Dry Age-Related Macular Degeneration
Age-related macular degeneration (AMD) is the most common form of irreversible blindness in the world, affecting 50 million people and costing $255 billion in the United States annually in direct costs. Approximately 25% of people over 80 years of age suffer from this condition.
Dry AMD is common among people over 50 and is caused by a thinning of the macula, the part of the retina responsible for central vision. While the peripheral vision of people with dry AMD is typically unaffected, the gradual loss of central vision can present limitations on reading and driving. The amount of vision loss depends on the location and extent of protein deposits, known as drusen, that form under the macula. As the disease progresses, loss of retinal cells results in permanent vision loss known as geographic atrophy (GA).
Several key cellular processes – autophagy, protein trafficking and lipid metabolism – are known to be dysregulated in AMD, which eventually leads to irreversible damage to the retinal pigment epithelial (RPE) cells. Damage to these specialized RPE cells results in the death of photoreceptors, without which visual information cannot be transmitted to the brain.
As a selective σ-2 antagonist, CT1812 targets the receptor believed to regulate the damage-response processes that are impaired in dry AMD.
The company intends to test whether CT1812 has a beneficial effect on the damage-response processes by advancing an oral formulation into clinical trials for dry AMD in 2021.
- It has been demonstrated in animal models that oral CT1812 is delivered to the eye in quantities believed to be therapeutic.
- Cognition is currently assessing the pharmacokinetics and behavioral characteristics of CT1812.
- Safety and tolerability of CT1812 has been characterized through completed and ongoing clinical trials in the company’s Alzheimer’s disease program.
- Subject to discussion with the FDA, we plan to initiate a Phase 2 study in appx 300 adults who have a diagnosis of dry AMD in the second half of 2021.
- Anatomical and functional assessments will be measured.