• Prescription Drug User Fee Act target action date is February 12, 2027
• If approved, tinlarebant would be the first-ever approved treatment for Stargardt Disease Type 1
  

Belite Bio, Inc (NASDAQ: BLTE) (“Belite Bio^®” or the “Company”), a clinical-stage drug development company focused on advancing novel therapeutics targeting degenerative retinal diseases that have significant unmet medical needs, today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review designation of the New Drug Application (NDA) for tinlarebant for the treatment of Stargardt Disease Type 1 (STGD1). The FDA has set a Prescription Drug User Fee Act (PDUFA) date of February 12, 2027.

The filing was based on the results from the Phase 3 DRAGON trial, which evaluated tinlarebant for the treatment of STGD1. In the study, tinlarebant demonstrated a statistically significant and clinically meaningful 35.7% reduction in the growth rate of atrophic retinal lesions, measured as definitely decreased autofluorescence (DDAF) by fundus autofluorescence imaging, compared with placebo. Tinlarebant has been generally well tolerated in clinical trials with side effects consistent with its mechanism of action. If approved, tinlarebant would be the first ever FDA-approved treatment option for STGD1, a rare, inherited retinal disease caused by mutations in the ABCA4 gene that leads to progressive and irreversible vision loss and affects an estimated 53,000 people in the U.S. alone.

“The acceptance of our NDA with Priority Review designation underscores the immediate need among the Stargardt disease community for an approved treatment option, and we believe it reinforces the quality and depth of the data that we’ve generated,” said Dr. Tom Lin, Chairman and Chief Executive Officer of Belite Bio. “Stargardt disease typically presents early in life and leads to progressive, irreversible vision loss. We are proud that, if approved, tinlarebant could positively impact the decline in vision that was previously inevitable. We look forward to working with the FDA through its review process and remain focused on our commercial readiness and execution following potential approval.”

“The acceptance of our NDA is an important milestone driven by the clinically meaningful results from our Phase 3 DRAGON trial, which showed reduction in atrophic retinal lesion growth rate compared to placebo, supporting tinlarebant's oral, once-daily mechanism,” said Dr. Hendrik Scholl, Chief Medical Officer of Belite Bio. “As a practicing physician, I have treated people living with Stargardt disease for more than 20 years and have seen firsthand the challenges that it brings. As of now, the only options that we have to offer people as their blindness progresses are visual aids, further emphasizing the impact that tinlarebant could have on people living with this debilitating disease, if approved. We continue to see enthusiasm across the Stargardt community at the prospect of this potential treatment.”