Belite Bio Achieves Major Milestone With Full Patient Enrollment in DRAGON II Trial for Stargardt's Disease

Belite Bio has successfully completed enrollment in the DRAGON II clinical trial, marking a significant step forward in the development of tinlarebant, an investigational oral therapy targeting Stargardt’s disease and related retinal conditions. The company enrolled 60 adolescent participants, including 15 from Japan, across sites in the United States, United Kingdom, and Japan. As per the regulatory timeline, the company is advancing toward FDA submission of its New Drug Application in the coming months.

Understanding Stargardt’s Disease and the Treatment Challenge

Stargardt’s disease represents one of the most common forms of inherited macular degeneration, causing progressive vision loss in adolescents and young adults. The condition occurs when toxic fatty materials accumulate on the macula—the central part of the retina responsible for clear, detailed vision. Patients with Stargardt’s disease face a unique challenge: no approved treatments currently exist, leaving this patient population with limited therapeutic options. Tinlarebant offers a potential breakthrough by targeting the underlying biological mechanism driving Stargardt’s disease progression.

DRAGON II Trial: Design and Enrollment Strategy

The DRAGON II study represents a comprehensive 24-month investigation into tinlarebant’s safety and effectiveness in adolescent patients. Researchers randomized 60 subjects, aged 12 to 20 years, to receive either the active treatment or placebo in equal proportions. This double-masked, placebo-controlled approach ensures rigorous evaluation of the drug’s true clinical benefit. The geographic diversity of the trial—spanning three countries—reflects the global scope of Stargardt’s disease and strengthens the validity of the findings across different populations.

How Tinlarebant Works: The Mechanism Behind the Therapy

Tinlarebant operates by reducing the accumulation of bisretinoid toxins within the eye—the harmful substances that drive retinal damage in Stargardt’s disease patients. These toxic compounds build up progressively, leading to the characteristic vision loss. By targeting this mechanism, tinlarebant represents the first therapeutic approach to demonstrate clinical efficacy specifically for Stargardt’s disease. The drug’s potential extends beyond Stargardt’s disease; it is also being evaluated for geographic atrophy, an advanced form of age-related macular degeneration that affects millions of older adults.

Regulatory Recognition and Accelerated Development Path

Tinlarebant has received multiple designations from regulatory authorities, underscoring its significance for patients with limited treatment alternatives. In the United States, the FDA has granted Breakthrough Therapy Designation, Fast Track Designation, and Rare Pediatric Disease Designation. The drug also holds Orphan Drug Designation across the U.S., Europe, and Japan, reflecting its status as a treatment for a rare condition. Japan has further recognized tinlarebant with Sakigake Designation, a program designed to expedite the review of innovative therapies. These regulatory endorsements support the accelerated development pathway toward approval.

Previous Trial Success and Current Timeline

The DRAGON trial, the pivotal Phase 3 study conducted globally, previously demonstrated tinlarebant’s effectiveness by achieving a 36% reduction in lesion growth compared to placebo—a substantial therapeutic benefit for Stargardt’s disease patients. Additionally, Belite Bio completed a Phase 3 trial known as PHOENIX, evaluating the drug in subjects with geographic atrophy. With DRAGON II enrollment now complete, the company is positioned to submit its regulatory application within the planned timeframe, potentially bringing the first approved treatment to Stargardt’s disease patients who have waited for therapeutic options.

Implications for Patients Living With Stargardt’s Disease

The completion of DRAGON II enrollment represents more than a corporate milestone—it signifies progress toward addressing an unmet medical need for Stargardt’s disease patients globally. For adolescents experiencing progressive vision loss, tinlarebant could offer a disease-modifying treatment that slows or halts deterioration. If approved, this would mark the first disease-altering therapy specifically designed for Stargardt’s disease, transforming the treatment landscape for this patient population and offering hope to families managing this inherited retinal condition.