Belite Bio completes submission to PMDA to initiate Dragon II trial of Tinlarebant for the treatment of adolescent Stargardt disease in Japan

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Belite Bio announced it has completed its submission to the Pharmaceuticals and Medical Devices Agency (PMDA) to initiate a clinical trial of Tinlarebant (LBS-008) for the treatment of adolescent Stargardt disease in Japan (Dragon II).

Tinlarebant is a novel oral therapy intended to reduce the accumulation of vitamin A-based toxins that cause retinal disease and also contribute to disease progression in geographic atrophy. Bisretinoids, vitamin A-based toxins, are by-products of the visual cycle, which is dependent on the supply of vitamin A (retinol) to the eye. According to the company, Tinlarebant works by reducing and maintaining levels of serum retinol binding protein 4 (RBP4), and by modulating the amount of retinol entering the eye, reduces the formation of bisretinoids.¹

A previous 24-month, Phase 2 trial of Tinlarebant in adolescent patients showed a sustained lower DDAF lesion growth in Tinlarebant-treated subjects (p<0.001), while 42% of Tinlarebant-treated subjects (5 out of 12) did not develop atrophic retinal lesions.²

The Dragon II trial is a combination of a Phase 1b open-label study evaluating the pharmacokinetics and pharmacodynamics of Tinlarebant in Japanese adolescent Stargardt disease subjects and a Phase 2/3 global study designed to evaluate the efficacy, safety and tolerability of Tinlarebant in adolescent Stargardt disease subjects. The Phase 2/3 global study is a multicenter, double-masked, placebo-controlled, randomized study.

According to the company, approximately 60 subjects, aged 12 to 20-years-old are targeted for enrollment in the Phase 2/3 portion of the trial with a 1:1 randomization. Data from Japanese subjects is intended to facilitate future NDA applications in Japan.¹

Tinlarebant has been granted Orphan Drug Designation in Japan for the treatment of Stargardt disease.¹

Stargardt disease is the most common inherited retinal dystrophy (causing blurring or loss of central vision) in both adults and children. It is caused by mutations in a retina-specific gene (ABCA4), which results in progressive accumulation of bisretinoids leading to retinal cell death and progressive loss of central vision.

References:

1. Belite Bio Announces PMDA Submission of Tinlarebant for Stargardt Disease Clinical Trial in Japan. Press release; March 22, 2024. Accessed March 25, 2024. https://investors.belitebio.com/news-releases/news-release-details/belite-bio-announces-pmda-submission-tinlarebant-stargardt

2. Belite Bio Presents Results from a 24-month, Phase 2 Study of Tinlarebant in Childhood-onset Stargardt Disease at the AAO Annual Meeting. Press release; November 6, 2023. Accessed March 25, 2024. https://investors.belitebio.com/news-releases/news-release-details/belite-bio-presents-results-24-month-phase-2-study-tinlarebant