Belite Bio announced the U.S. Food and Drug Administration (FDA) has approved its Investigational New Drug application (IND) for LBS-008 (BPN-14967), a first-in-class oral therapy for the treatment of atrophic Age-related Macular Degeneration (commonly known as dry AMD) and Stargardt disease, an inherited juvenile form of macular degeneration. LBS-008 will be one of the first drugs to graduate from the US National Institute of Health's (NIH) Blueprint Neurotherapeutics Network (Blueprint Program), 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.
"Initiation of the Phase 1 trial is an important milestone for LBS-008, which was funded by the NIH Blueprint Program starting from lead optimization to a preclinical proof of concept," said Dr. Konstantin Petrukhin, Associate Professor of Ophthalmic Science at Columbia University Medical Center, and inventor of the technology. "LBS-008 is expected to bring hope to millions of patients suffering from untreatable forms of macular degeneration."
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"We are excited to announce the start of this Phase 1 trial in the US together with the NIH," said Dr. Tom Lin, Chairman of Belite Bio. "We're encouraged by the ongoing favorable regulatory view of our technology, including a systematic review published by the UK National Institute for Health Research identifying reduction of RBP4 as the most promising treatments for dry AMD and Stargardt disease. We plan to continue to work closely with our collaborators to the next clinical and regulatory milestone."
LBS-008 is a first-in-class oral therapy that prevents the buildup of toxins (A2E) in the eye that cause Stargardt disease and contribute to atrophic Age-related Macular Degeneration (dry AMD).
Belite Bio holds the worldwide exclusive rights for LBS-008. LBS-008 received US and EU orphan drug designation (ODD) in 2017 and 2018, and rare pediatric disease designation (RPD) from the FDA in 2018 for the treatment of Stargardt disease.