Ophthalmology Times

Belite Bio announces Positive Results from Phase 3 DRAGON Trial for Stargardt Disease Treatment

September 15, ​2025 – Belite Bio today ​announced the successful completion of its Phase 3 ‍DRAGON trial evaluating tinlarebant for the treatment of Stargardt disease (STGD1), a rare inherited form of macular degeneration​ causing vision loss in children and young adults.‍ This marks a notable milestone as tinlarebant potentially represents the first-ever treatment ‌for this ⁤devastating condition, for which no approved therapies currently exist.

the multicenter, randomized, double-masked, placebo-controlled DRAGON study enrolled 104 participants aged 12 too 20 years old across ⁢11 regions – Australia, Belgium, the US, the UK, Germany, France, Switzerland, China, the Netherlands, Hong Kong, and Taiwan. Participants were ‍required to have a clinical diagnosis of STGD1 with at least one identified mutation‌ in the ABCA4 gene and a defined aggregate atrophic lesion size within 3 disc areas as⁤ imaged ‌by‍ fundus autofluorescence (FAF). The ⁢study utilized a 2:1 randomization ratio, favoring tinlarebant over placebo.

Tinlarebant⁤ works by minimizing ⁢and regulating levels of retinol binding protein 4 (RBP4), ‌the primary carrier of retinol from the liver to the eye, ultimately decreasing the buildup of vitamin A-based toxins called bisretinoids. These ​bisretinoids are believed to contribute to the progression of STGD1,and ​also ⁢geographic atrophy (GA) associated with age-related macular degeneration (AMD). The primary goal of the DRAGON trial was to measure the rate of ​atrophic lesion growth, alongside assessing the tolerability and safety ⁣of tinlarebant.

“This is an important milestone in our mission to bring a treatment to patients living with ⁢Stargardt disease,” said Belite Bio CEO ​and chairman Tom Lin, MMed, PhD, MBA. “With no approved therapies available today, tinlarebant has the​ potential to be the first treatment for this devastating inherited macular⁤ degeneration.”

Tinlarebant has received fast-track designation and rare pediatric disease designation in the US, ‌orphan drug designation in the US, Japan, and Europe, and the Sakigake designation‍ in Japan for the treatment of STGD1.

References:

1. Belite Bio wraps ⁤up Phase III trial of Stargardt disease ⁢treatment. ⁤Clinical Trials Arena.Published September 15,2025. Accessed⁣ September 15, 2025. https://www.clinicaltrialsarena.com/news/belite-bio-stargardt-disease/
2. Clinicaltrials.gov.Published 2025. Accessed September 15, 2025. https://clinicaltrials.gov/study/NCT05244304?term=DRAGON%20phase%203%20stargardt&rank=1

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## Syphilis: A rising Concern and Its Uncommon Presentations

Syphilis is a sexually transmitted infection (STI) spread through direct contact with skin sores [2].While commonly occurring, diagnosis can be problematic due too its varied‌ and sometimes ‌non-specific ‌initial presentation [2]. The‌ infection progresses in stages – primary, secondary, and tertiary ‌- each with distinct characteristics ​ [3]. Some individuals ‍may be asymptomatic⁢ yet still carry the bacteria [3].

A recent case report highlights an unusual manifestation‌ of ⁣syphilis: uveitis, an inflammation of the ⁢middle⁤ layer of the eye [2]. This presentation ⁤can be particularly challenging to diagnose as it can mimic other conditions. The case involved a 38-year-old man presenting with blurry vision, a skin rash, and a history of travel and sexual activity [2].

Ocular syphilis, a form ⁤of neurosyphilis,​ frequently enough ⁣presents as uveitis, which‍ can manifest as anterior, posterior, or panuveitis, retinitis, papillitis, or scleritis [2].Treatment ‍typically involves aggressive, prolonged intravenous penicillin​ [2].

It’s important to note that syphilis ⁢affects both men and women [1]. Early ⁢detection and treatment are‌ crucial to prevent progression to later,⁤ more severe stages.