Belite Bio Announces Positive Results from LBS-008 Trial for Dry AMD, Stargardt Disease

Belite Bio announced positive results from two Phase 1 clinical trials in the United States and Australia for LBS-008, a First-in-Class oral small molecule Retinol Binding Protein 4 (RBP4) specific antagonist aimed to treat atrophic Age-related Macular Degeneration (Dry AMD) and Stargardt disease, an inherited juvenile form of macular degeneration.

A total of 83 subjects were administered with LBS-008 either as a single dose or repeated doses for 14 consecutive days. Both studies concluded that pharmacological doses of LBS-008 were well tolerated and that LBS-008's PK / PD profile supports daily oral administration. Moreover, LBS-008 achieved potentially therapeutic-level target engagement.

"I was honored to have led the discovery of LBS-008 in the first and only Dry AMD project sponsored by the National Institutes of Health (NIH) Blueprint Neurotherapeutics Program (BPN). The positive results from the LBS-008 trial is a success of collaboration between NIH, Columbia University and Belite Bio. I am very excited to see LBS-008 entering the next phase and believe LBS-008 can halt the progression of Dry AMD and Stargardt disease." said Dr. Konstantin Petrukhin, Professor of Ophthalmic Science, Columbia University.

NIH BPN is focused on the development of promising treatments for nervous system disorders. "Dry AMD is a common cause of blindness for which there is no treatment," said Charles Cywin, Ph.D., program director at the NIH's National Institute of Neurological Disorders and Stroke who oversees BPN grants. "This project's transition to Phase 2 clinical trials is an example of how NIH BPN aims to help researchers translate promising ideas discovered in the lab into effective therapies for some of the most elusive neurological disorders." 

"We are pleased to have accomplished this major milestone together with the NIH BPN for which we are grateful to the BPN team for driving and supporting LBS-008's development from discovery to the completion of phase 1 that produced very promising results." said Dr. Tom Lin, CEO of Belite Bio.