Dec 1 (Reuters) – Belite Bio said on Monday that its experimental drug for a rare genetic eye disease met the main goal in a late-stage study, sending its shares up over 15% in premarket trading. The drug, tinlarebant, targets Stargardt disease type 1, a progressive eye disease causing vision loss in childhood or adolescence. […]
Health
Belite Bio’s drug for genetic eye disease meets main goal in late-stage trial
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Dec 1 (Reuters) – Belite Bio said on Monday that its experimental drug for a rare genetic eye disease met the main goal in a late-stage study, sending its shares up over 15% in premarket trading.
The drug, tinlarebant, targets Stargardt disease type 1, a progressive eye disease causing vision loss in childhood or adolescence. It affects more than 50,000 patients in the United States, according to the company.
The company said the drug slowed the growth of damaging lesions in the retina by 36% compared to placebo in a study of 104 patients aged 12 to 20.
The oral medication reduces the buildup of vitamin A-based toxins in the retina by targeting the RBP4 protein that limits the transportation of vitamin A to the eye.
Tinlarebant was well tolerated with only four patients stopping treatment due to side effects, the company said.
The eye condition currently has no FDA-approved treatments.
Belite Bio plans to file for U.S. regulatory approval in the first half of 2026.
(Reporting by Padmanabhan Ananthan in Bengaluru; Editing by Vijay Kishore)