Sirolimus impresses in Ph I wet AMD trial

MacuSight, a USA-based developer of therapeutics for the treatment of severe ocular conditions, says that it has seen positive data from a Phase I trial examining a proprietary formulation of sirolimus (rapamycin) in patients with wet age-related macular edema. The drug improved visual acuity in a manner consistent with observed anatomical retinal changes after a single administration.

Sirolimus is a highly-potent, broad-acting compound that is used in a wide range of disease indications. It combines immunosuppressive properties with considerable anti-angiogenic, anti-migratory and anti-proliferative activity.

The reported trial, an open-label assessment, recruited 30 wet AMD patients who were randomized to receive one of a range of doses of the drug, administered via either subconjunctival or intravetrial injection. In addition to the improvements in visual acuity, the drug also demonstrated a favorable safety and tolerability profile and reduced retinal thickness in some subjects.