Belite Bio announced that its Phase 3 DRAGON trial of Tinlarebant met the primary efficacy endpoint, showing a 36 % reduction in retinal lesion growth over 24 months compared with placebo in a cohort of 104 adolescents with Stargardt disease. The randomized, double‑masked study demonstrated a statistically significant treatment effect (p = 0.0033) in both the study eye and the fellow eye, with a 33–34 % reduction in lesion growth in the fellow eye.
The result positions Tinlarebant as the first potential oral therapy for Stargardt disease, a rare inherited macular degeneration that affects more than 50,000 patients in the United States and has no approved treatments. The drug has already received Breakthrough Therapy, Fast Track, and Rare Pediatric Disease designations in the U.S., and Orphan Drug status in the U.S., Europe, and Japan, underscoring the unmet medical need and the regulatory support for accelerated development.
Safety data from the trial were favorable, with only four treatment‑related discontinuations and no serious adverse events attributed to the drug. The safety profile, combined with the robust efficacy signal, strengthens the case for a single‑study approval pathway that could streamline regulatory review and bring the therapy to patients more quickly.
Chief Executive Officer Tom Lin said the results “mark a historic breakthrough in Stargardt disease, paving the way for the first potential treatment for this devastating condition and bringing new hope to patients and families.” Chief Medical Officer Hendrik Scholl added that the data “validate the scientific approach behind Tinlarebant’s development, demonstrating that reducing the accumulation of toxic byproducts in the retina can meaningfully slow disease progression.”
Analysts and investors reacted strongly to the data, citing the 36 % lesion‑growth reduction and the favorable safety profile as key drivers of optimism. The trial’s success also reinforces Belite Bio’s competitive position, as no other therapy has reached a pivotal study in this indication. The company’s guidance to submit regulatory filings in the first half of 2026 reflects confidence in the data and the regulatory momentum generated by the trial.
The DRAGON trial de‑risks the drug’s path to approval and opens the door to a potentially sizable market. Belite Bio’s cash runway and recent financing—$15 million in a registered direct offering and $125 million in a private placement—provide the resources needed to support the regulatory submission and subsequent commercialization activities.
Future milestones include the planned regulatory submission in early 2026, the ongoing Phase 2/3 DRAGON II trial in adolescents, and the Phase 3 PHOENIX trial for geographic atrophy in age‑related macular degeneration, which completed enrollment in July 2025. Successful outcomes in these studies could expand Tinlarebant’s indication portfolio and further strengthen the company’s market position.
In summary, Belite Bio’s Phase 3 DRAGON trial results represent a pivotal moment for the company and for patients with Stargardt disease, offering the first evidence of a meaningful, oral therapeutic option and setting the stage for a potentially transformative treatment pathway.
The content on BeyondSPX is for informational purposes only and should not be construed as financial or investment advice. We are not financial advisors. Consult with a qualified professional before making any investment decisions. Any actions you take based on information from this site are solely at your own risk.