Pipeline
Our lead candidate, Tinlarebant (LBS-008), was developed from our RBP4 intellectual property portfolio. It is a novel oral therapy intended to reduce the accumulation of toxic vitamin A byproducts in the retina, which are believed to drive disease progression in Stargardt disease, type 1 (STGD1), and contribute to geographic atrophy (GA). Tinlarebant has shown potential to slow the progression of STGD1 disease in our Phase 2 STGD1 study and is currently being evaluated in a Phase 3 study (DRAGON) and a Phase 2/3 study (DRAGON II) in adolescent STGD1 subjects. Tinlarebant is also being evaluated in a Phase 3 study (PHOENIX) in subjects with GA. The DRAGON and PHOENIX trials are multi-center, randomized, double‑masked, placebo-controlled studies to evaluate the safety and efficacy of Tinlarebant in patients.
Tinlarebant has been granted Orphan Drug Designation in the U.S., Europe and Japan, as well as Rare Pediatric Disease (RPD) designation and Fast Track Designation (FTD) in the U.S. and Sakigake (Pioneer Drug) Designation in Japan.
LBS-009 is an anti-RBP4 oral therapy currently in preclinical development. It targets liver disease, including non-alcoholic fatty liver disease (NAFLD), non-alcoholic steatohepatitis (NASH), and type 2 diabetes (T2D), which cumulatively impact more than 2.4 billion people worldwide. LBS-009 is a small molecule designed to compete with retinol for binding to RBP4, the primary carrier of vitamin A (retinol) from the liver to extra-hepatic target tissues. When bound to LBS-009, RBP4 can no longer bind to retinol and therefore cannot interact with a key accessory protein (transthyretin) that would otherwise prevent elimination of RBP4 from circulation. Consequently, the RBP4-LBS-009 complex gets removed from circulation by renal filtration. The available data suggest that modulation of circulating RBP4 with an RBP4 antagonist, like LBS-009, has the potential to provide a therapeutic benefit for patients suffering from metabolic diseases, including NAFLD, NASH, and T2D.
