Belite Bio completes last subject visit in phase 3 Stargardt trial

SAN DIEGO - Belite Bio Inc (NASDAQ:BLTE), a $2.25 billion market cap biotech company whose stock has surged over 40% in the past year, announced the completion of the last subject visit in its Phase 3 DRAGON clinical trial evaluating Tinlarebant for the treatment of Stargardt disease type 1 (STGD1), with topline data expected in the fourth quarter of 2025.

The trial enrolled 104 adolescent subjects across 11 jurisdictions globally, with 94 subjects completing the study. Participants were randomized in a 2:1 ratio of Tinlarebant to placebo, with the final study visit conducted on September 11. According to InvestingPro data, the company maintains strong liquidity with a current ratio of 23.15, providing ample resources for its clinical development programs.

"With no approved therapies available today, Tinlarebant has the potential to be the first treatment for this devastating inherited macular degeneration," said Dr. Tom Lin, Chairman and CEO of Belite Bio, in the press release.

The primary efficacy endpoint of the DRAGON trial is the growth rate of atrophic lesions, with safety and tolerability of Tinlarebant also being assessed. The company plans to file New Drug Applications in the first half of 2026. Analysts are optimistic about the company’s prospects, setting price targets between $80 and $110. InvestingPro subscribers can access 8 additional key insights about BLTE’s financial health and market position.

Tinlarebant is an oral therapy designed to reduce the accumulation of vitamin A-based toxins called bisretinoids that cause retinal disease in STGD1. The drug works by reducing serum retinol binding protein 4 levels, which modulates the amount of retinol entering the eye.

The treatment has received multiple regulatory designations, including Breakthrough Therapy, Fast Track, and Rare Pediatric Disease Designations in the U.S.; Orphan Drug Designation in the U.S., Europe, and Japan; and Pioneer Drug Designation in Japan for Stargardt disease.

Stargardt disease is an inherited macular degeneration for which there are currently no approved treatments. The stock, which typically moves counter to the market with a beta of -1.5, has attracted investor attention as the company progresses toward potential commercialization.

In other recent news, Belite Bio has reported better-than-expected earnings per share (EPS) for the second quarter of 2025. The company posted an EPS of -0.31 USD, surpassing analyst predictions of -0.39 USD, resulting in a positive surprise of 20.51%. In addition to its financial performance, Belite Bio completed a significant $275 million private investment in public equity (PIPE) transaction, raising approximately $125 million in gross proceeds with the potential for an additional $150 million through warrant exercises. The investment was led by RA Capital Management, with participation from several other firms, including Eventide Asset Management and Marshall Wace.

Benchmark has reiterated its Buy rating on Belite Bio, maintaining a price target of $80. The firm highlighted positive developments in Belite’s clinical programs, particularly the Phase 3 DRAGON trial, which received encouraging interim data analysis results. The Data Safety Monitoring Board has recommended the company seek accelerated regulatory approval. These developments underscore the company’s ongoing efforts in advancing its clinical-stage drug development for retinal diseases.

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