© Reuters.
SAN DIEGO – Belite Bio, Inc (NASDAQ: BLTE), a biopharmaceutical company, has recently completed the enrollment for a pivotal global Phase 3 trial of its drug Tinlarebant, targeted at treating Stargardt Disease (STGD1), a genetic eye disorder. The trial, known as DRAGON, includes 104 adolescent subjects across 11 countries, with interim results expected in the fourth quarter of 2024.
The company also announced its submission to the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan to initiate a clinical trial named DRAGON II. This trial will assess the drug’s pharmacokinetics and pharmacodynamics in Japanese adolescent subjects with STGD1 and will include a Phase 2/3 study to evaluate efficacy, safety, and tolerability.
Tinlarebant, an orally administered tablet, aims to slow the progression of STGD1 as well as Geographic Atrophy (GA) in advanced Dry Age-related Macular Degeneration (Dry AMD (NASDAQ:)). The drug has been designed to reduce the accumulation of vitamin A-based toxins in the retina, which are implicated in these conditions.
Data from a 24-month Phase 2 trial showed a sustained lower growth of atrophic retinal lesions in subjects treated with Tinlarebant compared to a similar cohort from the ProgStar study. Additionally, 42% of subjects treated with Tinlarebant did not develop atrophic retinal lesions during the treatment period.
Tinlarebant has received Orphan Drug Designation in Japan for STGD1 treatment and has also been granted Fast Track and Rare Pediatric Disease designations in the U.S., as well as Orphan Drug Designation in the U.S. and Europe.
STGD1 is the most prevalent inherited retinal dystrophy, leading to progressive vision loss, with no FDA-approved treatments currently available. Similarly, for GA, an advanced stage of Dry AMD, there are no FDA-approved orally administered treatments.
Belite Bio focuses on advancing therapeutics for retinal degenerative diseases and specific metabolic diseases with significant unmet medical needs. This information is based on a press release statement from Belite Bio.
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