Bringing Hope to Incurable Blindness
Zero Approved Treatments
For DRY Age-Related Macular Degeneration
Patients with age-related macular degeneration in the US
Stargardt Disease Juvenile onset macular degeneration (orphan disease)
Case of AMD worldwide with global direct healthcare cost of USD 255B
Addressable market in US alone. Total global market estimated at USD 40B
Overview:First-in-Class Oral Therapy for Treatment of Macular Degeneration
LBS-008 is a first-in-class oral therapy targeting Dry Age-Related Macular Degeneration (AMD) and Stargardt Disease, which are untreatable conditions that cause vision loss in adults and children, respectively.

LBS-008 is the most advanced and the first graduate candidate from the US National Institutes for Health’s (NIH) highly selective Blueprint Program for Neuroscience Research. The program leverages the resources of 15 NIH centers and $10M USD in funding to cover the development cost of LBS-008 through completion of Phase 1 clinical trials. LBS-008 has obtained US FDA and EMA Orphan Drug Designation in 2017/September and 2018/May respectively. LBS-008 is expected to enter first-in-human Phase 1 clinical trials in 2018.
Disease Profile:
Age-Related Macular Degeneration (AMD)
Age-related Macular Degeneration (AMD) is the leading cause of blindness in people over the age of 50 globally. While there are two forms of the disease, 90% of cases of severe vision loss are caused by dry AMD. Because no treatment currently exists, dry AMD is devastating for patients worldwide, affecting 10M people in the US alone.
Stargardt Disease
Stargardt disease is the inherited juvenile form macular degeneration, which affects 1 in 8,000-10,000 children. Patients suffer from progressive vision loss starting in childhood, with most patients becoming visually impaired by the age of 20. Currently, there is no treatment for Stargardt disease, which is an orphan indication.
Normal Retina
Healthy photoreceptors and RPE cells
RPE Changes
Dry AMD starts with insult to RPE cells Age-dependent accumulation of lipofuscin
Rods Die Cones Spared
Formation of drusen (plaque-like sub-RPE deposits) Complement activation (inflammation)
Cones Die
Progression to RPE atrophy and photoreceptor death Several potential intervention points
How it Works (Mechanism of Action):
Preventing the Toxic Accumulation of Lipofuscin
AMD is a condition that damages the retina, the light-sensitive part of the eye responsible for central vision. The inside layer of the retina is made of photoreceptors (rods and cones) that react to light and send signals to the brain. Immediately behind the layer of rods and cones is a supportive layer of retinal pigment epithelium (RPE) cells. In addition to providing photoreceptors with structural support, RPE cells also supply them with nutrients and remove waste. It is believed that AMD is caused by the abnormal buildup of a yellow pigment, called lipofuscin, on the RPE cells. As lipofuscin accumulates, it disrupts the rod and cones cells, causing them to die. Vision loss progresses with the death of photoreceptors.
Visual Cycle
11c-Ral and at-Ral are precursors to A2E, a natural and cytotoxic by-product that leads to lipofuscin accumulation in AMD and Stargardts. RBP4 propagates the cycle by mobilizing serum at-Rol into RPE.