A new drug for the treatment of atrophic age-related macular degeneration (AMD) and Stargardt disease, an inherited juvenile form of macular degeneration, has been given US Food and Drug Administration (FDA) approval for a Phase 1 trial.

Belite Bio, which is developing the drug, targets treatments for currently untreatable conditions in ophthalmology and metabolic diseases. It said FDA approval of its investigational new drug application (IND) for LBS-008 (BPN-14967), a first-in-class oral therapy, means the drug will be one of the first drugs to graduate from the US National Institute of Health's (NIH) blueprint neurotherapeutics network (Blueprint Programme), which has funded LBS-008's discovery and development, and will continue to provide support and funding until the completion of its Phase 1 single ascending dose (SAD) clinical trial.

Columbia University Associate Professor Konstantin Petrukhin, who invented the technology, said "LBS-008 is expected to bring hope to millions of patients suffering from untreatable forms of macular degeneration."

"We're encouraged by the ongoing favourable regulatory view of our technology, including a systematic review published by the UK National Institute for Health Research identifying reduction of RBP4 as one of the most promising treatments for dry AMD and Stargardt disease," said Belite Bio chair Dr Tom Lin.