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Belite Bio Reports Landmark Phase 3 Success For Tinlarebant In Stargardt Disease

By RTTNews Staff Writer   ✉  | Published:  | Google News Follow Us  | Join Us

Belite Bio, Inc. (BLTE) Monday announced positive topline results from its global Phase 3 DRAGON trial concerning Tinlarebant, marking the successful pivotal study in patients diagnosed with Stargardt disease type 1 (STGD1), a rare hereditary eye disorder that leads to progressive vision loss and currently lacks an approved treatment.

The trial involved 104 adolescent participants and successfully met its primary efficacy endpoint, demonstrating a 36 percent reduction in the growth rate of retinal lesions compared to placebo. Tinlarebant exhibited statistically significant advantages in both eyes and maintained a favorable safety profile, with only four patients discontinuing treatment due to adverse effects.

The company intends to submit global regulatory filings in the first half of 2026, bolstered by U.S. Breakthrough Therapy, Fast Track, and Orphan Drug designations. Chief Medical Officer Dr. Hendrik Scholl stated that the findings validate Tinlarebant's mechanism of action in reducing toxic retinal byproducts, potentially transforming the treatment landscape for inherited retinal disorders.

BLTE is currently trading at $133.23, down $4.21 or 3.06 percent on the Nasdaq.

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