesion growth rate of 35.7% versus placebo (p-value of 0.0033) as measured by retinal imaging.
A statistically significant treatment effect was also observed in the fellow eye for the primary endpoint with 33.6% lesion growth reduction (p = 0.041).
In addition, Tinlarebant slowed decreased autofluorescence (DAF) lesion growth, in the study eye by 33.7% (p = 0.027) and in the fellow eye by 32.7% (p = 0.017).
The 5 mg daily dose achieved a reduction in RBP4 levels by a mean of approximately 80% relative to baseline.
RPB4 levels returned to 84% of the baseline value at the End of Study (one to three months following drug cessation). Recovery of RBP4 concentration correlated well with the decreased Tinlarebant exposure.
Considering the progressive nature typically seen in STGD1, a further post-hoc analysis providing a specific data correlation showed that the treatment effect remained consistent with a p-value < 0.0001.
As expected, the overall change in visual acuity was minimal over the period of 24 months in both study groups, consistent with natural history data.
The safety profile remains consistent with what the company previously reported, and Tinlarebant was well-tolerated with only four treatment-related discontinuations.
The company plans to engage regulatory authorities to discuss potential next steps and to submit New Drug Applications for Tinlarebant in the first half of 2026.
Price Action: BLTE stock is up 11.3% at $153.05 during the premarket session at the last check on Monday.
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