The Case for Fludrocortisone Acetate (FA)

AMD is highly associated with activation of the immune system within the retina, which is dominated by the ‘complement system’.
In the early stages of activation of the complement system a specific cell type – the macrophage – accumulates in the macular region where they deposit the key complement protein, C3. Presence of C3-positive macrophages leads to downstream inflammatory events that are associated with death of the light-sensitive photoreceptors, and their supporting cells, the pigment epithelial cells causing blindness. This the hallmark feature of dry AMD.
In a mouse model of dry AMD, accumulation of macrophages – and thereby C3 – can be prevented by intraocular administration of fludrocortisone acetonide (FA). In these treated animals, development of dry AMD-like lesions is prevented. FA prevents macrophage activation (C3 expression) by downregulating cytokines, or pro-inflammatory chemicals, within the retina.
Treatment with FA in the mouse model of dry AMD also improves photoreceptor function, as measured using the electroretinogram (ERG).
In addition, FA boosts the functionality of cells responsible for reducing exudation and restoring a functional environment to the retina.
In human toxicity studies FA:
Has NO SIDE EFFECTS (no rise in pressure, no increased incidence of cataract)
Appears to improve night vision (low level light acuity)
Appears to slow the rate of spread of a macular lesion, compared to historical controls.

Run a Phase II, study that will demonstrate the utility of FA to manage dry AMD
Achieve FDA approval for the application of intraocular injection of FA:
To manage early AMD and prevent vision loss in the population, in the absence of serious side-effects.
For use as a co-therapy to anti-neovascular treatments (anti-VEGF) to accelerate recovery and offset refractility of those therapies.