Belite Bio completes submission to PMDA to initiate Dragon II trial of Tinlarebant for the treatment of adolescent Stargardt disease in Japan
Stargardt disease is the most common inherited retinal dystrophy (causing blurring or loss of central vision) in both adults and children.
(Image credit: AdobeStock/ironstealth)
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:
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
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
