Highlights

  • Belite Bio completed enrollment of 60 adolescents in the Phase 2/3 DRAGON II study.
  • The trial evaluates tinlarebant in Stargardt disease patients across three countries.
  • The company plans to submit a US NDA for tinlarebant in H1 2026.

Belite Bio, Inc (NASDAQ:BLTE), a clinical-stage biotechnology company, announced the completion of patient enrollment in its Phase 2/3 DRAGON II clinical trial for tinlarebant, a treatment under investigation for Stargardt disease type 1 (STGD1). The study enrolled 60 adolescent subjects, including 15 participants from Japan. Enrollment was conducted across clinical sites in Japan, the United States, and the United Kingdom, aligning with the trial’s original design and target.

DRAGON II is structured as a 24-month, randomized, double-masked, placebo-controlled study. Participants aged 12 to 20 years were randomized in a 1:1 ratio to receive either tinlarebant or placebo. The trial is designed to assess efficacy, safety, and tolerability in adolescents diagnosed with STGD1, a rare inherited retinal disorder that leads to progressive vision loss.

Understanding Stargardt Disease and Tinlarebant
Stargardt disease is caused by genetic mutations that result in the accumulation of fatty deposits on the macula, a central region of the retina responsible for detailed vision. This buildup damages retinal cells over time and impairs central vision.

Tinlarebant, Belite Bio’s lead investigational therapy, is an oral agent intended to limit the accumulation of bisretinoid toxins in the retina. These toxins are associated with disease progression in STGD1 and also play a role in geographic atrophy, an advanced form of dry age-related macular degeneration.

Regulatory and Clinical Development Context
Belite Bio plans to submit a New Drug Application to the US Food and Drug Administration in the first half of 2026. The company has also indicated that the DRAGON II study has been structured to support regulatory requirements in Japan, enabling a parallel pathway for potential review by the Pharmaceuticals and Medical Devices Agency.

Tinlarebant has received multiple regulatory designations, including Breakthrough Therapy, Fast Track, Rare Pediatric Disease, and Orphan Drug designations in the US, along with Orphan Drug status in Europe and Japan and Sakigake designation in Japan.

Share Performance
Belite Bio shares have traded at USD 167.34, up 3.00% on Jan 27 2026.