According to an article from Yahoo! Finance, biopharmaceutical company Belite Bio, Inc. has initiated the Phase 3 DRAGON clinical study to evaluate LBS-008 for individuals with Stargardt disease. The trial began enrolling patients in late August 2022. 

Belite Bio is a subsidiary of Lin Bioscience. On its website, Lin Bioscience describes LBS-008 as: 

an oral once-a-day treatment that can reduce and maintain the delivery of vitamin A (retinol) to the eye as a means to reduce the accumulation of toxic vitamin A byproducts in ocular tissue. This effect is achieved through the ability of LBS-008 to reduce and maintain the level of serum retinol binding protein 4, or RBP4, which carries retinol from the liver to the eye.

So far, this therapy has been granted Orphan Drug, Rare Pediatric Disease, and Fast Track designations within the United States, as well as Orphan Medicinal Product designation within the European Union.

Prior to this Phase 3 clinical trial, researchers have evaluated LBS-008 in multiple Phase 1 single-ascending dose trials and a Phase 2 clinical trial. Data shows that the treatment is safe, effective, and well-tolerated. In the Phase 2 trial, LBS-008 also improved visual acuity in 61.5% of participants.

Outside of Stargardt disease, Belite Bio is also exploring LBS-008 as a possible therapy for individuals with dry age-related macular degeneration (dry AMD). Given that no FDA-approved treatments exist for either of these indications, LBS-008 has the potential to fill some hugely unmet needs. 

What is Stargardt Disease?

Stargardt disease is an inherited retinal disorder which causes progressive vision loss in children and young adults. Normally, the retina, located in the back of the eye, helps sense light; it is responsible for clear, crisp central vision. But in Stargardt disease, rods and cones in the eyes die, causing issues with seeing in dim lighting, as well as difficulties seeing details and color. ABCA4 gene mutations are the most common cause of Stargardt disease, preventing the body from clearing vitamin A byproducts from photoreceptors. While it is rare for people with this disorder to become completely blind, many people lose vision to 20/200 or worse. Symptoms can include:

• Progressive vision loss in both eyes
• Loss of peripheral vision
• Color blindness
• Involuntary eye movements
• Sensitivity to bright light
• Difficulty adjusting when moving from light to dark environment
• Gray, black, or hazy spots in your central vision